ECE2022 Rapid Communications Rapid Communications 8: Calcium and Bone (7 abstracts)
Health related quality of life in adults with hypoparathyroidism in an exploratory analysis of the phase 2 PaTH forward trial of TransCon PTH
Andrea Palermo 1 , Tamara J Vokes 2 , Aliya Khan 3 , Mishaela Rubin 4 , Peter Schwarz 5 , Bart L Clarke 6 , Erik Eriksen 7 , Claudio Marcocci 8 , Uberto Pagotto 9 , Elena Tsourdi 10 , Tanja Sikjaer 11 , Meryl Brod 12 , Lori Mcleod 13 , Denka Markova 14 , Wahidullah Noori 14 , Walter Frank Eng 14 , Aimee Shu 14 & Alden Smith 14
1Campus Bio Medico University of Rome, Selcetta, Italy; 2The University of Chicago, Chicago, United States; 3McMaster University, Department of Endocrinology and Metabolism and Geriatrics, Hamilton, Canada; 4Columbia University, New York, United States; 5Rigshospitalet, Department of Endocrinology, København, Denmark; 6Mayo Clinic, Rochester, United States; 7Oslo University Hospital, Institute of Clinical Medicine, Oslo, Norway; 8University of Pisa, Pisa, Italy; 9Alma Mater Studiorum - Università di Bologna, School of Medicine and Surger, Bologna, Italy; 10Dresden University of Technology, Department of Medicine III and Center for Healthy Aging, Dresden, Germany; 11University of Aarhus, Department of Endocrinology and Diabetes, Aarhus, Denmark; 12The Brod Group, Mill Valley, United States; 13RTI Health Solutions, Durham, United States; 14Ascendis Pharma, Inc, Palo Alto, United States
Background: Patients with hypoparathyroidism experience significant physical and cognitive symptoms and reduced health-related quality of life (HRQoL). Conventional therapy for hypoparathyroidism does not fully alleviate diminished HRQoL. The Hypoparathyroidism Patient Experience Scales (HPES) were developed to assess disease-specific physical and cognitive symptoms as well as the impact of hypoparathyroidsm on HRQol. TransCon PTH, an investigational long-acting prodrug of parathyroid hormone (PTH[1-34]), is in development as a potential hormone replacement therapy for adults with hypoparathyroidism.
Methods: The phase 2, randomized, double blind placebo controlled 4-week PaTH Forward trial was followed by an open label extension period and enrolled 59 participants. HRQoL was assessed at Week 4 and Week 26 in an exploratory ad-hoc analysis using HPES and the 36-Item Short Form Survey (SF-36) to assess the role of HPES in measuring the impact of TransCon PTH on HRQoL
Results: Improvements in HPES scores were significantly greater for participants treated with TransCon PTH (n=44) compared with placebo (n=15) from baseline to Week 4 of the trial for both HPES-Total Symptom score (Mean difference [standard error] in scores -20.0 [4.6], P < 0.01; 84% of treated patients had improved scores versus 47% of placebo, P=0.013) and HPES-Total Impact Score (Mean difference [standard error] in scores -15.7 [5.0], P< 0.01; 75% of treated patients had improved scores versus 40% of placebo, P =0.025). All HRQoL assessments demonstrated continued score improvements in patients treated with TransCon PTH at Week 26 of the trial compared with baseline. Participants treated with TransCon PTH who had higher (worse) HPES (Impact & Symptom) scores at baseline demonstrated a greater magnitude of improvement at Week 26 as did participants with lower (worse) SF-36 scores at baseline. The Pearson correlation analysis showed that HPES results diverged from SF-36 results over time, with a greater number of domain scores having a correlation of -0.40 or larger at Week 4 compared with Week 26.
Conclusions: This exploratory ad-hoc analysis of the PaTH Forward trial demonstrated improved HRQoL and symptom scores in participants treated with TransCon PTH compared with placebo. In addition, correlation analyses identified the distinct value and dynamic range of HPES as disease-specific assessment tools in hypoparathyroidism. The decreased correlation between HPES and SF-36 over time in the trial indicates that HPES and SF-36 are not redundant.
Article tools
My recent searches
My recently viewed abstracts
Authors
Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
© Bioscientifica 2024 |
Privacy policy |
Cookie settings
BiosciAbstracts
Bioscientifica Abstracts is the gateway to a series of products that provide a permanent, citable record of abstracts for biomedical and life science conferences.
Find out more