دورية أكاديمية
أعرض في EDS

Improvement in quality of life after asfotase alfa treatment in adults with pediatric-onset hypophosphatasia: data from 5 patient-reported outcome measures.

التفاصيل البيبلوغرافية
العنوان: Improvement in quality of life after asfotase alfa treatment in adults with pediatric-onset hypophosphatasia: data from 5 patient-reported outcome measures.
المؤلفون: Dahir KM; Endocrinology and Diabetes, Vanderbilt University Medical Center, Nashville, TN 37232, United States., Ing SW; Division of Endocrinology, Diabetes, and Metabolism, Wexner Medical Center, Ohio State University, Columbus, OH 43210, United States., Deal C; Department of Rheumatology, Center for Osteoporosis and Metabolic Bone Disease, Cleveland Clinic, Cleveland, OH 44195, United States., Messali A; Health Economics and Outcomes Research (AM) and Medical Affairs (CD), Alexion, AstraZeneca Rare Disease, Boston, MA 02210, United States., Bates T; Health Economics and Outcomes Research (AM) and Medical Affairs (CD), Alexion, AstraZeneca Rare Disease, Boston, MA 02210, United States., Rush ET; Division of Clinical Genetics, Children's Mercy Kansas City, Kansas City, MO 64108, United States.; Department of Pediatrics, University of Missouri - Kansas City School of Medicine, Kansas City, MO 64108, United States.
المصدر: JBMR plus [JBMR Plus] 2024 May 07; Vol. 8 (8), pp. ziae062. Date of Electronic Publication: 2024 May 07 (Print Publication: 2024).
نوع المنشور: Journal Article
اللغة: English
بيانات الدورية: Publisher: Oxford University Press Country of Publication: England NLM ID: 101707013 Publication Model: eCollection Cited Medium: Internet ISSN: 2473-4039 (Electronic) Linking ISSN: 24734039 NLM ISO Abbreviation: JBMR Plus Subsets: PubMed not MEDLINE
أسماء مطبوعة: Publication: 2024- : Oxford : Oxford University Press
Original Publication: [Chichester, West Sussex, UK] : John Wiley & Sons, [2017]-
مستخلص: 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.
Competing Interests: K.D. is a clinical trial investigator and consultant for Alexion, AstraZeneca Rare Disease, Kyowa Kirin, and Ultragenyx and collaborates with AM Pharma. S.W.I. has received research grants paid to his institution from Alexion, Amgen, Calcilytix, Radius, Takeda, and Ultragenyx and has served on an advisory board and/or as a consultant for Amgen, Bone Health & Osteoporosis Foundation, Extend Biosciences, and Radius. C.D. collaborates with Alexion, AstraZeneca Rare Disease, Amgen, and Radius and receives grants for research from Radius. A.M. and T.B. are employees of Alexion, AstraZeneca Rare Disease, and own stock/stock options in that company. E.R. collaborates with Alexion, AstraZeneca Rare Disease, Kyowa Kirin, Ultragenyx, and Inozyme. He receives research support from Alexion, AstraZeneca Rare Disease and Ultragenyx. The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.
(© The Author(s) 2024. Published by Oxford University Press on behalf of the American Society for Bone and Mineral Research.)
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فهرسة مساهمة: Keywords: asfotase alfa; hypophosphatasia; patient-reported outcome measures; quality of life; real-world evidence
تواريخ الأحداث: Date Created: 20240715 Latest Revision: 20240716
رمز التحديث: 20240716
مُعرف محوري في PubMed: PMC11245646
DOI: 10.1093/jbmrpl/ziae062
PMID: 39006866
قاعدة البيانات: MEDLINE
الوصف
تدمد:2473-4039
DOI:10.1093/jbmrpl/ziae062