Real-world treatment, clinical outcomes and healthcare resource utilization among persons with hemophilia A by age

Caplan, Eleanor O.; Patel, Anisha M.; DeClue, Richard W; Sehman, Marina; Cornett, Daniel; Shaffer, Tiffany Fair; Raimundo, Karina; Suehs, Brandon T.

doi:10.2217/cer-2021-0109

Cited by 7 publications

(4 citation statements)

References 28 publications

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“…Consistent with former studies, this study suggestedthat prophylaxis therapy decreases the frequency of haemorrhages, which is bene cial to prevent the progression of haemophilic complications [3,9,10,14,17]. Furthermore, we add the new evidence that it also signi cantly relieves chronic pain related to haemophilia [37]. There was no statistical difference in the number of target joints and the probability of disability for children with different treatment regimens.…”

Section: Discussionsupporting

confidence: 88%

Treatment regimens, patient reported outcomes and health-related quality of life in children with moderate and severe haemophilia A in China: using real-world data

zhang

Zhang

Chen

2023

Preprint

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Background Prophylaxis therapy for children with moderate and severe haemophilia A (HA) is the optimal treatment regimen. The real-world treatment regimens, patient reported outcomes and health-related quality of life (HRQoL) in children with moderate and severe HA in China are less known.Objective This study aimed to describe the real-world treatment regimens and evaluate the association oftreatment regimens with comprehensive patient reported outcomes including bleeds, chronic pain, target joints, disability and HRQoL in children under 18 years old with HA in China.Methods Real-world data of a nationwide online cross-sectional survey in 2021 and patients’ coagulation factor utilization data from self-management records from 2020 to 2021 were merged. 373 eligible children were included and categorized by treatment regimens according to the Chinese guideline: on-demand, short-term prophylaxis and long-term prophylaxis treatment.Results Currently, in China, 4.8% of children with HA are receiving full-dose long-term prophylaxis treatment. Prophylaxis treatment was a significant positive predictor of better patient reported outcomes and HRQoL. For children with prophylaxis treatment, there were significantly fewer annual bleeds (p < 0.001), lower frequency of chronic pain(p < 0.001), and higher health utility scores(p < 0.01) and EQ-VAS scores(p < 0.05) than children with on-demand treatment.Conclusion Accessible long-term prophylaxis treatment should be promoted for children with moderate and severe HA in China and regular monitoring of their outcomes and HRQoL should be carried out.

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Section: Discussionsupporting

confidence: 88%

Treatment regimens, patient reported outcomes and health-related quality of life in children with moderate and severe haemophilia A in China: using real-world data

zhang

Zhang

Chen

2023

Preprint

View full text Add to dashboard Cite

show abstract

“…Consistent with former studies, this study suggested that prophylaxis therapy decreases the frequency of hemorrhages, which is beneficial to prevent the progression of hemophilic complications [ 3 , 9 , 10 , 14 , 17 ]. Furthermore, we add new evidence that it also significantly relieves chronic pain related to hemophilia [ 37 ]. There was no statistical difference in the number of target joints and the probability of disability for children with different treatment regimens.…”

Section: Discussionmentioning

confidence: 99%

Treatment regimens, patient reported outcomes and health-related quality of life in children with moderate and severe hemophilia A in China: using real-world data

Zhang

Chen

2023

Orphanet J Rare Dis

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Background Prophylaxis therapy for children with moderate and severe hemophilia A (HA) is the optimal treatment regimen. The real-world treatment regimens, patient-reported outcomes, and health-related quality of life (HRQoL) in children with moderate and severe HA in China are less known. Objective This study aimed to describe real-world treatment regimens and evaluate the association of treatment regimens with comprehensive patient-reported outcomes including bleeds, chronic pain, target joints, disability, and HRQoL in children under 18 years old with HA in China. Methods Real-world data of a nationwide online cross-sectional survey in 2021 and patients’ coagulation factor utilization data from self-management records from 2020 to 2021 were merged. 373 eligible children were included and categorized by treatment regimens according to the Chinese guideline: on-demand, short-term prophylaxis, and long-term prophylaxis treatment. Results Currently, in China, 4.8% of children with HA are receiving full-dose long-term prophylaxis treatment. Prophylaxis treatment was a significant positive predictor of better patient-reported outcomes and HRQoL. For children with prophylaxis treatment, there were significantly fewer annual bleeds (p < 0.001), lower frequency of chronic pain(p < 0.001), and higher health utility scores(p < 0.01) and EQ-VAS scores(p < 0.05) than children with on-demand treatment. Conclusion Accessible long-term prophylaxis treatment should be promoted for children with moderate and severe HA in China and regular monitoring of their outcomes and HRQoL should be carried out.

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“…[13][14][15][16][17][18] Currently, real-world studies of prophylactic FVIII use in hemophilia A are limited. 7,[19][20][21] HEM-POWR is an ongoing, Phase 4, observational, 36-month study assessing the effectiveness and safety of damoctocog alfa pegol in real-world PTPs. 22 The first interim analysis of the HEM-POWR real-world study showed that the recombinant FVIII prevented bleeding and was well tolerated, with no study drug-related adverse events (AEs) in a small group of patients.…”

Section: Introductionmentioning

confidence: 99%

“…Currently, real‐world studies of prophylactic FVIII use in hemophilia A are limited 7,19–21 . HEM‐POWR is an ongoing, Phase 4, observational, 36‐month study assessing the effectiveness and safety of damoctocog alfa pegol in real‐world PTPs 22 .…”

Section: Introductionmentioning

confidence: 99%

Interim analyses of the multinational real‐world prospective cohort HEM‐POWR study evaluating the effectiveness and safety of damoctocog alfa pegol in patients with hemophilia A

Reding,

Álvarez‐Román,

Castaman

et al. 2023

European J of Haematology

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ObjectivesTo assess effectiveness and safety of damoctocog alfa pegol in interim analyses of the ongoing real‐world hemophilia A HEM‐POWR study.MethodsHEM‐POWR (NCT03932201) is a multinational Phase 4 prospective observational study. The primary objective was annualized bleeding rate (ABR) in previously treated patients (PTPs) with hemophilia A. Secondary objectives included adverse events and number of affected joints.ResultsAt data cut‐off (August 17, 2022), the safety analysis set included 268 patients and the full analysis set (FAS) included 161 patients. The most common dosing regimen during observation period was prophylaxis (FAS = 158/161, 98.1%) every 3–4 days (twice weekly; FAS = 78/158, 49.4%) and a median (min, max) infusion dose of 37.5 (10, 72) IU/kg. PTPs receiving prophylactic damoctocog alfa pegol have fewer infusions compared with prior treatment. Median total ABR (Q1, Q3) was 0.0 (0.0, 1.8) and mean total ABR (SD) was 2.4 (8.2). The proportion of patients with no affected joints increased between initial visit and follow‐up. No FVIII inhibitors, treatment‐related adverse events, or deaths were reported.ConclusionsDamoctocog alfa pegol shows effectiveness and acceptable safety, as well as consistent utilization, in real‐world PTPs with hemophilia A, including in patients with non‐severe hemophilia and those with a history of inhibitors. Please see video for a summary of this study.

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Real-world treatment, clinical outcomes and healthcare resource utilization among persons with hemophilia A by age

Cited by 7 publications

References 28 publications

Treatment regimens, patient reported outcomes and health-related quality of life in children with moderate and severe haemophilia A in China: using real-world data

Treatment regimens, patient reported outcomes and health-related quality of life in children with moderate and severe haemophilia A in China: using real-world data

Treatment regimens, patient reported outcomes and health-related quality of life in children with moderate and severe hemophilia A in China: using real-world data

Interim analyses of the multinational real‐world prospective cohort HEM‐POWR study evaluating the effectiveness and safety of damoctocog alfa pegol in patients with hemophilia A

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