First‐year results of an expanded humanitarian aid programme for haemophilia in resource‐constrained countries

Pierce, Glenn F.; Haffar, Assad; Ampartzidis, Georgios; Peyvandi, Flora; Diop, S; El‐Ekiaby, Magdy; Berg, H. M. Van Den

doi:10.1111/hae.13409

Cited by 33 publications

(49 citation statements)

References 21 publications

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“…Factor replacement therapy is expensive,22 and most WFH National Member Countries (NMO) in resource-poor countries' category depend solely on donated factor concentrates from WFH's Humanitarian Aid Programme 15. These donations and establishment of NMO-supported HTCs in resource-poor nations have improved access of PWH to factor concentrates and thus improved management of haemophilia 23. This is in concordance with findings from the index study as most participants received care from HTCs and had consulted a doctor within 6 months of the study.…”

Section: Discussionsupporting

confidence: 80%

Health status of persons with hemophilia: A pilot survey from a resource-constrained Country

Okoye

Nwogoh

Adediran³

et al. 2019

Niger Med J

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Background: Most resource-poor countries are yet to develop standard hemophilia treatment center (HTC) despite improved outcome of health status of persons with hemophilia (PWH). Aim: This study aimed to evaluate the health status of PWH in Nigeria. Methodology: In this descriptive, cross-sectional study, modified prevalidated and pretested questionnaire (National health and Nutrition examination survey (NHANES) 2013 Health Status questionnaire (HSQ)) was consecutively administered to consenting PWH (pediatric and adult) attending the 2018 Annual General Meeting of the Haemophilia Foundation of Nigeria. The study's measurable outcome variables were calculated health status and its determinants. Association between the outcome variables and clinical characteristics of PWH was done using SPSS software version 22, and P < 0.05 was considered statistically significant. Results: Of the 36 PWH who participated in the survey, 50% had good health status, 38.9% had poor health status, while only 11.1% had excellent health status. A majority (88.9%) had access to HTC with <6 consultations in the past year. Nearly 47.2% were hospitalized for disease-related problem in the past year. There was nonsignificant difference between health status and disease type ( P = 0.751) and severity ( P = 0.086), treatment plan (0.496), type of treatment facility ( P = 0.152), and access to a doctor ( P = 0.67). Conclusion: Several PWH in resource-poor settings still suffer serious morbidity that impacts negatively on their health status. More robust (multicenter) research is needed to ascertain the true picture of health status of PWH in resource-poor countries.

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Section: Discussionsupporting

confidence: 80%

Health status of persons with hemophilia: A pilot survey from a resource-constrained Country

Okoye

Nwogoh

Adediran³

et al. 2019

Niger Med J

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“…Prevention of joint bleeds and subsequent arthropathy relies on regular prophylaxis with clotting factor concentrates (CFC) of FVIII or FIX, ideally initiated at a young age . In resource‐constrained countries, where 70% of PWH live, very few people have access to CFC, resulting in a high morbidity and mortality due to untreated bleedings and increased rates of musculoskeletal (MSK) complications . Physiotherapy actively contributes to preventing MSK complications because it helps maintain good mobility and sufficient strength.…”

Section: Introductionmentioning

confidence: 99%

Implementation and assessment of a self‐ and community‐based rehabilitation programme in patients with haemophilia from Côte d’Ivoire

Lobet

Meité²,

Koninckx

et al. 2019

Haemophilia

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Introduction:In resource-constrained countries, few patients with haemophilia (PWH) have access to clotting factor concentrates (CFC), with increased musculoskeletal (MSK) complications. Physiotherapy actively contributes to preventing MSK complications, minimizing joint damage and reducing pain. Aim: To assess the impact of a 20-week self-and community-based rehabilitation (CBR) programme in Ivorian PWH. Methods: Fifty participants underwent a clinical and functional baseline assessment with identification of joints' functional defects and initiation of an individualized exercise programme comprising exercises to improve strength, joint mobility and proprioception. Hemophilia Joint Health Score (HJHS), 2-minute walking test (2MWT), Timed Up and Go (TUG), goniometry and maximal isometric voluntary contractions using the MicroFET2 were performed at baseline (T1) and at Week 20 (T2).Results: At T2, there was a significant improvement in both the 2MWT and TUG tests.The HJHS total score decreased significantly from 23.6 ± 14.2 at T1 to 20.4 ± 13 at T2. A significant improvement in joint health was found in the left elbow, right knee and right ankle, with elements correlating with joint function responsible for these improvements. A strong programme adherence was observed, with 94% of participants reporting regular exercise performance and a high degree of satisfaction. Conclusion:The programme with its encouraging results is meant to be the first step towards a more ambitious project. Self-based and CBR programmes are inexpensive and efficient treatment options designed to minimize the detrimental effects of joint and muscle bleedings, and to increase the functional independence and quality of life of PWH with limited access to CFC and physiotherapy. K E Y W O R D SCôte d'Ivoire, developing country, exercise, haemophilia, physiotherapy, self-and communitybased rehabilitation programme

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“…In the last couple of years, several extended half‐life blood coagulation factor products gained regulatory approval for the treatment of haemophilia B . However, access to this most advanced treatment option remains limited to developed countries, and the need for standard half‐life recombinant or plasma‐derived FIX products is still high.…”

Section: Introductionmentioning

confidence: 99%

Postmarketing safety and effectiveness of recombinant factor IX (nonacog alfa) in Japanese patients with haemophilia B

et al. 2019

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IntroductionIn 2010, nonacog alfa became the first recombinant factor IX (rFIX) available in Japan for patients with haemophilia B.AimTo determine real‐world safety (adverse events, incidence of inhibitors) and effectiveness of nonacog alfa in Japan.MethodsThis multicentre, prospective, observational, postmarketing surveillance study enrolled previously treated and untreated patients (PTPs and PUPs, respectively) who were observed for 1 and 2 years, respectively, after initiating nonacog alfa therapy. Safety and effectiveness were assessed for each treatment type. Annualized bleeding rate (ABR) and incremental recovery of rFIX were also evaluated.ResultsOverall, 312 of 314 patients enrolled from 173 sites were eligible for the safety analysis set (PTPs, 281; PUPs, 28; other, 3). Mean age was 25.4 (PTPs) and 14.8 (PUPs) years. Haemophilic severity ranged from mild to severe, and 133 (42.6%) patients had haemophilic arthropathy. Of 285 patients (PTPs, 257; PUPs, 28) in the effectiveness set, 112 received on‐demand treatment for 1161 bleeding episodes (effectiveness rate, 93.7%) and 185 received routine prophylaxis (effectiveness rate, 95.5%). No spontaneous bleeding was observed in 52.4% of patients during prophylactic treatment. Median ABR was lower during routine prophylaxis (2.0) vs the rest of the observation period (8.3). A weak negative correlation was found between body weight and the reciprocal of rFIX recovery. Eleven adverse drug reactions occurred in 7 PTPs (2.2% [7/312]); recurrence of inhibitor was observed in 1 patient, but no new inhibitor developed in PTPs or PUPs.ConclusionNonacog alfa therapy is safe and effective in the real‐world scenario in Japan.

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First‐year results of an expanded humanitarian aid programme for haemophilia in resource‐constrained countries

Cited by 33 publications

References 21 publications

Health status of persons with hemophilia: A pilot survey from a resource-constrained Country

Health status of persons with hemophilia: A pilot survey from a resource-constrained Country

Implementation and assessment of a self‐ and community‐based rehabilitation programme in patients with haemophilia from Côte d’Ivoire

Postmarketing safety and effectiveness of recombinant factor IX (nonacog alfa) in Japanese patients with haemophilia B

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