A randomized study of very low‐dose factor VIII prophylaxis in severe haemophilia – A success story from a resource limited country

Verma, Shailendra Prasad; Tk, Dutta; Mahadevan, Subramanian; Nalini, P; Basu, Debdutta; Biswal, Niranjan; Ananthakrishnan, Ramesh; Charles, Deepak; Vinod, Kolar Vishwanath; Kumar, Kiran

doi:10.1111/hae.12838

Cited by 68 publications

(87 citation statements)

References 18 publications

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“…10,17,18 All of 12 haemophilia centres in this study are located in cities with a well-developed economy and are considered the most advanced diagnosis and treatment centres in China. Prophylaxis was not introduced to China until 2008.…”

Section: Discussionmentioning

confidence: 99%

Influence of medical insurance schemes and charity assistance projects on regular prophylaxis treatment of the boys with severe haemophilia A in China

Zhao

et al. 2017

Haemophilia

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Section: Discussionmentioning

confidence: 99%

Influence of medical insurance schemes and charity assistance projects on regular prophylaxis treatment of the boys with severe haemophilia A in China

Zhao

et al. 2017

Haemophilia

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“…The result supported other clinical trials proving that prophylaxis treatment is superior compare to episodic treatment in preventing joint damage progressivity in children with severe hemophilia. [18][19][20][21] In Indonesia, severe hemophilia chidren receive episodic treatment due to very expensive cost of factor concentrates.…”

Section: Discussionmentioning

confidence: 99%

Clinical and ultrasound joint outcomes in severe hemophilia A children receiving episodic treatment in Indonesian National Hemophilia Treatment Center

et al. 2017

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Background: Recurrent joint bleeds leading to arthropathy is the main problem in severe hemophilia children. This study aimed to investigate joint status in severe hemophilia A children receiving episodic treatment in Cipto Mangunkusumo Hospital, Jakarta.Methods: A cross-sectional study was conducted in Cipto Mangunkusumo Hospital as Indonesian National Hemophilia Treatment Center on children (4–18 years) with severe hemophilia A, who previously received episodic treatment, with no history of inhibitor factor VIII. Hemophilia Joint Health Score was evaluated according to HJHS version 2.1 2011. Joint ultrasonography was done for six index joints (bilateral elbows, knees and ankles) using Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) methods. Data of age of first joint bleed, number of target joints and inhibitor factor VIII were obtained from the Pediatric Hemophilia Registry and medical records.Results: There were 59 subjects aged 4 to 18 years. Twenty-nine out of 59 (49.2%) subjects experienced first joint bleed before of 2 years of age. The most common of joint bleeds was a right ankle. Mean total HJHS was 8.71±8.73. Subjects aged 4–10 years showed lower HJHS (4.6±3.7) as compared to subjects aged >10–18 years (12.3±10.3), p<0.001; 95% CI=4.9–13. Mean HEAD-US scores in subjects aged 4–10 years (18.7±5.6) was lower than in subjects aged >10–18 years (28±7.9), p<0.001, 95% CI= -12.9–-5.6.Conclusion: HJHS and HEAD-US scores of severe hemophilia A children receiving episodic treatment aged 4–10 years are lower compared to subjects aged >10–18 years, indicating more severe joint destruction in older children and progressivity of joint damage over time. It is important to start prophylactic treatment to prevent progressivity of joint damage.

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“…A 2012 pilot study demonstrated that daily prophylaxis is feasible, with a substantial reduction (30 %) in factor concentrate utilization - albeit with a higher bleed rate in certain patients, and a decreased quality of life [5]. Verma et al recently demonstrated that a very low dose, twice-weekly prophylaxis regimen in children was effective at lowering bleed rates compared to on-demand treatment [6]. …”

Section: Introductionmentioning

confidence: 99%

Adherence to treatment regimen and bleeding rates in a prospective cohort of youth and young adults on low-dose daily prophylaxis for severe hemophilia A

et al. 2016

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BackgroundWhen availability and/or affordability of anti-hemophilic factor concentrates are limited, optimal prophylaxis regimens in severe hemophilia A (HA) remain to be determined. In selected situations, low-dose daily prophylaxis (LDDP) may be an effective and economical option. The goal of our study was to evaluate if subjects on a LDDP regimen could achieve adherence and good clinical outcome.MethodsSeventeen subjects (age between 15.2 and 28.4) on LDDP suffering from severe/moderate HA were followed prospectively for 2 to 3 years as part of a health-related quality of life (HRQoL) study. Bleeding and treatments data were collected using electronic diaries and validated every three months. The SF-36 questionnaire was administered at the beginning of the study and then every 6 months until the end of the study.ResultsThe subjects (mean age 22.0, median 21.9, standard deviation 4.06), were all from a single centre and on LDDP for at least 12 months as part of their routine care before entering the study. Fifteen subjects were prescribed a daily dose of 500 IU factor VIII (FVIII) and 2 subjects received 1000 IU FVIII per day, resulting into a median dose of 7.1 IU/kg/day (ranging from 4 to 13 IU/kg/day) and of 2591 IU/kg/year. Median adherence (the percentage of the prescribed daily dose received) was 84 % (mean 80 %, range 57 % to 94 %) throughout the study. Seventy-six bleeds in the 6 index joints and 51 other types of bleeds were observed throughout the study. The median annualized bleeding rate in joints (ABRjoints) was 0.7 and the median annualized bleeding rate for all bleeds (ABRall) was 1.6. The Physical Component and Mental Component Summary scores of SF-36, and the Hemophilia Joint Health Score were not significantly different over the course of the study (respective medians of 49.8, 52.4 and 16.0 at entry; vs. 52.5, 51.5 and 16.0 upon exit).ConclusionsThis prospective longitudinal study in youth and young adults shows that LDDP may be associated with low ABRs, adequate adherence and HRQoL comparable to previously reported.

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A randomized study of very low‐dose factor VIII prophylaxis in severe haemophilia – A success story from a resource limited country

Abstract: To conclude, low-dose FVIII prophylaxis is cost effective, efficacious and a safe method of preventing joint bleeds and consequent joint damages.

Cited by 68 publications

References 18 publications

Influence of medical insurance schemes and charity assistance projects on regular prophylaxis treatment of the boys with severe haemophilia A in China

Influence of medical insurance schemes and charity assistance projects on regular prophylaxis treatment of the boys with severe haemophilia A in China

Clinical and ultrasound joint outcomes in severe hemophilia A children receiving episodic treatment in Indonesian National Hemophilia Treatment Center

Adherence to treatment regimen and bleeding rates in a prospective cohort of youth and young adults on low-dose daily prophylaxis for severe hemophilia A

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