An International Prophylaxis Study Group (IPSG) survey of prophylaxis in inhibitor positive children/adults with severe haemophilia

Carção, Manuel; Avila, Laura; Leissinger, Cindy; Blanchette, Victor S.; Aledort, L. M.

doi:10.1111/hae.13222

Cited by 3 publications

(4 citation statements)

References 10 publications

(10 reference statements)

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“…Prophylaxis with BPAs is especially burdensome, requiring infusions every other day . Consequently, the majority of PwHA with inhibitors receive episodic BPA treatment . The efficacy of BPAs in the treatment or prevention of bleeding has been shown to be suboptimal …”

Section: Introductionmentioning

confidence: 99%

Health‐related quality of life and health status in persons with haemophilia A with inhibitors: A prospective, multicentre, non‐interventional study (NIS)

Mahlangu

Oldenburg²,

Callaghan

et al. 2019

Haemophilia

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Introduction Real‐world data (RWD) on health‐related outcomes in persons with haemophilia A (PwHA) provide insights into patient needs and can guide clinical study design. A global, prospective, non‐interventional study (NIS; NCT02476942) collected detailed RWD on bleeding outcomes, health‐related quality of life (HRQoL) and health status in PwHA treated per local routine clinical practice. Aim To report HRQoL and health status in the adult/adolescent PwHA with inhibitors cohort in the NIS. Methods This cohort enrolled PwHA aged ≥12 years with high‐titre factor VIII inhibitor history. Participants remained on their usual treatment (no protocol‐specified interventions). Health‐related outcomes: Haemophilia Quality of Life Questionnaire for Adults (Haem‐A‐QoL), Haemophilia‐specific Quality of Life Questionnaire for Children Short Form (Haemo‐QoL SF), EuroQol 5‐Dimensions 5‐Levels (EQ‐5D‐5L) index utility score (IUS) and visual analogue scale (EQ‐VAS). Results One hundred three participants were enrolled on episodic (n = 75) or prophylactic treatment (n = 28); median (range) age, 31 (12‐75) years; median (range) observation time, 26 (4‐70) weeks. Haem‐A‐QoL scores indicated impairments in HRQoL aspects; comparable between episodic/prophylactic regimens and relatively consistent over time. Haemo‐QoL SF scores with both regimens varied over time, and appeared poorer with episodic than prophylactic treatment. IUS and EQ‐VAS were comparable between regimens, stable over time and lower on bleeding days. Mean proportions of missed work and school days were 16% and 23%, respectively; mean (standard deviation) number of days hospitalized was 3.2 (8.8) (comparable between groups). Conclusions These RWD demonstrate that PwHA with inhibitors have impaired HRQoL, despite standard treatment, and that more effective treatment options are needed.

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Section: Introductionmentioning

confidence: 99%

Health‐related quality of life and health status in persons with haemophilia A with inhibitors: A prospective, multicentre, non‐interventional study (NIS)

Mahlangu

Oldenburg²,

Callaghan

et al. 2019

Haemophilia

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“…Prophylaxis also protects from other forms of haemorrhage (including intracranial haemorrhage) . Recognizing its benefits, prophylaxis has become widely adopted, beginning first in more affluent and more recently in less affluent countries, as the ideal way of managing PWH …”

Section: Current Definitions Of Prophylaxis (A) Prophylaxis Defined mentioning

confidence: 99%

“…6 Recognizing its benefits, prophylaxis has become widely adopted, beginning first in more affluent and more recently in less affluent countries, as the ideal way of managing PWH. 7,8 Consensus definitions of prophylaxis have been developed according to when it is commenced (Table 1a) and according to its intensity (Table 1b). In addition, full time (or continuous prophylaxis) has been defined as being on prophylaxis for a minimum of 45 wk/y.…”

mentioning

confidence: 99%

Prophylaxis re‐visited: The potential impact of novel factor and non‐factor therapies on prophylaxis

et al. 2018

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“…After a detailed process of development and testing, the present version of the IPSG MRI scale was published in 2012, 7 tailored for scoring early and moderate MRI findings of HA. Several clinical trials 11‐13 and surveys 14,15 have utilized the IPSG MRI scale and assessed its cross‐sectional value.…”

Section: Introductionmentioning

confidence: 99%

Critical appraisal of the International Prophylaxis Study Group magnetic resonance image scale for evaluating haemophilic arthropathy

et al. 2020

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Bleeding into joint spaces (haemarthrosis) is a common complication in children with haemophilia. Repeated haemarthrosis leads to joint degradation and haemophilic arthropathy (HA). One goal of treating patients with haemophilia with prophylaxis (regular replacement therapy) is to decrease the frequency of joint bleeds and ultimately limit end organ damage. Observational and randomized controlled trials have demonstrated the effectiveness of prophylaxis. 1-3 However, validated and responsive clinical indices are still needed to monitor patient disease status and evaluate treatment regimens in longitudinal studies. Additionally, it is unclear how novel nonfactor therapeutics such as FVIII-mimetics and gene therapy affect joint health. If effective, a measure of early-stage HA could be instrumental to guide decisions between different prophylaxis regimens.

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An International Prophylaxis Study Group (IPSG) survey of prophylaxis in inhibitor positive children/adults with severe haemophilia

Cited by 3 publications

References 10 publications

Health‐related quality of life and health status in persons with haemophilia A with inhibitors: A prospective, multicentre, non‐interventional study (NIS)

Health‐related quality of life and health status in persons with haemophilia A with inhibitors: A prospective, multicentre, non‐interventional study (NIS)

Prophylaxis re‐visited: The potential impact of novel factor and non‐factor therapies on prophylaxis

Critical appraisal of the International Prophylaxis Study Group magnetic resonance image scale for evaluating haemophilic arthropathy

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