Abstract Number: PB0865
Meeting: ISTH 2020 Congress
Background: Hemophilia A and B are rare inherited bleeding disorders caused by deficiency of coagulation factor VIII (FVIII) and factor IX (FIX), respectively. These disorders are followed by recurrent episodes of bleeding, primarily into the joints and muscles.
Aims: Our study aims to find the efficacy of prophylactic treatment regimen for patients with severe hemophilia
Methods: Children between 4‐8 years with severe hemophilia A (FVIII < 1%) without inhibitors and a HJHS score of < 5 were offered prophylaxis with a plasma-derived clotting factor concentrates pdCFCs.
at a lower dose of 10‐15 IU/kg 2‐3 x/week, depending on weight and response. Their bleeding rate over 12 months prior to initiating prophylaxis was documented. All bleeding was documented while on prophylaxis, with special emphasis on joint or muscle bleeds. Inhibitors were monitored every 5‐10 exposures.
Results: A total of 28 children were treated with this prophylactic regimen. The median age was 6 years old.
The median annualized bleeding rate (AdBR) prior to prophylaxis was 4 (0‐15). No patient had any muscle or CNS bleeding during this period. Two patients developed transient inhibitors (low titer 1.1 BU, 2.5 BU & 2.0 BU respectively) after 7, 17 and 100 exposures. These resolved within 4‐12 weeks with continuing prophylaxis and have remained negative since. None of the other patients developed inhibitors after a median of 85 exposures (9 to 124). Compliance was complete except for one patient who missed the prophylactic treatment twice.
Conclusions: It is evident that decrease in bleeding rate, preventing destruction of joint and its function and improvement in quality of life can be achieved through low dose prophylaxis regimen. Therefore low dose prophylaxis may be considered as an initial step for pro-phylaxis regimen, particularly in resource limited settings.
To cite this abstract in AMA style:Fatima A. Finding the Efficacy of Low Dose Prophylactic Regimen in Children Diagnosed with Severe Hemophilia [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/finding-the-efficacy-of-low-dose-prophylactic-regimen-in-children-diagnosed-with-severe-hemophilia/. Accessed December 2, 2022.
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