Tailored prophylaxis in children with severe hemophilia: A four-year Iranian study
- 5 July 2021
- journal article
- research article
- Published by Elsevier BV in Transfusion and Apheresis Science
- Vol. 60 (6), 103212
- https://doi.org/10.1016/j.transci.2021.103212
Abstract
Background Prophylaxis the current standard care for patients with severe hemophilia should be planned to optimize the replacement therapy and minimize bleeding. We report our single-center experience of tailored prophylaxis in children affected by hemophilia A (HA) and hemophilia B (HB). Methods This study was conducted on 55 patients, under 15 years, with HA (PWHA, n: 46) and HB (PWHB, n: 9) between 2015 and 2019. According to the phenotype, three prophylaxis regimens: 25−50 unit/kg once, twice, or three-times a week for PWHA, and two: 30−50 unit/kg once or twice a week for PWHB were administered. Following the occurrence of > 3 joint bleeding, or > 4 soft tissue bleeding, or one spontaneous major bleeding in the last 3 months, the prophylaxis regimen is changed. Annualized bleeding rate (ABR), annualized joint bleeding rate (AJBR), target joints development, inhibitor development, and hemophilia joint health score (HJHS) also were assessed. Results A mean ± SD of 2520 ± 1045 IU/kg/yr coagulation factor (F) VIII was used to reduce ABR and AJBR from 1.02 ± 1.11 and 0.8 ± 1.3 (in the first year of the study) to 0.27 ± 0.44 (P < 0.001) and 0.19 ± 0.38 (P = 0.004) (at the end of the study) in PWHA, respectively. Furthermore, in PWHB, in the first year of the study, with using 2168 ± 1216 IU/kg coagulation FIX, ABR and AJBR were 0.19 ± 0.39 and 0.06 ± 0.1. At the end of the study, ABR and AJBR were 0.02 ± 0.05 (p = 0.156) and 0.01 ± 0.03 (p = 0.361), respectively. During the study period, the mean number of the target joints and mean HJHS were 0.25 ± 0.57 and 7.6 ± 2.1 for PWHA and 0 and 6.3 ± 1.8 for PWHB, respectively. Finally, 5 PWHA (11 %) did not need dose-escalation in their prophylaxis regimen, whereas 31 (67 %) and 10 (21 %) PWHA needed two and three infusions a week, respectively. In PWHB, 7 (78 %) and 2 (22 %) were adjusted to receive a once and twice weekly regimen, respectively. Conclusion Our results suggest that tailored prophylaxis is an effective strategy to reduce the rate of bleeding and optimize the replacement therapy in children with hemophilia.Keywords
This publication has 13 references indexed in Scilit:
- Hemophilia in IranHematology, 2016
- Intermediate-dose versus high-dose prophylaxis for severe hemophilia: comparing outcome and costs since the 1970sBlood, 2013
- New early prophylaxis regimen that avoids immunological danger signals can reduce FVIII inhibitor developmentHaemophilia, 2010
- Devising a best practice approach to prophylaxis in boys with severe haemophilia: evaluation of current treatment strategiesHaemophilia, 2010
- Treatment-related risk factors of inhibitor development in previously untreated patients with hemophilia A: the CANAL cohort studyBlood, 2007
- Tailored prophylaxis in severe hemophilia A: interim results from the first 5 years of the Canadian Hemophilia Primary Prophylaxis StudyJournal of Thrombosis and Haemostasis, 2006
- Environmental risk factors for inhibitor development in children with haemophilia A: a case–control studyBritish Journal of Haematology, 2005
- A prospective, longitudinal study of central venous catheter‐related deep venous thrombosis in boys with hemophiliaJournal of Thrombosis and Haemostasis, 2004
- A survey of factor prophylaxis in boys with haemophilia followed in North American haemophilia treatment centresHaemophilia, 2003
- Experience of prophylaxis treatment in children with severe haemophiliaHaemophilia, 2002