@article{Mariasanta Napolitano_Muriel Giansily-Blaizot_Alberto Dolce_Jean F. Schved_Guenter Auerswald_Jørgen Ingerslev_Jens Bjerre_Carmen Altisent_Pimlak Charoenkwan_Lisa Michaels_Ampaiwan Chuansumrit_Giovanni Di Minno_Ümran Caliskan_Guglielmo Mariani_2013, place={Pavia, Italy}, title={Prophylaxis in congenital factor VII deficiency: indications, efficacy and safety. Results from the Seven Treatment Evaluation Registry (STER)}, volume={98}, url={https://haematologica.org/article/view/6620}, DOI={10.3324/haematol.2012.074039}, abstractNote={Because of the very short half-life of factor VII, prophylaxis in factor VII deficiency is considered a difficult endeavor. The clinical efficacy and safety of prophylactic regimens, and indications for their use, were evaluated in factor VII-deficient patients in the Seven Treatment Evaluation Registry. Prophylaxis data (38 courses) were analyzed from 34 patients with severe factor VII deficiency (<1-45 years of age, 21 female). Severest phenotypes (central nervous system, gastrointestinal, joint bleeding episodes) were highly prevalent. Twenty-one patients received recombinant activated factor VII (24 courses), four received plasma-derived factor VII, and ten received freshfrozen plasma. Prophylactic schedules clustered into “frequent” courses (three times weekly, n=23) and “infrequent” courses (≤2 times weekly, n=15). Excluding courses for menorrhagia, “frequent” and “infrequent” courses produced 18/23 (78%) and 5/12 (41%) “excellent” outcomes, respectively; relative risk, 1.88; 95% confidence interval, 0.93-3.79; <em>P</em>=0.079. Long term prophylaxis lasted from 1 to >10 years. No thrombosis or new inhibitors occurred. In conclusion, a subset of patients with factor VII deficiency needed prophylaxis because of severe bleeding. Recombinant activated factor VII schedules based on “frequent” administrations (three times weekly) and a 90 μg/kg total weekly dose were effective. These data provide a rationale for long-term, safe prophylaxis in factor VII deficiency (<em><a href="http://clinicaltrials.gov">clinicaltrials.gov</a>: <a class="external-ref external-ref-type-clintrialgov" href="/lookup/external-ref?link_type=CLINTRIALGOV&amp;access_num=NCT01269138&amp;atom=%2Fhaematol%2F98%2F4%2F538.atom">NCT01269138</a></em&gt;).}, number={4}, journal={Haematologica}, author={Mariasanta Napolitano and Muriel Giansily-Blaizot and Alberto Dolce and Jean F. Schved and Guenter Auerswald and Jørgen Ingerslev and Jens Bjerre and Carmen Altisent and Pimlak Charoenkwan and Lisa Michaels and Ampaiwan Chuansumrit and Giovanni Di Minno and Ümran Caliskan and Guglielmo Mariani}, year={2013}, month={Mar.}, pages={538-544} }