Improvement in quality of life after asfotase alfa treatment in adults with pediatric-onset hypophosphatasia: data from 5 patient-reported outcome measures.
Kathryn McCrystal DahirSteven W IngChad DealAndrew MessaliToby BatesEric T RushPublished in: JBMR plus (2024)
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.
Keyphrases
- patient reported outcomes
- replacement therapy
- patient reported
- public health
- end stage renal disease
- healthcare
- global health
- mental health
- chronic pain
- case report
- newly diagnosed
- chronic kidney disease
- cross sectional
- climate change
- electronic health record
- prognostic factors
- pain management
- depressive symptoms
- neuropathic pain
- big data
- combination therapy
- young adults
- peritoneal dialysis
- machine learning
- quality improvement
- spinal cord
- risk assessment
- clinical practice
- psychometric properties
- human health