Patient-Reported Outcomes in Palopegteriparatide-Treated Adults with Hypoparathyroidism: PaTH Forward Trial Extension

Mishaela Rubin; Andrea Palermo; Tamara Vokes; Aliya A Khan; Peter Schwarz; Filomena Cetani; Uberto Pagotto; Elena Tsourdi; Tanja Sikjaer; Kathryn M Pfeiffer; Meryl Brod; Lori D McLeod; Carol Zhao; Wahidullah Noori; Aimee D Shu; Alden R Smith

doi:10.1210/clinem/dgaf653

Patient-Reported Outcomes in Palopegteriparatide-Treated Adults with Hypoparathyroidism: PaTH Forward Trial Extension

Mishaela Rubin, Andrea Palermo, Tamara Vokes, Aliya A Khan, Peter Schwarz, Filomena Cetani, Uberto Pagotto, Elena Tsourdi, Tanja Sikjaer, Kathryn M Pfeiffer, Meryl Brod, Lori D McLeod, Carol Zhao, Wahidullah Noori, Aimee D Shu, Alden R Smith

Afdeling for Nyre- og Hormonsygdomme CKO

Abstract

CONTEXT: Individuals with hypoparathyroidism experience a range of physical and cognitive symptoms and reduced quality of life (QoL) despite management with conventional therapy (active vitamin D and calcium).

OBJECTIVE: This analysis investigated the long-term impact of parathyroid hormone (PTH) replacement therapy with palopegteriparatide (YORVIPATH®) on symptoms, daily functioning, and well-being in adults with chronic hypoparathyroidism. Associations between patient characteristics and changes in patient-reported outcomes (PROs) were also analyzed.

METHODS: PaTH Forward was a phase 2 clinical trial of palopegteriparatide with a 4-week randomized, double-blind, placebo-controlled period followed by an open-label extension period lasting through trial week 266. PRO measures were collected at baseline, weeks 4, 12, 26, 58, and annually thereafter through the end of the trial. The Hypoparathyroidism Patient Experience Scales (HPES) assess disease-specific symptoms and impacts on functioning and well-being. The Short-Form Health Survey (SF-36v2) measures general health-related QoL. Data were analyzed using descriptive statistics and Mixed Models for Repeated Measures (MMRM).

RESULTS: Palopegteriparatide treatment demonstrated significant improvements from baseline in disease-specific symptoms and impacts on daily functioning and well-being at week 12, which were sustained through week 110. Mean changes in PROs met thresholds for clinically meaningful within-patient improvement. Significant improvements in general health-related QoL were also shown in SF-36v2 scores. Results were generally similar across demographics and patient characteristics.

CONCLUSION: Through week 110 of the PaTH Forward trial, PTH replacement therapy with palopegteriparatide was associated with significant improvements in disease-specific symptoms and impacts on daily functioning and well-being, as well as general health-related QoL.

Originalsprog	Engelsk
Tidsskrift	The Journal of clinical endocrinology and metabolism
ISSN	0021-972X
DOI	https://doi.org/10.1210/clinem/dgaf653
Status	E-pub ahead of print - 9 dec. 2025

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10.1210/clinem/dgaf653Licens: CC BY

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Rubin, M., Palermo, A., Vokes, T., Khan, A. A., Schwarz, P., Cetani, F., Pagotto, U., Tsourdi, E., Sikjaer, T., Pfeiffer, K. M., Brod, M., McLeod, L. D., Zhao, C., Noori, W., Shu, A. D., & Smith, A. R. (2025). Patient-Reported Outcomes in Palopegteriparatide-Treated Adults with Hypoparathyroidism: PaTH Forward Trial Extension. The Journal of clinical endocrinology and metabolism. Advance online publication. https://doi.org/10.1210/clinem/dgaf653

Rubin, M, Palermo, A, Vokes, T, Khan, AA, Schwarz, P, Cetani, F, Pagotto, U, Tsourdi, E, Sikjaer, T, Pfeiffer, KM, Brod, M, McLeod, LD, Zhao, C, Noori, W, Shu, AD & Smith, AR 2025, 'Patient-Reported Outcomes in Palopegteriparatide-Treated Adults with Hypoparathyroidism: PaTH Forward Trial Extension', The Journal of clinical endocrinology and metabolism. https://doi.org/10.1210/clinem/dgaf653

@article{c64c98cd63ea473c903df53da6263fe4,

title = "Patient-Reported Outcomes in Palopegteriparatide-Treated Adults with Hypoparathyroidism: PaTH Forward Trial Extension",

abstract = "CONTEXT: Individuals with hypoparathyroidism experience a range of physical and cognitive symptoms and reduced quality of life (QoL) despite management with conventional therapy (active vitamin D and calcium).OBJECTIVE: This analysis investigated the long-term impact of parathyroid hormone (PTH) replacement therapy with palopegteriparatide (YORVIPATH{\textregistered}) on symptoms, daily functioning, and well-being in adults with chronic hypoparathyroidism. Associations between patient characteristics and changes in patient-reported outcomes (PROs) were also analyzed.METHODS: PaTH Forward was a phase 2 clinical trial of palopegteriparatide with a 4-week randomized, double-blind, placebo-controlled period followed by an open-label extension period lasting through trial week 266. PRO measures were collected at baseline, weeks 4, 12, 26, 58, and annually thereafter through the end of the trial. The Hypoparathyroidism Patient Experience Scales (HPES) assess disease-specific symptoms and impacts on functioning and well-being. The Short-Form Health Survey (SF-36v2) measures general health-related QoL. Data were analyzed using descriptive statistics and Mixed Models for Repeated Measures (MMRM).RESULTS: Palopegteriparatide treatment demonstrated significant improvements from baseline in disease-specific symptoms and impacts on daily functioning and well-being at week 12, which were sustained through week 110. Mean changes in PROs met thresholds for clinically meaningful within-patient improvement. Significant improvements in general health-related QoL were also shown in SF-36v2 scores. Results were generally similar across demographics and patient characteristics.CONCLUSION: Through week 110 of the PaTH Forward trial, PTH replacement therapy with palopegteriparatide was associated with significant improvements in disease-specific symptoms and impacts on daily functioning and well-being, as well as general health-related QoL.",

author = "Mishaela Rubin and Andrea Palermo and Tamara Vokes and Khan, {Aliya A} and Peter Schwarz and Filomena Cetani and Uberto Pagotto and Elena Tsourdi and Tanja Sikjaer and Pfeiffer, {Kathryn M} and Meryl Brod and McLeod, {Lori D} and Carol Zhao and Wahidullah Noori and Shu, {Aimee D} and Smith, {Alden R}",

note = "{\textcopyright} The Author(s) 2025. Published by Oxford University Press on behalf of the Endocrine Society.",

year = "2025",

month = dec,

day = "9",

doi = "10.1210/clinem/dgaf653",

language = "English",

journal = "The Journal of clinical endocrinology and metabolism",

issn = "0021-972X",

publisher = "Endocrine Society",

}

TY - JOUR

T1 - Patient-Reported Outcomes in Palopegteriparatide-Treated Adults with Hypoparathyroidism

T2 - PaTH Forward Trial Extension

AU - Rubin, Mishaela

AU - Palermo, Andrea

AU - Vokes, Tamara

AU - Khan, Aliya A

AU - Schwarz, Peter

AU - Cetani, Filomena

AU - Pagotto, Uberto

AU - Tsourdi, Elena

AU - Sikjaer, Tanja

AU - Pfeiffer, Kathryn M

AU - Brod, Meryl

AU - McLeod, Lori D

AU - Zhao, Carol

AU - Noori, Wahidullah

AU - Shu, Aimee D

AU - Smith, Alden R

PY - 2025/12/9

Y1 - 2025/12/9

N2 - CONTEXT: Individuals with hypoparathyroidism experience a range of physical and cognitive symptoms and reduced quality of life (QoL) despite management with conventional therapy (active vitamin D and calcium).OBJECTIVE: This analysis investigated the long-term impact of parathyroid hormone (PTH) replacement therapy with palopegteriparatide (YORVIPATH®) on symptoms, daily functioning, and well-being in adults with chronic hypoparathyroidism. Associations between patient characteristics and changes in patient-reported outcomes (PROs) were also analyzed.METHODS: PaTH Forward was a phase 2 clinical trial of palopegteriparatide with a 4-week randomized, double-blind, placebo-controlled period followed by an open-label extension period lasting through trial week 266. PRO measures were collected at baseline, weeks 4, 12, 26, 58, and annually thereafter through the end of the trial. The Hypoparathyroidism Patient Experience Scales (HPES) assess disease-specific symptoms and impacts on functioning and well-being. The Short-Form Health Survey (SF-36v2) measures general health-related QoL. Data were analyzed using descriptive statistics and Mixed Models for Repeated Measures (MMRM).RESULTS: Palopegteriparatide treatment demonstrated significant improvements from baseline in disease-specific symptoms and impacts on daily functioning and well-being at week 12, which were sustained through week 110. Mean changes in PROs met thresholds for clinically meaningful within-patient improvement. Significant improvements in general health-related QoL were also shown in SF-36v2 scores. Results were generally similar across demographics and patient characteristics.CONCLUSION: Through week 110 of the PaTH Forward trial, PTH replacement therapy with palopegteriparatide was associated with significant improvements in disease-specific symptoms and impacts on daily functioning and well-being, as well as general health-related QoL.

AB - CONTEXT: Individuals with hypoparathyroidism experience a range of physical and cognitive symptoms and reduced quality of life (QoL) despite management with conventional therapy (active vitamin D and calcium).OBJECTIVE: This analysis investigated the long-term impact of parathyroid hormone (PTH) replacement therapy with palopegteriparatide (YORVIPATH®) on symptoms, daily functioning, and well-being in adults with chronic hypoparathyroidism. Associations between patient characteristics and changes in patient-reported outcomes (PROs) were also analyzed.METHODS: PaTH Forward was a phase 2 clinical trial of palopegteriparatide with a 4-week randomized, double-blind, placebo-controlled period followed by an open-label extension period lasting through trial week 266. PRO measures were collected at baseline, weeks 4, 12, 26, 58, and annually thereafter through the end of the trial. The Hypoparathyroidism Patient Experience Scales (HPES) assess disease-specific symptoms and impacts on functioning and well-being. The Short-Form Health Survey (SF-36v2) measures general health-related QoL. Data were analyzed using descriptive statistics and Mixed Models for Repeated Measures (MMRM).RESULTS: Palopegteriparatide treatment demonstrated significant improvements from baseline in disease-specific symptoms and impacts on daily functioning and well-being at week 12, which were sustained through week 110. Mean changes in PROs met thresholds for clinically meaningful within-patient improvement. Significant improvements in general health-related QoL were also shown in SF-36v2 scores. Results were generally similar across demographics and patient characteristics.CONCLUSION: Through week 110 of the PaTH Forward trial, PTH replacement therapy with palopegteriparatide was associated with significant improvements in disease-specific symptoms and impacts on daily functioning and well-being, as well as general health-related QoL.

U2 - 10.1210/clinem/dgaf653

DO - 10.1210/clinem/dgaf653

M3 - Journal article

C2 - 41365829

SN - 0021-972X

JO - The Journal of clinical endocrinology and metabolism

JF - The Journal of clinical endocrinology and metabolism

ER -

Patient-Reported Outcomes in Palopegteriparatide-Treated Adults with Hypoparathyroidism: PaTH Forward Trial Extension

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