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<p>In absence of any epidemiological studies, it is expected that there are at least 1 lakh PWH in India (Hemophilia Complications&#xa0;2009, World Federation Hemophilia 2005, Hemophilia Federation (India), Kar and Potnis-Lele <i>Haemophilia</i> 7: 561-7. 82, 2001, Kar and Potnis-Lele <i>Haemophilia</i> 10: 301-4, 2004). Due to lack of awareness, diagnostic as well as treatment facilities, many of the patients are on cryoprecipitate/ Factor concentrate, as episodic treatment only. All severe hemophiliacs between 1 and 6 years were screened for inclusion and exclusion criteria and then enrolled in study after informed consent from parents. All patients were assessed for bleeding history, HJHS, FISH and CHAQ at baseline and every 3 monthly. <b>11.1%</b> of children were on secondary prophylaxis and <b>88.9%</b> were on primary prophylaxis. Three patients developed spontaneous breakthrough bleed during the study. There was <b>77.97%</b> reduction in AJBR, <b>70.8%</b> in ABR and <b>80%</b> in school absenteeism. CHAQ score showed − <b>83%</b> improvement. 9 (<b>50%)</b> patients were on home-therapy. The study had <b>100%</b> compliance. The average dose of factor required <b>13.29</b> unit/kg/patient/dose and <b>1382.96</b> unit/kg/patient/year. Prophylaxis with low dose Factor concentrate with escalation protocol can decrease the frequency of joint and other hemorrhages, reduce school absenteeism and prevent joint damage.</p>

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Low Dose Escalating Factor Concentrate Prophylaxis in Patients with Severe Hemophilia in a Comprehensive Hemophilia Care Centre of Western India

  • 期刊论文

  • 发表日期：

    2025年9月20日

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  • Aniket B Mohite,
  • S. Chandrakala,
  • Farah Jijina,
  • Nilesh Wasekar,
  • Mayura Bane,
  • Shrimati Shetty,
  • Rucha Patil

摘要

In absence of any epidemiological studies, it is expected that there are at least 1 lakh PWH in India (Hemophilia Complications 2009, World Federation Hemophilia 2005, Hemophilia Federation (India), Kar and Potnis-Lele Haemophilia 7: 561-7. 82, 2001, Kar and Potnis-Lele Haemophilia 10: 301-4, 2004). Due to lack of awareness, diagnostic as well as treatment facilities, many of the patients are on cryoprecipitate/ Factor concentrate, as episodic treatment only. All severe hemophiliacs between 1 and 6 years were screened for inclusion and exclusion criteria and then enrolled in study after informed consent from parents. All patients were assessed for bleeding history, HJHS, FISH and CHAQ at baseline and every 3 monthly. 11.1% of children were on secondary prophylaxis and 88.9% were on primary prophylaxis. Three patients developed spontaneous breakthrough bleed during the study. There was 77.97% reduction in AJBR, 70.8% in ABR and 80% in school absenteeism. CHAQ score showed − 83% improvement. 9 (50%) patients were on home-therapy. The study had 100% compliance. The average dose of factor required 13.29 unit/kg/patient/dose and 1382.96 unit/kg/patient/year. Prophylaxis with low dose Factor concentrate with escalation protocol can decrease the frequency of joint and other hemorrhages, reduce school absenteeism and prevent joint damage.