The PARADIGHM (physicians advancing disease knowledge in hypoparathyroidism) registry for patients with chronic hypoparathyroidism: study protocol and interim baseline patient characteristics

Abstract Background The PARADIGHM registry of adult and pediatric patients with chronic hypoparathyroidism evaluates the long-term safety and effectiveness of treatment with recombinant human parathyroid hormone, rhPTH(1-84), and describes the clinical disease course under conditions of routine clin...

Descripción completa

Guardado en:

Detalles Bibliográficos
Autores principales:	Neil Gittoes, Lars Rejnmark, Steven W. Ing, Maria Luisa Brandi, Sigridur Björnsdottir, Stefanie Hahner, Lorenz C. Hofbauer, Pascal Houillier, Aliya A. Khan, Michael A. Levine, Michael Mannstadt, Dolores M. Shoback, Tamara J. Vokes, Pinggao Zhang, Claudio Marelli, John Germak, Bart L. Clarke
Formato:	article
Lenguaje:	EN
Publicado:	BMC 2021
Materias:	Chronic hypoparathyroidism parathyroid hormone, patient registry quality of life, rhPTH(1-84) symptoms Diseases of the endocrine glands. Clinical endocrinology RC648-665
Acceso en línea:	https://doaj.org/article/12c9c84405614bd69518853f175c18a9
Etiquetas:	Agregar Etiqueta Sin Etiquetas, Sea el primero en etiquetar este registro!

id	oai:doaj.org-article:12c9c84405614bd69518853f175c18a9
record_format	dspace
spelling	oai:doaj.org-article:12c9c84405614bd69518853f175c18a92021-11-21T12:02:16ZThe PARADIGHM (physicians advancing disease knowledge in hypoparathyroidism) registry for patients with chronic hypoparathyroidism: study protocol and interim baseline patient characteristics10.1186/s12902-021-00888-21472-6823https://doaj.org/article/12c9c84405614bd69518853f175c18a92021-11-01T00:00:00Zhttps://doi.org/10.1186/s12902-021-00888-2https://doaj.org/toc/1472-6823Abstract Background The PARADIGHM registry of adult and pediatric patients with chronic hypoparathyroidism evaluates the long-term safety and effectiveness of treatment with recombinant human parathyroid hormone, rhPTH(1-84), and describes the clinical disease course under conditions of routine clinical practice. In this first report, we detail the registry protocol and describe the baseline characteristics of two adult patient cohorts from an interim database analysis. One cohort after study entry were prescribed rhPTH(1-84), and the other cohort received conventional therapy of calcium and active vitamin D. Methods An observational study of patients with chronic hypoparathyroidism in North America and Europe, collecting data for ≥10 years per patient. Main outcome measures were baseline patient demographics, clinical characteristics, medications, and disease outcome variables of symptoms, biochemical parameters, and health assessments. Baseline is the enrollment assessment for all variables except biochemical measurements in patients treated with rhPTH(1-84); those measurements were the most recent value before the first rhPTH(1-84) dose. Exclusion criteria applied to the analysis of specified outcomes included pediatric patients, patients who initiated rhPTH(1-84) prior to enrollment, and those who received rhPTH(1-34). Clinically implausible biochemical outlier data were excluded. Results As of 30 June 2019, data of 737 patients were analyzed from 64 centers; 587 (80%) were women, mean ± SD age 49.1±16.45 years. At enrollment, symptoms reported for patients later prescribed rhPTH(1-84) (n=60) and those who received conventional therapy (n=571), respectively, included fatigue (51.7%, 40.1%), paresthesia (51.7%, 29.6%), muscle twitching (48.3%, 21.9%), and muscle cramping (41.7%, 33.8%). Mean serum total calcium, serum phosphate, creatinine, and estimated glomerular filtration rate were similar between cohorts. Health-related quality of life (HRQoL) 36-item Short Form Health Survey questionnaire scores for those later prescribed rhPTH(1-84) were generally lower than those for patients in the conventional therapy cohort. Conclusions At enrollment, based on symptoms and HRQoL, a greater percentage of patients subsequently prescribed rhPTH(1-84) appeared to have an increased burden of disease than those who received conventional therapy despite having normal biochemistry measurements. PARADIGHM will provide valuable real-world insights on the clinical course of hypoparathyroidism in patients treated with rhPTH(1-84) or conventional therapy in routine clinical practice. Trial registration EUPAS16927, NCT01922440Neil GittoesLars RejnmarkSteven W. IngMaria Luisa BrandiSigridur BjörnsdottirStefanie HahnerLorenz C. HofbauerPascal HouillierAliya A. KhanMichael A. LevineMichael MannstadtDolores M. ShobackTamara J. VokesPinggao ZhangClaudio MarelliJohn GermakBart L. ClarkeBMCarticleChronic hypoparathyroidismparathyroid hormone, patient registryquality of life, rhPTH(1-84)symptomsDiseases of the endocrine glands. Clinical endocrinologyRC648-665ENBMC Endocrine Disorders, Vol 21, Iss 1, Pp 1-12 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	Chronic hypoparathyroidism parathyroid hormone, patient registry quality of life, rhPTH(1-84) symptoms Diseases of the endocrine glands. Clinical endocrinology RC648-665
spellingShingle	Chronic hypoparathyroidism parathyroid hormone, patient registry quality of life, rhPTH(1-84) symptoms Diseases of the endocrine glands. Clinical endocrinology RC648-665 Neil Gittoes Lars Rejnmark Steven W. Ing Maria Luisa Brandi Sigridur Björnsdottir Stefanie Hahner Lorenz C. Hofbauer Pascal Houillier Aliya A. Khan Michael A. Levine Michael Mannstadt Dolores M. Shoback Tamara J. Vokes Pinggao Zhang Claudio Marelli John Germak Bart L. Clarke The PARADIGHM (physicians advancing disease knowledge in hypoparathyroidism) registry for patients with chronic hypoparathyroidism: study protocol and interim baseline patient characteristics
description	Abstract Background The PARADIGHM registry of adult and pediatric patients with chronic hypoparathyroidism evaluates the long-term safety and effectiveness of treatment with recombinant human parathyroid hormone, rhPTH(1-84), and describes the clinical disease course under conditions of routine clinical practice. In this first report, we detail the registry protocol and describe the baseline characteristics of two adult patient cohorts from an interim database analysis. One cohort after study entry were prescribed rhPTH(1-84), and the other cohort received conventional therapy of calcium and active vitamin D. Methods An observational study of patients with chronic hypoparathyroidism in North America and Europe, collecting data for ≥10 years per patient. Main outcome measures were baseline patient demographics, clinical characteristics, medications, and disease outcome variables of symptoms, biochemical parameters, and health assessments. Baseline is the enrollment assessment for all variables except biochemical measurements in patients treated with rhPTH(1-84); those measurements were the most recent value before the first rhPTH(1-84) dose. Exclusion criteria applied to the analysis of specified outcomes included pediatric patients, patients who initiated rhPTH(1-84) prior to enrollment, and those who received rhPTH(1-34). Clinically implausible biochemical outlier data were excluded. Results As of 30 June 2019, data of 737 patients were analyzed from 64 centers; 587 (80%) were women, mean ± SD age 49.1±16.45 years. At enrollment, symptoms reported for patients later prescribed rhPTH(1-84) (n=60) and those who received conventional therapy (n=571), respectively, included fatigue (51.7%, 40.1%), paresthesia (51.7%, 29.6%), muscle twitching (48.3%, 21.9%), and muscle cramping (41.7%, 33.8%). Mean serum total calcium, serum phosphate, creatinine, and estimated glomerular filtration rate were similar between cohorts. Health-related quality of life (HRQoL) 36-item Short Form Health Survey questionnaire scores for those later prescribed rhPTH(1-84) were generally lower than those for patients in the conventional therapy cohort. Conclusions At enrollment, based on symptoms and HRQoL, a greater percentage of patients subsequently prescribed rhPTH(1-84) appeared to have an increased burden of disease than those who received conventional therapy despite having normal biochemistry measurements. PARADIGHM will provide valuable real-world insights on the clinical course of hypoparathyroidism in patients treated with rhPTH(1-84) or conventional therapy in routine clinical practice. Trial registration EUPAS16927, NCT01922440
format	article
author	Neil Gittoes Lars Rejnmark Steven W. Ing Maria Luisa Brandi Sigridur Björnsdottir Stefanie Hahner Lorenz C. Hofbauer Pascal Houillier Aliya A. Khan Michael A. Levine Michael Mannstadt Dolores M. Shoback Tamara J. Vokes Pinggao Zhang Claudio Marelli John Germak Bart L. Clarke
author_facet	Neil Gittoes Lars Rejnmark Steven W. Ing Maria Luisa Brandi Sigridur Björnsdottir Stefanie Hahner Lorenz C. Hofbauer Pascal Houillier Aliya A. Khan Michael A. Levine Michael Mannstadt Dolores M. Shoback Tamara J. Vokes Pinggao Zhang Claudio Marelli John Germak Bart L. Clarke
author_sort	Neil Gittoes
title	The PARADIGHM (physicians advancing disease knowledge in hypoparathyroidism) registry for patients with chronic hypoparathyroidism: study protocol and interim baseline patient characteristics
title_short	The PARADIGHM (physicians advancing disease knowledge in hypoparathyroidism) registry for patients with chronic hypoparathyroidism: study protocol and interim baseline patient characteristics
title_full	The PARADIGHM (physicians advancing disease knowledge in hypoparathyroidism) registry for patients with chronic hypoparathyroidism: study protocol and interim baseline patient characteristics
title_fullStr	The PARADIGHM (physicians advancing disease knowledge in hypoparathyroidism) registry for patients with chronic hypoparathyroidism: study protocol and interim baseline patient characteristics
title_full_unstemmed	The PARADIGHM (physicians advancing disease knowledge in hypoparathyroidism) registry for patients with chronic hypoparathyroidism: study protocol and interim baseline patient characteristics
title_sort	paradighm (physicians advancing disease knowledge in hypoparathyroidism) registry for patients with chronic hypoparathyroidism: study protocol and interim baseline patient characteristics
publisher	BMC
publishDate	2021
url	https://doaj.org/article/12c9c84405614bd69518853f175c18a9
work_keys_str_mv	AT neilgittoes theparadighmphysiciansadvancingdiseaseknowledgeinhypoparathyroidismregistryforpatientswithchronichypoparathyroidismstudyprotocolandinterimbaselinepatientcharacteristics AT larsrejnmark theparadighmphysiciansadvancingdiseaseknowledgeinhypoparathyroidismregistryforpatientswithchronichypoparathyroidismstudyprotocolandinterimbaselinepatientcharacteristics AT stevenwing theparadighmphysiciansadvancingdiseaseknowledgeinhypoparathyroidismregistryforpatientswithchronichypoparathyroidismstudyprotocolandinterimbaselinepatientcharacteristics AT marialuisabrandi theparadighmphysiciansadvancingdiseaseknowledgeinhypoparathyroidismregistryforpatientswithchronichypoparathyroidismstudyprotocolandinterimbaselinepatientcharacteristics AT sigridurbjornsdottir theparadighmphysiciansadvancingdiseaseknowledgeinhypoparathyroidismregistryforpatientswithchronichypoparathyroidismstudyprotocolandinterimbaselinepatientcharacteristics AT stefaniehahner theparadighmphysiciansadvancingdiseaseknowledgeinhypoparathyroidismregistryforpatientswithchronichypoparathyroidismstudyprotocolandinterimbaselinepatientcharacteristics AT lorenzchofbauer theparadighmphysiciansadvancingdiseaseknowledgeinhypoparathyroidismregistryforpatientswithchronichypoparathyroidismstudyprotocolandinterimbaselinepatientcharacteristics AT pascalhouillier theparadighmphysiciansadvancingdiseaseknowledgeinhypoparathyroidismregistryforpatientswithchronichypoparathyroidismstudyprotocolandinterimbaselinepatientcharacteristics AT aliyaakhan theparadighmphysiciansadvancingdiseaseknowledgeinhypoparathyroidismregistryforpatientswithchronichypoparathyroidismstudyprotocolandinterimbaselinepatientcharacteristics AT michaelalevine theparadighmphysiciansadvancingdiseaseknowledgeinhypoparathyroidismregistryforpatientswithchronichypoparathyroidismstudyprotocolandinterimbaselinepatientcharacteristics AT michaelmannstadt theparadighmphysiciansadvancingdiseaseknowledgeinhypoparathyroidismregistryforpatientswithchronichypoparathyroidismstudyprotocolandinterimbaselinepatientcharacteristics AT doloresmshoback theparadighmphysiciansadvancingdiseaseknowledgeinhypoparathyroidismregistryforpatientswithchronichypoparathyroidismstudyprotocolandinterimbaselinepatientcharacteristics AT tamarajvokes theparadighmphysiciansadvancingdiseaseknowledgeinhypoparathyroidismregistryforpatientswithchronichypoparathyroidismstudyprotocolandinterimbaselinepatientcharacteristics AT pinggaozhang theparadighmphysiciansadvancingdiseaseknowledgeinhypoparathyroidismregistryforpatientswithchronichypoparathyroidismstudyprotocolandinterimbaselinepatientcharacteristics AT claudiomarelli theparadighmphysiciansadvancingdiseaseknowledgeinhypoparathyroidismregistryforpatientswithchronichypoparathyroidismstudyprotocolandinterimbaselinepatientcharacteristics AT johngermak theparadighmphysiciansadvancingdiseaseknowledgeinhypoparathyroidismregistryforpatientswithchronichypoparathyroidismstudyprotocolandinterimbaselinepatientcharacteristics AT bartlclarke theparadighmphysiciansadvancingdiseaseknowledgeinhypoparathyroidismregistryforpatientswithchronichypoparathyroidismstudyprotocolandinterimbaselinepatientcharacteristics AT neilgittoes paradighmphysiciansadvancingdiseaseknowledgeinhypoparathyroidismregistryforpatientswithchronichypoparathyroidismstudyprotocolandinterimbaselinepatientcharacteristics AT larsrejnmark paradighmphysiciansadvancingdiseaseknowledgeinhypoparathyroidismregistryforpatientswithchronichypoparathyroidismstudyprotocolandinterimbaselinepatientcharacteristics AT stevenwing paradighmphysiciansadvancingdiseaseknowledgeinhypoparathyroidismregistryforpatientswithchronichypoparathyroidismstudyprotocolandinterimbaselinepatientcharacteristics AT marialuisabrandi paradighmphysiciansadvancingdiseaseknowledgeinhypoparathyroidismregistryforpatientswithchronichypoparathyroidismstudyprotocolandinterimbaselinepatientcharacteristics AT sigridurbjornsdottir paradighmphysiciansadvancingdiseaseknowledgeinhypoparathyroidismregistryforpatientswithchronichypoparathyroidismstudyprotocolandinterimbaselinepatientcharacteristics AT stefaniehahner paradighmphysiciansadvancingdiseaseknowledgeinhypoparathyroidismregistryforpatientswithchronichypoparathyroidismstudyprotocolandinterimbaselinepatientcharacteristics AT lorenzchofbauer paradighmphysiciansadvancingdiseaseknowledgeinhypoparathyroidismregistryforpatientswithchronichypoparathyroidismstudyprotocolandinterimbaselinepatientcharacteristics AT pascalhouillier paradighmphysiciansadvancingdiseaseknowledgeinhypoparathyroidismregistryforpatientswithchronichypoparathyroidismstudyprotocolandinterimbaselinepatientcharacteristics AT aliyaakhan paradighmphysiciansadvancingdiseaseknowledgeinhypoparathyroidismregistryforpatientswithchronichypoparathyroidismstudyprotocolandinterimbaselinepatientcharacteristics AT michaelalevine paradighmphysiciansadvancingdiseaseknowledgeinhypoparathyroidismregistryforpatientswithchronichypoparathyroidismstudyprotocolandinterimbaselinepatientcharacteristics AT michaelmannstadt paradighmphysiciansadvancingdiseaseknowledgeinhypoparathyroidismregistryforpatientswithchronichypoparathyroidismstudyprotocolandinterimbaselinepatientcharacteristics AT doloresmshoback paradighmphysiciansadvancingdiseaseknowledgeinhypoparathyroidismregistryforpatientswithchronichypoparathyroidismstudyprotocolandinterimbaselinepatientcharacteristics AT tamarajvokes paradighmphysiciansadvancingdiseaseknowledgeinhypoparathyroidismregistryforpatientswithchronichypoparathyroidismstudyprotocolandinterimbaselinepatientcharacteristics AT pinggaozhang paradighmphysiciansadvancingdiseaseknowledgeinhypoparathyroidismregistryforpatientswithchronichypoparathyroidismstudyprotocolandinterimbaselinepatientcharacteristics AT claudiomarelli paradighmphysiciansadvancingdiseaseknowledgeinhypoparathyroidismregistryforpatientswithchronichypoparathyroidismstudyprotocolandinterimbaselinepatientcharacteristics AT johngermak paradighmphysiciansadvancingdiseaseknowledgeinhypoparathyroidismregistryforpatientswithchronichypoparathyroidismstudyprotocolandinterimbaselinepatientcharacteristics AT bartlclarke paradighmphysiciansadvancingdiseaseknowledgeinhypoparathyroidismregistryforpatientswithchronichypoparathyroidismstudyprotocolandinterimbaselinepatientcharacteristics
_version_	1718419286579478528

The PARADIGHM (physicians advancing disease knowledge in hypoparathyroidism) registry for patients with chronic hypoparathyroidism: study protocol and interim baseline patient characteristics

Ejemplares similares