ECE2023 Poster Presentations Calcium and Bone (83 abstracts)
TransCon PTH Improves Health-Related Quality of Life and Reduces Work Limitations in Adults With Hypoparathyroidism: Patient-Reported Outcomes in the Phase 3 PaTHway Trial
Andrea Palermo 1 , Aliya Khan 2 , Mishaela Rubin 3 , Peter Schwarz 4 , Dolores M. Shoback 5 , Claudia Gagnon 6,7 , Filomena Cetani 8 , Bart L. Clarke 9 , Elena Tsourdi 10 , Lynn Kohlmeier 11 , Tanja Sikjaer 12 , Stephanie M Kaiser 13 , Bryant Lai 14 , John Le 14 , Jenny Ukena 14 , Christopher Sibley 14 , Aimee Shu 14 , Xubei An 14 , Wahidullah Noori 14 , Alden Smith 14 & Tamara J. Vokes 15
1Campus Bio Medico University of Rome, Selcetta, Italy; 2McMaster University, Department of Endocrinology and Metabolism and Geriatrics, Hamilton, Canada; 3Columbia University, New York, NY, United States; 4Rigshospitalet, Department of Endocrinology, København, Denmark; 5UCSF/VA Medical Center, San Francisco, United States; 6CHU de Québec-Université Laval Research Centre, Québec, Canada; 7Université Laval, Department of Medicine, Québec City, Canada; 8University Hospital of Pisa, Endocrine Unit, Pisa, Italy; 9Mayo Clinic, Rochester, NY, United States; 10Dresden University of Technology, Department of Medicine III and Center for Healthy Aging, Dresden, Germany; 11Endocrinology and Spokane Osteoporosis, Spokane, WA, United States; 12University of Aarhus, Department of Endocrinology and Diabetes, Aarhus, Denmark; 13Dalhousie University, Nova Scotia, Canada; 14Ascendis Pharma, Inc, Palo Alto, CA, United States; 15The University of Chicago, Chicago, IL, United States
Background: Individuals with hypoparathyroidism often experience a range of symptoms associated with reduced health-related quality of life (HRQoL) and work productivity. Conventional therapy aims to alleviate hypocalcemia and acute symptoms but fails to restore normal parathyroid hormone (PTH) physiology or improve HRQoL. In the PaTHway trial, 79% of participants treated with TransCon PTH vs. 5% placebo (P<0.0001) met the primary efficacy endpoint (normal serum calcium and independence from conventional therapy [no active vitamin D and ≤ 600 mg/day of calcium]) (Khan AA et al, JBMR, 2022). This analysis assessed the impact of TransCon PTH on hypoparathyroidism-related symptoms, functioning, and well-being; HRQoL; and work limitations in the PaTHway trial.
Methods: PaTHway is a randomized, double-blind, placebo-controlled, 26-week phase 3 trial designed to evaluate the efficacy, safety, and tolerability of TransCon PTH. Patient-Reported Outcomes were collected at baseline and Weeks 10, 20, and 26. Descriptive statistics are reported for the Hypoparathyroidism Patient Experience Scale (HPES), 36-Item Short-Form Health Survey (SF-36), and Work Limitations Questionnaire (WLQ). Higher scores are associated with improved HRQoL (SF-36) but greater symptom frequency/impact (HPES) and more self-reported difficulties at work (WLQ).
Results: Overall, 82 participants received at least one dose of study drug, and 79 completed blinded treatment (60 TransCon PTH, 19 placebo). The TransCon PTH group had greater improvements in all mean HPES scores than placebo. With 26 weeks of TransCon PTH treatment, mean HPES-Symptom cognitive and physical domain scores improved from 39.3 and 41.4 at baseline to 18.1 and 21.6, respectively. Mean HPES-Impact daily life, psychological well-being, physical functioning, and social life and relationship domain scores ranged from 25.2-35.9 at baseline and improved to 11.4-17.3. With TransCon PTH, but not placebo, all mean SF-36 subscale scores improved from baseline to Week 26; mean physical functioning, role emotional, role physical, vitality, social function, and bodily pain scores improved into the normative range for the United States general population from Week 10 through 26. Employed participants treated with TransCon PTH had lower mean WLQ scores at Weeks 10, 20, and 26 than placebo. With TransCon PTH, mean WLQ scores improved from 32.0 at baseline to 11.3 at Week 26 for time management, 27.8 to 8.9 for mental-interpersonal demands, 31.2 to 9.9 for output tasks, and 20.4 to 9.0 for physical tasks.
Conclusions: TransCon PTH reduced hypoparathyroidism-related symptoms and self-reported difficulties at work and increased functioning and well-being. Longitudinal improvements in HRQoL were also seen with TransCon PTH treatment.
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Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
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