Abstract
OUTCOME OF DOMICILIARY TREATMENT OF LOW DOSE ESCALATING FACTOR CONCENTRATE PROPHYLAXIS IN SEVERE PWH FROM LOW SOCIOECONOMIC STRATA

Dr. Aniket B. Mohite*, Dr. Chandrakala S., Dr. Farah Jijina, Dr. Mayura Bane, Akshata Rahate, Dr. Shrimati Shetty#, Dr. Rucha Patil

ABSTRACT

Hemophilia A has prevalence of 1 patient every 10,000 male births and Hemophilia B 1 patient every 30,000 male births.1-5 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 1-10 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. Out of 35 patients, 7 patients were PUPs. Sixteen patients developed spontaneous breakthrough bleed during the study. There was 72.9% reduction in AJBR, 60.83% in ABR and 95.21% in school absenteeism. CHAQ score showed - 68.32% improvement and the joint scores improved significantly. 18 (51.42%) patients were on home-therapy. Most of these families (82.8%) were from lower strata of society belonging to Kupuswamy classification III and IV. The study had 100% compliance. The average dose of plasma derived factor required 11.663 unit/kg/patient/dose and 1209.89 unit/kg/patient/year. Conclusions: 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 in children with severe hemophilia. Home therapy is possible in the developing countries with dedicated training even in low socioeconomic strata of society.

Keywords: PWH, Factor, Episodic, breakthrough bleed, FISH, HJHS, CHAQ.


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