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Health-related quality of life in paediatric haemophilia B patients treated with rIX-FP.

ABSTRACT: INTRODUCTION:Frequent infusions and bleeds can impact on the health-related quality of life (HRQoL) of paediatric haemophilia B patients. rIX-FP (IDELVION® ) is a fusion protein linking recombinant factor IX with recombinant albumin, and is associated with low bleeding rates with a weekly regimen, which could improve HRQoL. AIMS:To measure the effect of rIX-FP prophylaxis on the HRQoL of paediatric patients and treatment satisfaction in their caregivers using the Haemo-QoL and Hemo-SATP questionnaires, respectively. METHODS:At baseline and end-of-study (EOS), patients 4-11 years old participating in the PROLONG-9FP program answered the Haemo-QoL questionnaire and gave information on their socio-demographic data and physical activity. Caregivers completed the Hemo-SatP . Minimal important differences (MID) (|Cohen's d| > 0.5) between baseline and EOS and the number of responders (patients with meaningful subject-level improvements over time) at EOS were calculated. RESULTS:Twenty patients (age group I: 4-7 years old [n = 12]; age group II: 8-12 years old [n = 8]) completed the Haemo-QoL questionnaire at baseline. MIDs were found in age group I representing improvement for "physical health" (d = -0.547) domain; 60% of patients were responders for "physical health." In age group II, MIDs were seen in most domains; 71.4% patients were responders in "total score." In caregivers, improvements were seen for most domains of the Hemo-SatP with a small effect size. Fewer patients missed school when treated with rIX-FP and 94.1% patients maintained their physical activity level. CONCLUSION:Prophylaxis with rIX-FP led to substantial improvements in HRQoL in paediatric patients and treatment satisfaction in caregivers.

SUBMITTER: von Mackensen S

PROVIDER: S-EPMC6916427 | biostudies-literature | 2019 Jan

REPOSITORIES: biostudies-literature

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Health-related quality of life in paediatric haemophilia B patients treated with rIX-FP.

von Mackensen Sylvia S Shah Jinesh J Seifert Wilfried W Kenet Gili G

Haemophilia : the official journal of the World Federation of Hemophilia 20181114 1

<h4>Introduction</h4>Frequent infusions and bleeds can impact on the health-related quality of life (HRQoL) of paediatric haemophilia B patients. rIX-FP (IDELVION<sup>®</sup> ) is a fusion protein linking recombinant factor IX with recombinant albumin, and is associated with low bleeding rates with a weekly regimen, which could improve HRQoL.<h4>Aims</h4>To measure the effect of rIX-FP prophylaxis on the HRQoL of paediatric patients and treatment satisfaction in their caregivers using the Haemo- ...[more]

PMID: 30427091

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Health-related quality of life in paediatric haemophilia B patients treated with rIX-FP.

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Health-related quality of life in paediatric haemophilia B patients treated with rIX-FP.

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