rhPTH(1-84) for hypoparathyroidism: a randomized study of patient-reported outcomes

Maria Luisa Brandi; Tamara Vokes; Natasha M. Appelman-Dijkstra; Olulade Ayodele; Brigitte Decallonne; Renate de Jongh; Manuel Díaz-Curiel; William Fraser; Richard D. Finkelman; Ansgar Heck; Steven W. Ing; Peter Kamenický; Aliya A. Khan; Christopher S. Kovacs; Bruno Lapauw; Graham Leese; Giovanna Mantovani; Guillermo Martínez Díaz-Guerra; Laura Masi; Miguel Melo; Andrea Palermo; Narendra L. Reddy; Lars Rejnmark; Elena Tokareva; Marie-Christine Vantyghem; Suwei Wang; Mark Warren; Brian Yan

doi:10.1093/ejendo/lvaf148

rhPTH(1-84) for hypoparathyroidism: a randomized study of patient-reported outcomes

Maria Luisa Brandi
, Tamara Vokes
, Natasha M. Appelman-Dijkstra
, Olulade Ayodele (Lead / Corresponding author)
, Brigitte Decallonne
, Renate de Jongh
, Manuel Díaz-Curiel
, William Fraser
, Richard D. Finkelman
, Ansgar Heck
, Steven W. Ing
, Peter Kamenický
, Aliya A. Khan
, Christopher S. Kovacs
, Bruno Lapauw
, Graham Leese
, Giovanna Mantovani
, Guillermo Martínez Díaz-Guerra
, Laura Masi
, Miguel Melo

Andrea Palermo, Narendra L. Reddy, Lars Rejnmark, Elena Tokareva, Marie-Christine Vantyghem, Suwei Wang, Mark Warren, Brian Yan

Diabetes Endocrinology and Reproductive Biology

Research output: Contribution to journal › Article › peer-review

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Abstract

Objective: To assess the impact of recombinant human parathyroid hormone (1-84) [rhPTH(1-84)] compared with placebo, in combination with conventional therapy with vitamin D and/or calcium supplements, on health-related quality of life (HRQoL) in patients with symptomatic chronic hypoparathyroidism (cHypoPT). Design: Randomized, double-blind, placebo-controlled, phase 3b-4 study (ClinicalTrials.gov ID: NCT03324880). Methods: Eligible patients with symptomatic cHypoPT were randomized to receive subcutaneous rhPTH(1-84) 25-100 µg/day or placebo. The primary endpoint was the change from baseline to week 26 in Hypoparathyroidism Symptom Diary (HypoPT-SD) symptom subscale score. Key secondary endpoints were changes from baseline to week 26 in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue and in 36-item Short Form Health Survey physical component summary (SF-36v2 PCS). Results: In total, 93 patients were randomized to receive treatment: 45 received rhPTH(1-84) and 48 received placebo. Change from baseline to week 26 in HypoPT-SD symptom subscale score was significantly greater (improved) in the rhPTH(1-84) group than in the placebo group (difference in least-squares mean changes, −0.53; 95% confidence interval, −0.90 to −0.15, P = .003). Key secondary endpoints, changes between baseline and week 26 in the FACIT-Fatigue and SF-36v2 PCS scores were also significantly greater (improved) in the rhPTH(1-84) group than in the placebo group. The safety profile of rhPTH(1-84) was consistent with previous findings, and no new safety signals were identified. Conclusions: rhPTH(1-84) alongside conventional therapy improved symptom burden (as measured by the HypoPT-SD) and HRQoL to a greater extent than conventional therapy alone in patients with symptomatic cHypoPT.

Original language	English
Pages (from-to)	310-319
Number of pages	10
Journal	European Journal of Endocrinology
Volume	193
Issue number	2
Early online date	25 Jul 2025
DOIs	https://doi.org/10.1093/ejendo/lvaf148
Publication status	Published - Aug 2025

Keywords

human parathyroid hormone treatment
hypoparathyroidism
patient-reported outcomes
rhPTH(1-84)

ASJC Scopus subject areas

Endocrinology, Diabetes and Metabolism
Endocrinology

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

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10.1093/ejendo/lvaf148Licence: CC BY

Final published versionFinal published version, 584 KBLicence: CC BY

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Brandi, M. L., Vokes, T., Appelman-Dijkstra, N. M., Ayodele, O., Decallonne, B., de Jongh, R., Díaz-Curiel, M., Fraser, W., Finkelman, R. D., Heck, A., Ing, S. W., Kamenický, P., Khan, A. A., Kovacs, C. S., Lapauw, B., Leese, G., Mantovani, G., Díaz-Guerra, G. M., Masi, L., ... Yan, B. (2025). rhPTH(1-84) for hypoparathyroidism: a randomized study of patient-reported outcomes. European Journal of Endocrinology, 193(2), 310-319. https://doi.org/10.1093/ejendo/lvaf148

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title = "rhPTH(1-84) for hypoparathyroidism: a randomized study of patient-reported outcomes",

abstract = "Objective: To assess the impact of recombinant human parathyroid hormone (1-84) [rhPTH(1-84)] compared with placebo, in combination with conventional therapy with vitamin D and/or calcium supplements, on health-related quality of life (HRQoL) in patients with symptomatic chronic hypoparathyroidism (cHypoPT). Design: Randomized, double-blind, placebo-controlled, phase 3b-4 study (ClinicalTrials.gov ID: NCT03324880). Methods: Eligible patients with symptomatic cHypoPT were randomized to receive subcutaneous rhPTH(1-84) 25-100 µg/day or placebo. The primary endpoint was the change from baseline to week 26 in Hypoparathyroidism Symptom Diary (HypoPT-SD) symptom subscale score. Key secondary endpoints were changes from baseline to week 26 in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue and in 36-item Short Form Health Survey physical component summary (SF-36v2 PCS). Results: In total, 93 patients were randomized to receive treatment: 45 received rhPTH(1-84) and 48 received placebo. Change from baseline to week 26 in HypoPT-SD symptom subscale score was significantly greater (improved) in the rhPTH(1-84) group than in the placebo group (difference in least-squares mean changes, −0.53; 95\% confidence interval, −0.90 to −0.15, P = .003). Key secondary endpoints, changes between baseline and week 26 in the FACIT-Fatigue and SF-36v2 PCS scores were also significantly greater (improved) in the rhPTH(1-84) group than in the placebo group. The safety profile of rhPTH(1-84) was consistent with previous findings, and no new safety signals were identified. Conclusions: rhPTH(1-84) alongside conventional therapy improved symptom burden (as measured by the HypoPT-SD) and HRQoL to a greater extent than conventional therapy alone in patients with symptomatic cHypoPT.",

keywords = "human parathyroid hormone treatment, hypoparathyroidism, patient-reported outcomes, rhPTH(1-84)",

author = "Brandi, \{Maria Luisa\} and Tamara Vokes and Appelman-Dijkstra, \{Natasha M.\} and Olulade Ayodele and Brigitte Decallonne and \{de Jongh\}, Renate and Manuel D{\'i}az-Curiel and William Fraser and Finkelman, \{Richard D.\} and Ansgar Heck and Ing, \{Steven W.\} and Peter Kamenick{\'y} and Khan, \{Aliya A.\} and Kovacs, \{Christopher S.\} and Bruno Lapauw and Graham Leese and Giovanna Mantovani and D{\'i}az-Guerra, \{Guillermo Mart{\'i}nez\} and Laura Masi and Miguel Melo and Andrea Palermo and Reddy, \{Narendra L.\} and Lars Rejnmark and Elena Tokareva and Marie-Christine Vantyghem and Suwei Wang and Mark Warren and Brian Yan",

note = "Publisher Copyright: {\textcopyright} 2025 The Author(s)",

year = "2025",

month = aug,

doi = "10.1093/ejendo/lvaf148",

language = "English",

volume = "193",

pages = "310--319",

journal = "European Journal of Endocrinology",

issn = "0804-4643",

publisher = "Oxford University Press",

number = "2",

}

Brandi, ML, Vokes, T, Appelman-Dijkstra, NM, Ayodele, O, Decallonne, B, de Jongh, R, Díaz-Curiel, M, Fraser, W, Finkelman, RD, Heck, A, Ing, SW, Kamenický, P, Khan, AA, Kovacs, CS, Lapauw, B, Leese, G, Mantovani, G, Díaz-Guerra, GM, Masi, L, Melo, M, Palermo, A, Reddy, NL, Rejnmark, L, Tokareva, E, Vantyghem, M-C, Wang, S, Warren, M & Yan, B 2025, 'rhPTH(1-84) for hypoparathyroidism: a randomized study of patient-reported outcomes', European Journal of Endocrinology, vol. 193, no. 2, pp. 310-319. https://doi.org/10.1093/ejendo/lvaf148

TY - JOUR

T1 - rhPTH(1-84) for hypoparathyroidism

T2 - a randomized study of patient-reported outcomes

AU - Brandi, Maria Luisa

AU - Vokes, Tamara

AU - Appelman-Dijkstra, Natasha M.

AU - Ayodele, Olulade

AU - Decallonne, Brigitte

AU - de Jongh, Renate

AU - Díaz-Curiel, Manuel

AU - Fraser, William

AU - Finkelman, Richard D.

AU - Heck, Ansgar

AU - Ing, Steven W.

AU - Kamenický, Peter

AU - Khan, Aliya A.

AU - Kovacs, Christopher S.

AU - Lapauw, Bruno

AU - Leese, Graham

AU - Mantovani, Giovanna

AU - Díaz-Guerra, Guillermo Martínez

AU - Masi, Laura

AU - Melo, Miguel

AU - Palermo, Andrea

AU - Reddy, Narendra L.

AU - Rejnmark, Lars

AU - Tokareva, Elena

AU - Vantyghem, Marie-Christine

AU - Wang, Suwei

AU - Warren, Mark

AU - Yan, Brian

PY - 2025/8

Y1 - 2025/8

N2 - Objective: To assess the impact of recombinant human parathyroid hormone (1-84) [rhPTH(1-84)] compared with placebo, in combination with conventional therapy with vitamin D and/or calcium supplements, on health-related quality of life (HRQoL) in patients with symptomatic chronic hypoparathyroidism (cHypoPT). Design: Randomized, double-blind, placebo-controlled, phase 3b-4 study (ClinicalTrials.gov ID: NCT03324880). Methods: Eligible patients with symptomatic cHypoPT were randomized to receive subcutaneous rhPTH(1-84) 25-100 µg/day or placebo. The primary endpoint was the change from baseline to week 26 in Hypoparathyroidism Symptom Diary (HypoPT-SD) symptom subscale score. Key secondary endpoints were changes from baseline to week 26 in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue and in 36-item Short Form Health Survey physical component summary (SF-36v2 PCS). Results: In total, 93 patients were randomized to receive treatment: 45 received rhPTH(1-84) and 48 received placebo. Change from baseline to week 26 in HypoPT-SD symptom subscale score was significantly greater (improved) in the rhPTH(1-84) group than in the placebo group (difference in least-squares mean changes, −0.53; 95% confidence interval, −0.90 to −0.15, P = .003). Key secondary endpoints, changes between baseline and week 26 in the FACIT-Fatigue and SF-36v2 PCS scores were also significantly greater (improved) in the rhPTH(1-84) group than in the placebo group. The safety profile of rhPTH(1-84) was consistent with previous findings, and no new safety signals were identified. Conclusions: rhPTH(1-84) alongside conventional therapy improved symptom burden (as measured by the HypoPT-SD) and HRQoL to a greater extent than conventional therapy alone in patients with symptomatic cHypoPT.

AB - Objective: To assess the impact of recombinant human parathyroid hormone (1-84) [rhPTH(1-84)] compared with placebo, in combination with conventional therapy with vitamin D and/or calcium supplements, on health-related quality of life (HRQoL) in patients with symptomatic chronic hypoparathyroidism (cHypoPT). Design: Randomized, double-blind, placebo-controlled, phase 3b-4 study (ClinicalTrials.gov ID: NCT03324880). Methods: Eligible patients with symptomatic cHypoPT were randomized to receive subcutaneous rhPTH(1-84) 25-100 µg/day or placebo. The primary endpoint was the change from baseline to week 26 in Hypoparathyroidism Symptom Diary (HypoPT-SD) symptom subscale score. Key secondary endpoints were changes from baseline to week 26 in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue and in 36-item Short Form Health Survey physical component summary (SF-36v2 PCS). Results: In total, 93 patients were randomized to receive treatment: 45 received rhPTH(1-84) and 48 received placebo. Change from baseline to week 26 in HypoPT-SD symptom subscale score was significantly greater (improved) in the rhPTH(1-84) group than in the placebo group (difference in least-squares mean changes, −0.53; 95% confidence interval, −0.90 to −0.15, P = .003). Key secondary endpoints, changes between baseline and week 26 in the FACIT-Fatigue and SF-36v2 PCS scores were also significantly greater (improved) in the rhPTH(1-84) group than in the placebo group. The safety profile of rhPTH(1-84) was consistent with previous findings, and no new safety signals were identified. Conclusions: rhPTH(1-84) alongside conventional therapy improved symptom burden (as measured by the HypoPT-SD) and HRQoL to a greater extent than conventional therapy alone in patients with symptomatic cHypoPT.

KW - human parathyroid hormone treatment

KW - hypoparathyroidism

KW - patient-reported outcomes

KW - rhPTH(1-84)

UR - https://www.scopus.com/pages/publications/105013638884

U2 - 10.1093/ejendo/lvaf148

DO - 10.1093/ejendo/lvaf148

M3 - Article

C2 - 40711996

AN - SCOPUS:105013638884

SN - 0804-4643

VL - 193

SP - 310

EP - 319

JO - European Journal of Endocrinology

JF - European Journal of Endocrinology

IS - 2

ER -

rhPTH(1-84) for hypoparathyroidism: a randomized study of patient-reported outcomes

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Keywords

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