Quality of Life in Hypoparathyroidism Improves With rhPTH(1-84) Throughout 8 Years of Therapy

Author:

Tabacco Gaia12ORCID,Tay Yu-Kwang Donovan3,Cusano Natalie E4,Williams John1,Omeragic Beatriz1,Majeed Rukhana1,Almonte Maximo Gomez5,Rubin Mishaela R1,Bilezikian John P1

Affiliation:

1. Division of Endocrinology, Department of Medicine, College of Physicians & Surgeons, Columbia University, New York, New York

2. Unit of Endocrinology and Diabetes, Department of Medicine, University Campus Bio-Medico, Rome, Italy

3. Department of Medicine, Sengkang General Hospital, Singapore

4. Division of Endocrinology, Department of Medicine, Lenox Hill Hospital, New York, New York

5. Division of Cardiology, Department of Medicine, Wyckoff Heights Medical Center, New York, New York

Abstract

Abstract Context Calcium and vitamin D treatment does not improve reduced quality of life (QOL) in hypoparathyroidism. Recombinant human (rh) PTH(1-84) therapy improves QOL metrics for up to 5 years. Data on QOL beyond this time point are not available. Objectives To evaluate the effects of 8 years of rhPTH(1-84) therapy on QOL and factors associated with long-term benefit. Design Prospective, open-label trial. Setting Referral center. Patients Twenty patients with hypoparathyoidism. Main Outcome Measures RAND 36-Item Short Form Health Survey (SF-36). Results rhPTH therapy led to substantial improvement in five of the eight SF-36 domains [vitality, social functioning (SF), mental health (MH), bodily pain (BP) and general health] and three of these domains (SF, MH, BP) were no longer lower than the reference population. The improvement in the mental component summary (MCS) score was sustained through 8 years, while the physical component summary (PCS) score improved through 6 years. A lower baseline QOL score was associated with greater improvement. A threshold value <238 (MCS) and <245 (PCS) predicted long-term improvement in 90% and 100% of the cohort, respectively. In patients whose calcium supplementation was reduced, MCS and PCS scores improved more than those whose supplementation did not decline to the same extent. Improvement in PCS was greater in patients whose calcitriol dosage was reduced and duration of disease was shorter. Conclusions rhPTH(1-84) improves long-term well-being in hypoparathyroidism. The improvements are most prominent in those with impaired SF-36 at baseline and those whose requirements for conventional therapy decreased substantially.

Funder

National Institutes of Health

Shire

Food and Drug Administration

Publisher

The Endocrine Society

Subject

Biochemistry (medical),Clinical Biochemistry,Endocrinology,Biochemistry,Endocrinology, Diabetes and Metabolism

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