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Asfotase Alfa Significantly Enhances Quality of Life in Adults with Hypophosphatasia

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In a groundbreaking study, researchers have demonstrated the positive impact of asfotase alfa on the quality of life for adults suffering from pediatric-onset hypophosphatasia (HPP). Hypophosphatasia is a rare metabolic disorder characterized by deficient activity of tissue-nonspecific alkaline phosphatase. This study, which enrolled 50 patients, utilized a series of patient-reported outcomes (PROs) to assess the effectiveness of the treatment over a 12-month period.

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Significant Improvements Detected Early

The longitudinal survey conducted via telephone included a variety of PROs, such as the Patient Health Questionnaire-9 (PHQ-9) and the Patient-Reported Outcomes Measurement Information System 29 (PROMIS-29). Initial evaluations were performed before the commencement of asfotase alfa therapy, followed by subsequent assessments at three, six, and twelve months. By the three-month mark, notable improvements from baseline were observed in PHQ-9 scores (10.6 vs 5.8, P < .0001) and several PROMIS-29 domains, including physical function, anxiety, fatigue, and sleep disturbances.

Long-term Benefits Evident

These benefits were not only early but also sustained over time. By the end of the study, the improvements in quality of life, as indicated by PHQ-9 and PROMIS-29 scores, persisted. Moreover, the Routine Assessment of Patient Index Data 3 (RAPID3) scores reflected enhanced functional status, pain tolerance, and global health estimates. Market access to asfotase alfa could be pivotal for broader patient communities, offering a substantial improvement in daily activities and overall well-being.

Furthermore, by the six-month milestone, an increased capacity for work and reduced presenteeism were recorded, as evidenced by the Work Productivity and Activity Impairment Questionnaire: Specific Health Problem (WPAI:SHP) scores. Specifically, presenteeism dropped from 39.6% to 14.1%, and work productivity loss decreased from 41.9% to 14.1%, both with high statistical significance (P < .0001).

Concrete Inferences

– Early and sustained improvements in mental health and physical function.

– Significant reduction in anxiety, fatigue, and sleep disturbances.

– Enhanced work productivity and reduced presenteeism.

– Wider market access to asfotase alfa could benefit more HPP patients.

In conclusion, asfotase alfa treatment has been shown to significantly enhance the quality of life for adults with pediatric-onset HPP. This study highlights not only the clinical benefits but also the potential socio-economic advantages, such as improved work productivity, further validating the importance of market access to this transformative therapy.

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Original Article:

JBMR Plus. 2024 May 7;8(8):ziae062. doi: 10.1093/jbmrpl/ziae062. eCollection 2024 Aug.

ABSTRACT

Hypophosphatasia (HPP) is a rare, inherited metabolic disorder caused by deficient tissue-nonspecific alkaline phosphatase activity. This study assessed the impact of treatment with asfotase alfa on patient-reported outcomes (PROs) in adults with pediatric-onset HPP. A longitudinal, telephone-based survey was administered to eligible individuals enrolled in a patient support program. Interviews were conducted at study entry (prior to asfotase alfa initiation) and after 3, 6, and 12 mo. PROs-Patient Health Questionnaire-9 [PHQ-9], Work Productivity and Activity Impairment Questionnaire: Specific Health Problem [WPAI:SHP], Patient-Reported Outcomes Measurement Information System 29 [PROMIS-29], and Routine Assessment of Patient Index Data 3 [RAPID3]-were assessed at each time point. Appropriate statistical tests were performed to assess score changes. Among 50 enrolled patients (mean age: 46 yr [SD: 15.4]; 80% female; 94% White), 49 were evaluable at 3 mo, 44 at 6 mo, and 29 at 12 mo. By month 3, statistically significant improvements from baseline were detected in PHQ-9 scores (10.6 vs 5.8 [P < .0001]), PROMIS-29 domain scores (overall physical function: 38.0 vs 43.0 [P = .001]; anxiety: 57.5 vs 51.5 [P = .0011]; fatigue: 63.3 vs 55.3 [P < .0001]; sleep disturbances: 58.8 vs 54.3 [P = .0099]; ability to participate in social roles and activities: 42.6 vs 47.7 [P = .0012]; and pain interference: 63.8 vs 58.4 [P = .001]), and RAPID3 domain scores (functional status: 2.7 vs 1.1 [P < .0001]; pain tolerance: 6.0 vs 3.2 [P < .0001]; and global health estimate: 5.1 vs 2.7 [P < .0001]). Improvements persisted at month 12. Patients also showed improvements in WPAI:SHP domain scores at month 6 (presenteeism: 39.6% vs 14.1% [P < .0001] and work productivity loss: 41.9% vs 14.1% [P < .0001]). Treatment with asfotase alfa was associated with improved quality of life across several domains.

PMID:39006866 | PMC:PMC11245646 | DOI:10.1093/jbmrpl/ziae062


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