https://haematologica.org/issue/feedHaematologica2024-04-01T19:37:47+00:00Ferrata Storti Foundationoffice@haematologica.orgOpen Journal Systems<p><strong>Open access journal of the Ferrata-Storti Foundation, a no profit organization. This journal was funded in 1920.</strong></p>https://haematologica.org/article/view/11514Anti-CD19 chimeric antigen receptor T-cell therapy for B-cell acute lymphoid leukemia2024-04-01T19:37:45+00:00Aaron P. Rapoportarapoport@umm.edu2024-04-01T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.283972Eligibility criteria: too big, too small or just right?2024-03-31T22:02:52+00:00Ehab Atallaheatallah@mcw.edu2023-10-12T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.284056More than a lonca-shot: beating the odds in relapsed/ refractory diffuse large B-cell lymphoma2024-03-31T22:02:53+00:00Carrie HoStephen D. Smithssmith50@fredhutch.org2023-10-05T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.284085Using machine learning to unravel the intricacy of acute myeloid leukemia2024-03-31T22:02:54+00:00Luca GuarneraValeria Viscontevisconv@ccf.org2023-10-12T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.284105MYC overexpression: adding another piece to the puzzle of high-risk mantle cell lymphoma2024-03-31T22:02:56+00:00Anita Kumarkumara@mskcc.org2023-10-19T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.284188First-line MAPK inhibition in pediatric histiocytosis: are we ready?2024-03-31T22:02:56+00:00Oussama Ablaoussama.abla@sickkids.ca2023-11-16T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.284272A key to engineering natural killer cells to attack acute myeloid leukemia2024-03-31T22:02:57+00:00Yosuke MasamotoMineo Kurokawakurokawa@m.u-tokyo.ac.jp2023-11-02T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.283966How we manage a high D-dimer2024-03-31T22:02:59+00:00Massimo FranchiniDaniele FocosiMario Piergiulio PezzoPier Mannuccio Mannuccipiermannuccio.mannucci@policlinico.mi.it<p>D-dimer, a soluble fibrin degradation product that originates from plasmin-induced degradation of cross-linked fibrin, is an important biomarker of coagulation activation and secondary fibrinolysis that is routinely used to rule out venous thromboembolism (VTE), and to evaluate the risk of VTE recurrence, as well as the optimal duration of anticoagulant therapy. Besides VTE, D-dimer may be high due to physiologic conditions, including aging, pregnancy, and strenuous physical activity. In addition, several disorders have been associated with increased D-dimer levels, ranging from disseminated intravascular coagulation to infectious diseases and cancers. Thus, it is far from unusual for hematologists to have to deal with ambulatory individuals with increased D-dimer without signs or symptoms of thrombus formation. This narrative review is dedicated to the management of these cases by the hematologist.</p>2023-10-26T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.283723Use, variability, and justification of eligibility criteria for phase II and III clinical trials in acute leukemia2024-03-31T22:03:01+00:00Andrew Hantelandrew_hantel@dfci.harvard.eduMarlise R. LuskinIrum KhanElizabeth WarnerAnand A. PatelThomas P. WalshDaniel J. DeAngeloChristopher S. LathanGregory A. Abel<p>Clinical trial eligibility criteria can unfairly exclude patients or unnecessarily expose them to known risks if criteria are not concordant with drug safety. There are few data evaluating the extent to which acute leukemia eligibility criteria are justified. We analyzed criteria and drug safety data for front-line phase II and/or III acute leukemia trials with start dates 1/1/2010-12/31/2019 registered on clinicaltrials.gov. Multivariable analyses assessed concordance between criteria use and safety data (presence of criteria with a safety signal, or absence of criteria without a signal), and differences between criteria and safety-based limits. Of 250 eligible trials, concordant use of ejection fraction criteria was seen in 34.8%, corrected QT level (QTc) in 22.4%, bilirubin in 68.4%, aspartate transaminase/alanine aminotransferase (AST/ALT) in 58.8%, renal function in 68.4%, human immunodeficiency virus (HIV) in 54.8%, and hepatitis B and C in 42.0% and 41.2%. HIV and hepatitis B and C criteria use was concordant with safety data (adjusted Odds Ratios 2.04 [95%CI: 1.13, 3.66], 2.64 [95%CI: 1.38, 5.04], 2.27 [95%CI: 1.20, 4.32]) but organ function criteria were not (all P>0.05); phase III trials were not more concordant. Bilirubin criteria limits were the same as safety-based limits in 16.0% of trials, AST/ALT in 18.1%, and renal function in 13.9%; in 75.7%, 51.4%, and 56.5% of trials, criteria were more restrictive, respectively, by median differences of 0.2, 0.5, and 0.5 times the upper limits of normal. We found limited drug safety justifications for acute leukemia eligibility criteria. These data define criteria use and limits that can be rationally modified to increase patient inclusion and welfare.</p>2023-08-10T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.282761Association of <i>NUDT15</i> gene polymorphism with adverse reaction, treatment efficacy, and dose of 6-mercaptopurine in patients with acute lymphoblastic leukemia: a systematic review and meta-analysis2024-03-31T22:03:04+00:00Shan DuXuefei HuangXia HeMian MaoMin ChenRong ZhangHuikai ShaoZiyan LvXinxia Liucupflysea@163.comJunlan Chuanchuanjunlan@foxmail.com<p>6-mercaptopurine (6-MP) serves as the backbone in the maintenance regimens of acute lymphoblastic leukemia (ALL). We aimed to evaluate the influence of <em>NUDT15</em> gene polymorphism on the risk of myelosupression, hepatotoxicity and interruption of 6-MP, as well as treatment efficacy and dose of 6-MP in ALL patients. A total of 24 studies with 3,374 patients were included in this meta-analysis. We found 9-fold higher risk of 6-MP induced leukopenia (odds ratio [OR] =9.00, 95% confidence interval [CI]: 3.73-21.74) and 2.5-fold higher risk of 6-MP-induced neutropenia (OR=2.52, 95% CI: 1.72-3.69) for <em>NUDT15</em> c.415C>T variant carriers in the dominant model. Moreover, we found that the dose intensity of 6-MP in ALL patients with one NUDT15 c.415C>T variant alleles (CT) was 19% less than that in wild-type patients (CC) (mean differences: 19.43%, 95% CI: -25.36 to -13.51). The tolerable dose intensity of 6-MP in <em>NUDT15</em> c.415C>T homozygote variant (TT) and heterozygote variant (CT) carriers was 49% and 15% less than that in wild-type patients, respectively. The <em>NUDT15</em> c.415C>T variant group (CT+TT) had seven times (OR=6.98, 95% CI: 2.83-17.22) higher risk of developing 6-MP intolerance than the CC group. However, <em>NUDT15</em> c.415C>T polymorphism did not appear significantly associated with hepatotoxicity, treatment interruption or relapse incidence. We concluded that <em>NUDT15</em> c.415C>T was a good predictor for 6-MP-induced myelosuppression in ALL patients. The dose intensity of 6-MP in ALL patients with <em>NUDT15</em> c.415C>T variants was significantly lower than that in wild-type patients. This research provided a basis for further investigation into relations between <em>NUDT15</em> gene and adverse reaction, treatment efficacy and dose intensity of 6-MP.</p>2023-10-05T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.283613Susceptibility of pediatric acute lymphoblastic leukemia to STAT3 inhibition depends on p53 induction2024-03-31T22:03:07+00:00Luca GasparoliClemence VirelyAlexia TsakaneliNoelia CheDarren EdwardsJack BartramMichael HubankDeepali PalOlaf HeidenreichJoost H.A. MartensJasper de BoerOwen Williamsowen.williams@ucl.ac.uk<p>Advances in the clinical management of pediatric B-cell acute lymphoblastic leukemia (B-ALL) have dramatically improved outcomes for this disease. However, relapsed and high-risk disease still contribute to significant numbers of treatment failures. Development of new, broad range therapies is urgently needed for these cases. We previously reported the susceptibility of <em>ETV6-RUNX1<sup>+</sup></em> pediatric B-ALL to inhibition of signal transducer and activator of transcription 3 (STAT3) activity. In the present study, we demonstrate that pharmacological or genetic inhibition of STAT3 results in p53 induction and that CRISPR-mediated <em>TP53</em> knockout substantially reverses susceptibility to STAT3 inhibition. Furthermore, we demonstrate that sensitivity to STAT3 inhibition in patient-derived xenograft (PDX) B-ALL samples is not restricted to any particular disease subtype, but rather depends on TP53 status, the only resistant samples being <em>TP53</em> mutant. Induction of p53 following STAT3 inhibition is not directly dependent on MDM2 but correlates with degradation of MDM4. As such, STAT3 inhibition exhibits synergistic in vitro and in vivo anti-leukemia activity when combined with MDM2 inhibition. Taken together with the relatively low frequency of <em>TP53</em> mutations in this disease, these data support the future development of combined STAT3/ MDM2 inhibition in the therapy of refractory and relapsed pediatric B-ALL.</p>2023-10-05T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.283437Extended exposure to low doses of azacitidine induces differentiation of leukemic stem cells through activation of myeloperoxidase2024-03-31T22:03:10+00:00Danny V. JeyarajuMaryam AlapaAnn PolonskaiaAlberto RisueñoPrakash SubramanyamAmit AnandKaushik GhoshCharalampos KyriakopoulosDaiane HemerichRose HurrenXiaoming WangMarcela GrondaAarif AhsanHsiling ChiuGeethu ThomasEvan F. LindDaniel L MenezesAaron D. SchimmerPatrick R. HagnerAnita GandhiAnjan G. Thakurtaanjan.thakurta@ndorms.ox.ac.uk<p>Oral azacitidine (oral-Aza) treatment results in longer median overall survival (OS) (24.7 vs. 14.8 months in placebo) in patients with acute myeloid leukemia (AML) in remission after intensive chemotherapy. The dosing schedule of oral-Aza (14 days/28-day cycle) allows for low exposure of Aza for an extended duration thereby facilitating a sustained therapeutic effect. However, the underlying mechanisms supporting the clinical impact of oral-Aza in maintenance therapy remain to be fully understood. In this preclinical work, we explore the mechanistic basis of oral-Aza/extended exposure to Aza through in vitro and in vivo modeling. In cell lines, extended exposure to Aza results in sustained DNMT1 loss, leading to durable hypomethylation, and gene expression changes. In mouse models, extended exposure to Aza, preferentially targets immature leukemic cells. In leukemic stem cell (LSC) models, the extended dose of Aza induces differentiation and depletes CD34+CD38- LSC. Mechanistically, LSC differentiation is driven in part by increased myeloperoxidase (MPO) expression. Inhibition of MPO activity either by using an MPO-specific inhibitor or blocking oxidative stress, a known mechanism of MPO, partly reverses the differentiation of LSC. Overall, our preclinical work reveals novel mechanistic insights into oral-Aza and its ability to target LSC.</p>2023-11-09T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.283606Prognostic value of European LeukemiaNet 2022 criteria and genomic clusters using machine learning in older adults with acute myeloid leukemia2024-03-31T22:03:14+00:00Silvia ParkTong Yoon KimByung-Sik Chocbscho@catholic.ac.krDaehun KwagJong-Mi LeeMyungShin KimYonggoo KimJamin KooAnjali RamanTae Kon KimHee-Je Kim<p>This study aimed to validate the new European Leukemia Net (ELN) 2022 criteria for genetic risk stratification in older adults with acute myeloid leukemia (AML) and to determine the most likely set of clusters of similar cytogenetic and mutation properties correlated with survival outcomes in three treatment groups: intensive chemotherapy (IC), hypomethylating agents (HMA) alone, and HMA plus venetoclax (HMA/VEN). The study included 279 patients (aged ≥60 years) who received IC (N=131), HMA (N=76), and HMA/VEN (N=72) between July 2017 and October 2021. No significant differences were observed in survival among the groups according to ELN 2022 risk stratification. Unsupervised hierarchical clustering analysis identified nine genomic clusters (C1-9) with varying survival outcomes depending on treatment type. For example, C4 (predominant for core binding factor-AML) displayed a favorable prognosis in the IC group, but not in the HMA or HMA/VEN groups. The HMA/VEN group had better outcomes than the HMA group in many clusters (C1, 2, 3, and 5); however, the addition of VEN to HMA or IC did not improve the survival outcomes compared with those of HMA alone in C7 and C9 (predominant for -5, del(5q), -7, -17/abn(17p), complex karyotypes, and mutated TP53). The study highlights the limitations of ELN genetic risk stratification in older adults with AML. It emphasizes the need for a more comprehensive approach that considers co-occurring somatic mutations to guide treatment selection in older adults with AML.</p>2023-09-14T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.282915Immune checkpoint molecule DNAM-1/CD112 axis is a novel target for natural killer-cell therapy in acute myeloid leukemia2024-03-31T22:03:17+00:00Yuta KaitoEmi SugimotoFumi NakamuraYutaka TsukuneMakoto SasakiShunsuke YuiHiroki YamaguchiSusumu GoyamaYasuhito NannyaKinuko MitaniHideto TamuraYoichi Imaiimaiyo-tky@umin.ac.jp<p>Acute myeloid leukemia (AML) is a hematologic malignancy that frequently relapses, even if remission can be achieved with intensive chemotherapy. One known relapse mechanism is the escape of leukemic cells from immune surveillance. Currently, there is no effective immunotherapy for AML because of the lack of specific antigens. Here, we aimed to elucidate the association between CD155 and CD112 in AML cell lines and primary AML samples and determine the therapeutic response. Briefly, we generated NK-92 cell lines (NK-92) with modified DNAX-associated molecule 1 (DNAM-1) and T-cell immunoglobulin and ITIM domain (TIGIT), which are receptors of CD155 and CD112, respectively. Analysis of 200 cases of AML indicated that the survival of patients with high expression of CD112 was shorter than that of patients with low expression. NK-92 DNAM-1 exhibited enhanced cytotoxic activity against AML cell lines and primary cells derived from patients with AML. DNAM-1 induction in NK-92 cells enhanced the expression of cytotoxicity-related genes, thus overcoming the inhibitory activity of TIGIT. Between CD155 and CD112, CD112 is an especially important target for natural killer (NK)-cell therapy of AML. Using a xenograft model, we confirmed the enhanced antitumor effect of NK-92 DNAM-1 compared with that of NK-92 alone. We also discovered that CD112 (Nectin-2), an immune checkpoint molecule belonging to the Nectin/Nectin-like family, functions as a novel target of immunotherapy. In conclusion, modification of the DNAM-1/CD112 axis in NK cells may be an effective novel immunotherapy for AML. Furthermore, our findings suggest that the levels of expression of these molecules are potential prognostic markers in AML.</p>2023-09-21T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.283812HLA-C*04:09N is expressed at the cell surface and triggers peptide-specific T-cell activation2024-03-31T22:03:20+00:00Carlotta WeltersMarthe-Lina WeltersSerena StadlerLars BullingerJulian StrobelHolger HacksteinArunraj DhamodaranThomas BlankensteinLeo Hansmannleo.hansmann@ukr.de<p>The null allele HLA-C*04:09N differs from HLA-C*04:01 in a frameshift mutation within its cytoplasmic domain, resulting in translation of 32 additional amino acids that are assumed to prevent cell surface expression. However, we recently identified a multiple myeloma-reactive T-cell receptor (TCR) that appeared to recognize antigen presented on HLA-C*04:09N and encouraged us to ask whether HLA-C*04:09N, albeit not easily detectable at the cell surface, can present antigen sufficient for T-cell activation. We generated two HLA-class I-deficient cell lines, re-expressed HLAC* 04:09N, detected HLA expression by flow cytometry, and tested for T-cell activation using a cytomegalovirus peptide- specific HLA-C*04:01-restricted TCR. In both cell lines, HLA-C*04:09N expression was detectable at the cell surface and could be enhanced by IFN-γ exposure. Recombinant HLA-C*04:09N expression was sufficient for T-cell activation in vitro, which could be blocked by an HLA-class I-specific antibody, suggesting HLA-TCR interaction at the cell surface. Peripheral blood mononuclear cells isolated from an individual who physiologically expressed HLA-C*04:09N triggered peptide-specific T-cell activation, confirming our results with cells with natural HLA expression levels. In conclusion, we present peptide-specific HLA-C*04:09N-restricted T-cell activation and suggest consideration of this allele in the appropriate clinical context, such as allogeneic stem cell transplantation, or in the setting of cellular therapy.</p>2023-09-28T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.283581Tissue factor pathway inhibitor is associated with risk of venous thromboembolism and all-cause mortality in patients with cancer2024-03-31T22:03:23+00:00Cornelia EnglischFlorian MoikJohannes ThalerSilvia KoderNigel MackmanMatthias PreusserIngrid PabingerCihan Aycihan.ay@meduniwien.ac.at<p>Venous thromboembolism (VTE) is a common complication in patients with cancer. Data on the role of natural inhibitors of coagulation for occurrence of cancer-associated VTE are limited, thus, we investigated the association of tissue factor pathway inhibitor (TFPI) with risk of VTE and all-cause mortality in patients with cancer. Total TFPI antigen levels were measured with a commercially available enzyme-linked immunosorbant assay in patients included in the Vienna Cancer and Thrombosis Study, a prospective observational cohort study with the primary outcome VTE. Competing risk analysis and Cox regression analysis were performed to explore the association of TFPI levels with VTE and all-cause mortality. TFPI was analyzed in 898 patients (median age 62 years; interquartile range [IQR], 53-68; 407 (45%) women). Sixty-seven patients developed VTE and 387 died (24-month cumulative risk 7.5% and 42.1%, respectively). Patients had median TFPI levels at study inclusion of 56.4 ng/mL (IQR, 45.7-70.0), with highest levels in tumor types known to have a high risk of VTE (gastroesophageal, pancreatic and brain cancer: 62.0 ng/mL; IQR, 52.0-75.0). In multivariable analysis adjusting for age, sex, cancer type and stage, TFPI levels were associated with VTE risk (subdistribution hazard ratio per doubling =1.63, 95% confidence interval [CI]: 1.03-2.57). When patients with high and intermediate/low VTE risk were analyzed separately, the association remained independently associated in the high risk group only (subdistribution hazard ratio =2.63, 95% CI: 1.40-4.94). TFPI levels were independently associated with all-cause mortality (hazard ratio =2.36, 95% CI: 1.85-3.00). In cancer patients increased TFPI levels are associated with VTE risk, specifically in patients with high-risk tumor types, and with all-cause mortality.</p>2023-10-12T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.283295Dabrafenib and trametinib in Langerhans cell histiocytosis and other histiocytic disorders2024-03-31T22:03:26+00:00Eily CournoyerJustin FerrellSusan SharpAnish RayMichael JordanChristopher DandoyMichael GrimleySomak RoyRobert LorsbachArnold C. MerrowAdam NelsonAllison BartlettJennifer PicarsicAshish KumarAshish.kumar@cchmc.org<p>The standard treatment for Langerhans cell histiocytosis (LCH) is chemotherapy, although the failure rates are high. Since MAP-kinase activating mutations are found in most cases, BRAF- and MEK-inhibitors have been used successfully to treat patients with refractory or relapsed disease. However, data on long-term responses in children are limited and there are no data on the use of these inhibitors as first-line therapy. We treated 34 patients (26 with LCH, 2 with juvenile xanthogranuloma, 2 with Rosai-Dorfman disease, and 4 with presumed single site-central nervous system histiocytosis) with dabrafenib and/or trametinib, either as first line or after relapse or failure of chemotherapy. Sixteen patients, aged 1.3-21 years, had disease that was recurrent or refractory to chemotherapy, nine of whom had multisystem LCH with risk-organ involvement. With a median treatment duration of 4.3 years, 15 (94%) patients have sustained favorable responses. Eighteen patients, aged 0.2-45 years, received an inhibitor as first-line treatment. All of these have had sustained favorable responses, with a median treatment duration of 2.5 years. Three patients with presumed isolated central nervous system/pituitary stalk histiocytosis had stabilization or improvement of their disease. Overall, inhibitors were well tolerated. Five patients with single-system LCH discontinued therapy and remain off therapy without recurrence. In contrast, all four patients with multisystem disease who discontinued therapy had to restart treatment. Our data suggest that children suffering from histiocytoses can be treated safely and effectively with dabrafenib or trametinib. Additional studies are, however, needed to determine the long-term safety and optimal duration of therapy.</p>2023-09-21T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.282898Immunochemotherapy plus lenalidomide for high-risk mantle cell lymphoma with measurable residual disease evaluation2024-03-31T22:03:30+00:00Zachary D. Epstein-PetersonEsther DrillUmut AyparConnie Lee BatleviPhilip CaronAhmet DoganPamela DrullinskyJohn GerecitanoPaul A. HamlinCaleb HoAllison JacobAshlee JosephLeana LaraqueMatthew J. MatasarAlison J. MoskowitzCraig H. MoskowitzChelsea MullinsColette OwensGilles SallesHeiko SchöderDavid J. StrausAnas YounesAndrew D. ZelenetzAnita Kumarkumara@mskcc.org<p>Chemoimmunotherapy followed by consolidative high-dose therapy with autologous stem cell rescue was a standard upfront treatment for fit patients with mantle cell lymphoma (MCL) in first remission; however, treatment paradigms are evolving in the era of novel therapies. Lenalidomide is an immunomodulatory agent with known efficacy in treating MCL. We conducted a single-center, investigator-initiated, phase II study of immunochemotherapy incorporating lenalidomide, without autologous stem cell transplant consolidation, enriching for patients with high-risk MCL (clinicaltrials gov. Identifier: NCT02633137). Patients received four cycles of lenalidomide-R-CHOP, two cycles of R-HiDAC, and six cycles of R-lenalidomide. The primary endpoint was rate of 3-year progression-free survival. We measured measurable residual disease (MRD) using a next-generation sequencing-based assay after each phase of treatment and at 6 months following end-oftreatment. We enrolled 49 patients of which 47 were response evaluable. By intent-to-treat, rates of overall and complete response were equivalent at 88% (43/49), one patient with stable disease, and two patients had disease progression during study; 3-year progression-free survival was 63% (primary endpoint not met) and differed by TP53 status (78% wild-type vs. 38% ALT; P=0.043). MRD status was prognostic and predicted long-term outcomes following R-HiDAC and at 6 months following end-of-treatment. In a high-dose therapy-sparing, intensive approach, we achieved favorable outcomes in TP53- wild-type MCL, including high-risk cases. We confirmed that sequential MRD assessment is a powerful prognostic tool in patients with MCL.</p>2023-08-31T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.283174Outcome of combined modality treatment in first-line for stage I(E) peripheral T-cell lymphoma; a nationwide population-based cohort study from the Netherlands2024-03-31T22:03:34+00:00Frederik O. MeeuwesMirian BrinkWouter PlattelMarjolein W.M. van der PoelMarie José KerstenMariëlle WondergemLara BöhmerF.J. Sherida H. Woei-A-JinOtto VisserRimke OostvogelsPatty M. JansenKaren J. NeelisAnne P.G. CrijnsLaurien A. DaniëlsTjeerd J.F. SnijdersJoost S.P. VermaatGerwin A. HulsMarcel Nijlandm.nijland@umcg.nl<p>Peripheral T-cell lymphomas (PTCL) comprise a heterogeneous group of mature T-cell neoplasms with an unfavorable prognosis; presentation with stage I(E) disease is uncommon. In clinical practice, an abbreviated chemotherapy treatment regimen combined with radiotherapy (combined modality treatment [CMT]) is commonly used, although evidence from clinical trials is lacking. The aim of this nationwide population-based cohort study is to describe first-line treatment and outcome of patients with stage I(E) PTCL. All newly diagnosed patients ≥18 years with stage I(E) anaplastic large cell lymphoma (ALCL), angioimmunoblastic T-cell lymphoma (AITL) and peripheral T-cell lymphoma NOS (PTCL not otherise specified [NOS]) in 1989-2020 were identified in the Netherlands Cancer Registry. Patients were categorized according to treatment regimen, i.e., chemotherapy (CT), radiotherapy (RT), CMT, other therapy and no treatment. The primary endpoint was overall survival (OS). Patients with stage I(E) ALCL, AITL and PTCL NOS (n=576) were most commonly treated with CMT (28%) or CT (29%), 2% underwent SCT. RT only was given in 18%, and 8% received other therapy and 16% no treatment. Overall, the 5-year OS was 59%. According to subtype, 5-year OS was superior for ALCL as compared to PTCL NOS and AITL (68% vs. 55% and 52%, respectively; P=0.03). For patients treated with CMT, 5-year OS was significantly higher (72%) as compared to patients treated with either CT or RT alone (55% and 55%, respectively; P<0.01). In multivariable analysis, age per year increment (hazard ratio [HR] =1.06, 95% confidence interval [CI]: 1.05-1.07), male sex (HR=1.53, 95% CI: 1.23-1.90), and CT, or no treatment (HR=1.64, 95% CI: 1.21-2.21, and HR=1.55, 95% CI: 1.10-2.17, respectively) were associated with a higher risk of mortality. For stage I(E) ALCL, AITL and PTCL NOS, 5-year OS is 59%, comparing favorably to historical outcome in advanced-stage disease. Superior outcome estimates were observed in patients treated with CMT.</p>2023-10-05T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.283352MYC protein is a high-risk factor in mantle cell lymphoma and identifies cases beyond morphology, proliferation and <i>TP53</i>/p53 – a Nordic Lymphoma Group study2024-03-31T22:03:38+00:00Joana M. RodriguesPeter HollanderLina SchmidtEirinaios GkikaMasoud RazmaraDarshan KumarChristian GeislerKirsten GrønbækChristian W. EskelundRiikka RätyArne KolstadChrister SundströmIngrid GlimeliusAnna PorwitMats JerkemanSara Eksara.ek@immun.lth.se<p>The transcription factor <em>MYC</em> is a well-described oncogene with an important role in lymphomagenesis, but its significance for clinical outcome in mantle cell lymphoma (MCL) remains to be determined. We performed an investigation of the expression of MYC protein in a cohort of 251 MCL patients complemented by analyses of structural aberrations and mRNA, in a sub-cohort of patients. Fourteen percent (n=35) of patients showed high <em>MYC</em> protein expression with >20% positive cells (MYC<sup>high</sup>), among whom only one translocation was identified, and 86% (n=216) of patients showed low MYC protein expression. Low copy number gains of MYC were detected in ten patients, but with no correlation to <em>MYC</em> protein levels. However, MYC mRNA levels correlated significantly to <em>MYC</em> protein levels with a R2 value of 0.76. Patients with a MYC<sup>high</sup> tumor had both an independent inferior overall survival and an inferior progression-free survival (hazard ratio [HR]=2.03, 95% confidence interval [95% CI]: 1.2-3.4 and HR=2.2, 95% CI: 1.04-4.6, respectively) when adjusted for additional high-risk features. Patients with MYChigh tumors also tended to have additional high-risk features and to be older at diagnosis. A subgroup of 13 patients had concomitant MYC<sup>high</sup> expression and <em>TP53</em>/p53 alterations and a substantially increased risk of progression (HR=16.9, 95% CI: 7.4-38.3) and death (HR=7.8, 95% CI: 4.4-14.1) with an average overall survival of only 0.9 years. In summary, we found that at diagnosis a subset of MCL patients (14%) overexpressed MYC protein, and had a poor prognosis but that MYC rearrangements were rare. Tumors with concurrent MYC overexpression and <em>TP53</em>/p53 alterations pinpointed MCL patients with a dismal prognosis with a median overall survival of less than 3 years. We propose that MYC needs to be assessed beyond the current high-risk factors in MCL in order to identify cases in need of alternative treatment.</p>2023-08-31T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.283459Loncastuximab tesirine in relapsed/refractory diffuse large B-cell lymphoma: long-term efficacy and safety from the phase II LOTIS-2 study2024-03-31T22:03:41+00:00Paolo F. Caimicaimip@ccf.orgWeiyun Z. AiJuan Pablo AlderuccioKirit M. ArdeshnaMehdi HamadaniBrian HessBrad S. KahlJohn RadfordMelhem SolhAnastasios StathisPier Luigi ZinzaniYing WangYajuan QinLuqiang WangZhiying Cindy XuCarmelo Carlo-Stella<p>Therapies that demonstrate durable, long-term responses with manageable safety and tolerability are needed for patients with relapsed/refractory diffuse large B-cell lymphoma (R/R DLBCL). Loncastuximab tesirine (loncastuximab tesirine-lpyl [Lonca]), an anti-CD19 antibody conjugated to a potent pyrrolobenzodiazepine dimer, demonstrated single-agent antitumor activity in the pivotal phase II LOTIS-2 study in heavily pretreated patients with R/R DLBCL. Here we present updated efficacy and safety analyses from LOTIS-2, performed for all patients and in subsets of patients with a complete response (CR), including patients with CR who were event-free (no progressive disease or death) for ≥1 year and ≥2 years from cycle 1, day 1 of treatment. Lonca was administered every 3 weeks (0.15 mg/kg for 2 cycles; 0.075 mg/kg for subsequent cycles). As of the final data cutoff (September 15, 2022; median follow-up: 7.8 months [range, 0.3-42.6]), 70 of 145 (48.3%) patients achieved an overall response. Thirty-six (24.8%) patients achieved CR, of which 16 (44%) and 11 (31%) were event-free for ≥1 year and ≥2 years, respectively. In the all-treated population, the median overall survival was 9.5 months; the median progression-free survival was 4.9 months. Among patients with CR, median overall survival and progression-free survival were not reached, with 24-month overall and progression-free survival rates of 68.2% (95% CI: 50.0-81.0) and 72.5% (95% CI: 48.2-86.8), respectively. No new safety concerns were detected. With additional follow-up, Lonca continued to demonstrate durable, long-term responses with manageable safety and tolerability in patients with CR (clinicaltrials gov. Identifier: NCT03589469).</p>2023-08-31T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.283557Polatuzumab vedotin plus bendamustine and rituximab or obinutuzumab in relapsed/refractory follicular lymphoma: a phase Ib/II study2024-03-31T22:03:44+00:00Christopher R. FlowersCRFlowers@mdanderson.orgMatthew J. MatasarAlex F. HerreraMark HertzbergSarit AssoulineJudit DemeterAndrew McMillanAmitkumar MehtaStephen OpatMarek TrnňnýLisa MusickJamie HirataAnnie YangLaurie H. Sehn<p>Follicular lymphoma (FL) is the most common type of indolent non-Hodgkin lymphoma. Despite treatment advances that have improved outcomes for patients with relapsed or refractory (R/R) FL, many patients still die from progressive disease or treatment-related toxicities. In the phase Ib/II GO29365 study (clinicaltrials.gov 02257567), the safety and efficacy of polatuzumab vedotin plus bendamustine and rituximab (Pola-BR) versus bendamustine and rituximab (BR) alone, and polatuzumab vedotin plus bendamustine and obinutuzumab (Pola-BG) as a single-arm cohort were evaluated in patients with R/R FL. Following the phase Ib safety run-in, patients were randomized 1:1 to receive Pola-BR or BR alone in the phase II stage; a separate non-randomized Pola-BG cohort was examined in the phase Ib/II expansion stage. Primary endpoints included safety and tolerability (phase Ib) and positron emission tomography complete response (PET-CR) rate by independent review committee (phase II). Overall, 112 patients were enrolled (phase Ib safety run-in: Pola-BR, N=6; phase II randomized cohort: Pola-BR, N=39; BR, N=41; phase Ib/II expansion cohort: Pola-BG, N=26). PET-CR rates were 66.7% (phase Ib safety run-in, Pola-BR); 69.2% (phase II randomized, Pola-BR); 63.4% (phase II randomized, BR); and 65.4% (phase Ib/II expansion Pola-BG). There was a higher occurrence of cytopenias with Pola-BR and Pola-BG than with BR; serious adverse events were more frequent with Pola-BR (61.4%) and Pola-BG (46.2%) than with BR (29.3%). Overall, this analysis does not demonstrate a benefit of adding Pola to BR or BG regimens for patients with R/R FL.</p>2023-09-28T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.282789Ribosomal protein S3 mediates drug resistance of proteasome inhibitor: potential therapeutic application in multiple myeloma2024-03-31T22:03:47+00:00Gege ChenXuejie GaoXinyan JiaYingcong WangLi XuDandan YuShuaikang ChangHui DengKe HuGuanli WangBo LiZhijian XuYumeng LuHuaping WangTing ZhangDongliang SongGuang YangXiaosong WuHuabin ZhuWeiliang Zhuwlzhu@simm.ac.cnJumei Shishijumei@tongji.edu.cn<p>Multiple myeloma (MM) remains incurable due to drug resistance. Ribosomal protein S3 (RPS3) has been identified as a non-Rel subunit of NF-κB. However, the detailed biological roles of RPS3 remain unclear. Here, we report for the first time that RPS3 is necessary for MM survival and drug resistance. RPS3 was highly expressed in MM, and knockout of RPS3 in MM inhibited cell growth and induced cell apoptosis both in vitro and in vivo. Overexpression of RPS3 mediated the proteasome inhibitor resistance of MM and shortened the survival of MM tumor-bearing animals. Moreover, our present study found an interaction between RPS3 and the thyroid hormone receptor interactor 13 (TRIP13), an oncogene related to MM tumorigenesis and drug resistance. We demonstrated that the phosphorylation of RPS3 was mediated by TRIP13 via PKCδ, which played an important role in activating the canonical NF-κB signaling and inducing cell survival and drug resistance in MM. Notably, the inhibition of NF-κB signaling by the small-molecule inhibitor targeting TRIP13, DCZ0415, was capable of triggering synergistic cytotoxicity when combined with bortezomib in drug-resistant MM. This study identifies RPS3 as a novel biomarker and therapeutic target in MM.</p>2023-09-28T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.283062Single-cell analysis of the CD8<sup>+</sup> T-cell compartment in multiple myeloma reveals disease specific changes are chiefly restricted to a CD69<sup>-</sup> subset suggesting potent cytotoxic effectors exist within the tumor bed2024-03-31T22:03:51+00:00James Favalorojames.favaloro@health.nsw.gov.auChristian E. Bryantchristian.bryant@health.nsw.gov.au.Edward AbadirSamuel GardinerShihong YangTracy KingNajah NassifLisa M. SedgerRichard BoyleDouglas E. JoshuaP. Joy Ho<p>Multiple myeloma (MM) is an incurable disease of the bone marrow (BM) characterized by the uncontrolled proliferation of neoplastic plasma cells. While CD8<sup>+</sup> T cells have an established role in disease control, few studies have focused on these cells within the MM tumor microenvironment (TME). We analyzed CD8<sup>+</sup> T cells in the BM and peripheral blood (PB) of untreated patients with MM and non-myeloma controls using flow cytometry, mass cytometry and single-cell RNA sequencing, using several novel bioinformatics workflows. Inter-tissue differences were most evident in the differential expression of Granzymes B and K, which were strongly associated with two distinct subsets of CD8<sup>+</sup> T cells delineated by the expression of CD69, accounting for roughly 50% of BM-CD8<sup>+</sup> T cells of all assessed cohorts. While few differences were observable between health and disease in the BM-restricted CD8CD69<sup>+</sup> T-cell subset, the CD8<sup>+</sup>CD69<sup>-</sup> T-cell subset in the BM of untreated MM patients demonstrated increased representation of highly differentiated effector cells and evident compositional parallels between the PB, absent in age-matched controls, where a marked reduction of effector cells was observed. We demonstrate the transcriptional signature of BM-CD8<sup>+</sup> T cells from patients with MM more closely resembles TCR-activated CD8<sup>+</sup> T cells from age-matched controls than their resting counterparts.</p>2023-10-05T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.283698Targeting T<sub>FH</sub> cells is a novel approach for donor-specific antibody desensitization of allograft candidates: an <i>in vitro</i> and <i>in vivo</i> study2024-03-31T22:03:55+00:00Ning MaWei-Bing WuXiang-Yu ZhaoLan-Ping XuXiao-Hui ZhangYu WangXiao-Dong MoYuan-Yuan ZhangXiao-Su ZhaoYu-Qian SunYi-Fei ChengKai-Yan LiuYing-Jun Changrmcyj@bjmu.edu.cnXiao-Jun Huangxjhrm@medmail.com.cn<p>The presence of donor-specific antibodies (DSA) are associated with graft failure either following human leukocyte antigen (HLA)-mismatched allogeneic stem cell transplantation or after organ transplantation. Although targeting B cells and plasma cells have been used for desensitization, there have been reports of failure. T-follicular helper (T<sub>fh</sub>) cells assist B cells in differentiating into antibody-secreting plasma cells. We used haploidentical allograft as a platform to investigate the possibility of targeting T<sub>fh</sub> cells to desensitize DSA. The quantities of circulating Tfh (cT<sub>fh</sub>) cell subsets in allograft candidates were abnormal, and these cells, including the cT<sub>fh</sub>2 and cT<sub>fh</sub>em cell subsets, were positively related to the production of anti-HLA antibodies. <em>Ex vivo</em> experiments showed that the cT<sub>fh</sub> cells of anti-HLA antibody-positive allograft candidates could induce B cells to differentiate into DSA-producing plasmablasts. The immune synapse could be involved in the assistance of cT<sub>fh</sub> cells to B cells in antibody production. <em>In vitro</em> experiments and<em> in vivo</em> clinical pilot studies indicated that targeting cTfh cells with sirolimus can inhibit their auxiliary function in assisting B cells. <em>Ex vivo</em> and <em>in vivo</em> studies demonstrated the effect of sirolimus and rituximab on DSA desensitization compared with either sirolimus or rituximab alone (60%, 43.75%, and 30%, respectively). Our findings provide new insight into the role of T<sub>fh</sub> cells in the pathogenesis of DSA production in HLA-mismatched transplant candidates. Our data also indicate that targeting Tfh cells is a novel strategy for DSA desensitization and combination of sirolimus and rituximab might be a potential therapy. The prospective cohort of this study is registered at http://www.chictr.org.cn as #ChiCTR-OPC-15006672.</p>2023-10-12T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.282914Genome-wide assessment of genetic risk loci for childhood acute lymphoblastic leukemia in Japanese patients2024-03-31T22:04:00+00:00Mayumi HangaiTakahisa KawaguchiMasatoshi TakagiKeitaro MatsuoSoyoung JeonCharleston W.K. ChiangAndrew T. DewanAdam J. de SmithToshihiko ImamuraYasuhiro OkamotoAkiko M. SaitoTakao DeguchiMichiaki KuboYoichi TanakaYoko AyukawaToshinari HoriKentaro OhkiNobutaka KiyokawaTakeshi InukaiYuki ArakawaMakiko MoriDaisuke HasegawaDaisuke TomizawaHiroko FukushimaYuki YuzaYasushi NoguchiYuichi TaneyamaSetsuo OtaHiroaki GotoMasakatsu YanagimachiDai KeinoKazutoshi KoikeDaisuke ToyamaYozo NakazawaKozue NakamuraKoichi MoriwakiYujin SekinakaDaisuke MoritaShinsuke HirabayashiYosuke HosoyaYuri YoshimotoHiroki YoshiharaMiwa OzawaShinobu KobayashiNaho MorisakiTshewang GyeltshenOsamu TakahashiYukinori OkadaMakiko MatsudaToshihiro TanakaJohji InazawaJunko TakitaYasushi IshidaAkira OharaCatherine MetayerJoseph L. WiemelsXiaomei MaShuki MizutaniKatsuyoshi KohYukihide MomozawaKeizo HoribeFumihiko MatsudaMotohiro KatoAtsushi ManabeKevin Y. Urayamakevurayama@gmail.com2023-10-26T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.283060BH3 profiling as pharmacodynamic biomarker for the activity of BH3 mimetics2024-03-31T22:04:09+00:00Rongqing Aaron PanYouzhen WangShumei QiuMariana Villalobos-OrtizJeremy RyanErick MorrisEnsar HalilovicAnthony LetaiAnthony_Letai@dfci.harvard.edu2023-10-26T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.283716Subcutaneous injection of IHP-102 prevents lung vaso-occlusion in sickle cell disease mice2024-03-31T22:04:10+00:00Rikesh K. DubeyRavi VatsTomasz BrzoskaTomasz W. KaminskiOmika KatochJesus TejeroGabriel NjikangJohn PaderiPrithu Sunddpsundd@versiti.org2023-10-19T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.283742Long-term outcome after autologous <i>BCR::ABL1</i>-negative peripheral blood stem cell transplantation in adults with Philadelphia-positive acute lymphoblastic leukemia: a comparative study2024-03-31T22:04:12+00:00Leo CaillotMathieu LeclercEmmanuel Jacques Raphael SleimanIvan SlomaOrianne Wagner-BallonAlexis ClaudelFlorence BeckerichRabah RedjoulChristine RobinVincent ParinetCecile PautasDehbia MenoucheSelwa BouledrouaLudovic CabanneYakout Nait-SidenasEric GautierHelene RouardIngrid LafonYves ChalandonNicolas BoisselDenis CaillotSebastien Maurysebastien.maury@aphp.fr2023-11-30T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.283802Glofitamab monotherapy induces high complete response rates and manageable safety in Chinese patients with heavily pretreated relapsed or refractory diffuse large B-cell lymphoma2024-03-31T22:04:15+00:00Yu-Qin SongHui-Lai ZhangHui-Qiang HuangQing-Yuan ZhangHong-Mei JingChao WangChun WuDong-Hang LiYu DaiKathryn HumphreyJun Zhuzhujun@csco.org.cn2023-10-19T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.283864Low-dose azacitidine, pioglitazone and all-<i>trans</i> retinoic acid is safe in patients aged ≥60 years with acute myeloid leukemia refractory to standard induction chemotherapy (AMLSG 26-16/AML-ViVA): results of the safety run-in phase2024-03-31T22:04:17+00:00Daniel Heudoblerdaniel.heudobler@ukr.deFlorian LukeJoachim HahnMatthias GrubePavla SchlosserStephan KremersThomas SudhoffJorg WestermannMarie Luise Hutter-KronkeRichard F. SchlenkDaniela WeberPeter PaschkaFlorian ZemanHartmut DohnerWolfgang HerrAlbrecht ReichleSimone Thomas2023-10-26T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.283884Comorbidity indices for prognostic evaluation in multiple myeloma: a comprehensive evaluation of the Revised Myeloma Comorbidity Index and other comorbidity indices with pro- and retrospective applications2024-03-31T22:04:20+00:00Katja SchoellerGabriele IhorstHeike ReinhardtMaximilian HollerSophia ScheubeckGeorg HergetRalph WäschMonika Engelhardtmonika.engelhardt@uniklinik-freiburg.de2023-11-30T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.283924Catheter-related thrombosis in stem cell recipients: comparison of different types of catheter2024-03-31T22:04:22+00:00Slawomir Milczarekslawomir.milczarek@pum.edu.plPiotr KuligOliwia PiotrowskaAlina ZuchmanskaAnna BielikowiczBogusław Machalinski2023-11-02T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.284050Small myeloid subclones are present at diagnosis of multiple myeloma in patients who develop secondary myelodysplastic syndromes2024-03-31T22:04:24+00:00Guillaume EscureElise FournierCynthia SaadeLama Hasan Bou IssaInès AribRémi TilmontNicolas GazeauBinta M. ThiamMorgane ChovetMaxime DelforgeNicolas GowerLéa FléchonDoriane CavalieriPaul ChauvetMorgane NudelLaure GoursaudCéline BerthonBruno QuesnelThierry FaconClaude PreudhommeNicolas DuployezSalomon Maniersalomon.manier@chu-lille.fr2023-10-19T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.284095Factor VIII genotype and the risk of developing highresponding or low-responding inhibitors in severe hemophilia A: data from the PedNet Hemophilia Cohort of 1,202 children2024-03-31T22:04:27+00:00Nadine G. Anderssonnadine.gretenkort_andersson@med.lu.seVeerle LabarqueMutlu Kartal-KaessFernando PintoTorben Stamm MikkelsenRolf LjungPedNet Study Group2023-10-26T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.284169CD38 expression by plasma cells in extramedullary multiple myeloma2024-03-31T22:04:28+00:00Laura NotarfranchiFabrizio AccardiCristina ManciniEugenia MartellaSabrina BonominiRoberta SegretoRosanna VescoviniAnna Benedetta Dalla PalmaGabriella SammarelliGiannalisa TodaroPaola StortiJessica Burroughs-GarciaNicolas Thomas IannozziVincenzo RaimondiOxana LunguStefania RicciLuisa CraviottoNicola Giulianinicola.giuliani@unipr.it2023-11-09T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.283563A patient with minimal myeloma treatment who survived for 20 years2024-03-31T22:04:31+00:00Alenka Djarmila Behsenalenka.d.behsen@ntnu.noEsten Nymoen VandsembTobias Schmidt SlørdahlHenrik Hjorth-HansenPetter Quist-PaulsenKristine MisundAnne-Marit SponaasAnders Waage2023-10-05T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/haematol.2023.284547Emergence of <i>TP53</i> mutation during lenalidomide therapy of myelodysplastic syndrome with del(5q) and its subsequent disappearance following salvage therapy with decitabine2024-03-31T22:04:33+00:00Naseema Gangatgangat.naseema@mayo.eduNaresh BellamKaaren ReichardAyalew Tefferitefferi.ayalew@mayo.edu2023-11-30T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/11515<i>Erratum</i> to: Retrospective analysis of pacritinib in patients with myelofibrosis and severe thrombocytopenia2024-04-01T19:37:46+00:00Srdan Verstovseksrdanverstovsek@gmail.comRuben MesaMoshe TalpazJean-Jacques KiladjianClaire N. HarrisonStephen T. OhAlessandro M. VannucchiRaajit RampalBart L. ScottSarah A. BuckleyAdam R. CraigKarisse Roman-TorresJohn O. Mascarenhas2024-04-01T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundationhttps://haematologica.org/article/view/11516<i>Erratum</i> to: Utility of clinical comprehensive genomic characterization for diagnostic categorization in patients presenting with hypocellular bone marrow failure syndromes2024-04-01T19:37:47+00:00Piers Blomberypiers.blombery@petermac.orgLucy FoxGeorgina L. RylandElla R. ThompsonJennifer LickissMichelle McBeanSatwica YerneniAlison TrainerDavid HughesAnthea GreenwayFrancoise MechinaudErica M. WoodGraham J. LieschkeJeff SzerPasquale BarbaroJohn RoyJoel WightElly LynchMelissa MartynClara GaffDavid Ritchie2024-04-01T00:00:00+00:00Copyright (c) 2024 Ferrata Storti Foundation