Multicenter Registry Study of Chronic Hypoparathyroidism in Chinese Adults (PaTHwayR)

The Status of Diagnosis and Treatment of Chronic Hypoparathyroidism in Chinese Adults: a Multicenter Registry Study

This multicenter registry study aims to understand the status of diagnosis and treatment, quality of life, medical costs and unmet medical needs of adult patients with chronic hypoparathyroidism in China.

Study Overview

• Status: Completed
• Conditions: Chronic Hypoparathyroidism
• Intervention / Treatment: Other: No intervention would be taken

Detailed Description

Hypoparathyroidism (HP) is a rare (orphan) endocrine disease with low calcium and inappropriately low circulating parathyroid hormone levels, most often in adults secondary to thyroid surgery. The present study contains four parts.

The first part is a multicenter registry study. According to our predefined search criteria determined through pilot study, all patients admitted between June 1, 2020 and May 31, 2021 will be searched from the hospital information system and patients diagnosed with HP and those with suspected HP will be retrieved. The diagnosis of all or a random sample of these patients will be reviewed by endocrinologists through medical records. Patients with a confirmed diagnosis of chronic HP will served as the study population of the first part of the study.

The second part is a multicenter hospital-based survey, to investigate the missed diagnosis, quality of life, medical costs, medical experience and unmet medical needs in adult patients with chronic HP. All local patients with definite chronic HP and those with suspected HP will be invited to participate in the hospital on-site investigation. Among them, suspected HP patients need to first undergo necessary examinations to confirm the diagnosis of HP.

The third part is the verification of the reliability and validity of two quality of life scales, the Short-Form Six-Dimension version 2 (SF-6Dv2) scale and Hypoparathyroidism Patient Experience Scale (HPES). The first 100 patients with chronic HP who participate in the on-site investigation in the Peking Union Medical College Hospital will be invited.

The fourth part is a survey on physicians about the unmet need of chronic HP. All endocrinologists from each site will be invited to take part in the survey.

• Study Type: Observational
• Enrollment (Actual): 524

Contacts and Locations

• Study Contact: Name: Huijuan Li; Phone Number: 612 82805564; Email: pucri_lihj@bjmu.edu.cn

Study Locations

• China
  • Beijing
    • Beijing, Beijing, China
      • Peking Union Medical College Hospital
  • Dongbei
    • Shenyang, Dongbei, China
      • The First Hospital of China Medical University
  • Guangdong
    • Guangzhou, Guangdong, China
      • The First Affiliated Hospital , Sun Yat-sen University
  • Hunan
    • Changsha, Hunan, China
      • The Second Xiangya Hospital Of Central South University
  • Shanghai
    • Shanghai, Shanghai, China
      • Shanghai Sixth People's Hospital
  • Sichuan
    • Chengdu, Sichuan, China
      • West China Hospital of Sichuan University
  • Xinjiang
    • Urumqi, Xinjiang, China
      • The first affiliated hospital of Xinjiang medical university

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Part 1. All patients admitted between June 1, 2020 and May 31, 2021 will be searched in the information system according to predefined search criteria. Patients diagnosed with HP and those with suspected HP will be retrieved. Patients with definite diagnosis of chronic HP will serve as the study population of this part.

Part 2. All local patients with a definite diagnosis of chronic HP and those with suspected HP will be invited to take part in the on-site survey.

Part 3. The first 100 patients with definite chronic HP who participate in the on-site survey in the Peking Union Medical College Hospital will be invited to participate.

Part 4. All endocrinologists at each center will be invited to participate.

Description

This study consists of four parts, the eligibility criteria of each part is described below:

Part 1:

Search criteria of chronic HP:

Inclusion Criteria:

• Existing diagnosis of HP

Exclusion Criteria:

• Age<18 at the time of screening

Search criteria of suspected HP:

Inclusion Criteria:

• Hypocalcemia plus neck surgery
• Hypocalcemia plus convulsions/tetany/epilepsy
• Hypocalcemia plus PTH<upper limit, and no hypoalbuminemia
• Hypocalcemia without any of the following conditions: neck surgery, convulsions/tetany/epilepsy, PTH testing, hypoalbuminemia, critical illness and vitamin D deficiency-related diseases.

Exclusion Criteria:

• Age<18 at the time of screening

Diagnosis of chronic HP:

Inclusion Criteria:

• Hypocalcemia (albumin adjusted total calcium < lower limit) with inappropriate reduction of serum PTH level (PTH < upper limit).
• The course of hypoparathyroidism should be more than 6 months.

Part 2

Inclusion Criteria:

• Patients with chronic HP confirmed through medical records review
• Patients with suspected HP confirmed through medical records review
• Obtain written informed consent

Exclusion Criteria:

• Non-local residents

Part 3

Inclusion Criteria:

• Patients with chronic HP
• Participating in the on-site survey in the Peking Union Medical College Hospital

Part 4

Inclusion criteria:

• Endocrinologists involved in HP care from 7 sites
• Obtain written informed consent

Study Plan

How is the study designed?

Design Details

• Observational Models: Other
• Time Perspectives: Cross-Sectional

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The proportion of patients with different classification of hypoparathyroidism (HP)	HP is classified into postsurgical and non-postsurgical HP.	0 day （cross-sectional）
The proportion of patients with different symptoms of HP	The most common symptoms of HP include tetany, seizures, laryngospasm, numbness, blood in the urine, renal colic and vision loss.	0 day （cross-sectional）
The proportion of patients with positive Chvostek sign	Including the situation at the time of diagnosis and the current situation	0 day （cross-sectional）
The proportion of patients with positive Trousseau's sign	Including the situation at the time of diagnosis and the current situation	0 day （cross-sectional）
The proportion of patients with abnormal corrected calcium	Including the situation at the time of diagnosis and the current situation	0 day （cross-sectional）
The proportion of patients with abnormal parathyroid hormone (PTH)	The situation at the time of diagnosis	0 day （cross-sectional）
The proportion of patients with abnormal 24-hour urine calcium	Including the situation at the time of diagnosis and the current situation	0 day （cross-sectional）
The proportion of patients with different first-visit reasons	First-visit reason was excerpted from previous medical records	0 day （cross-sectional）
The proportion of patients with different first-visit departments	First-visit department was excerpted from previous medical records	0 day （cross-sectional）

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The prevalence of chronic HP in adults visiting top hospitals in China	Number of patients diagnosed with chronic HP / Total number of outpatients and inpatients during the year	0 day （cross-sectional）
The proportion of patients with chronic HP receiving treatment	Number of patients receiving any kind of HP treatment/ Total number of patients diagnosed with chronic HP	0 day （cross-sectional）
The proportion of patients with chronic HP meeting treatment targets	HP treatment targets defined as normal blood calcium levels or/+ normal urine calcium level	0 day （cross-sectional）
The proportion of patients with HP complications	Number of patients with complications / Total number of patients with chronic HP	0 day （cross-sectional）
The proportion of patients with missed diagnosis of chronic HP	Patients with missed diagnosis of chronic HP / Total number of patients with chronic HP	0 day （cross-sectional）
The proportion of patients with previous experience of misdiagnosis of HP	Misdiagnosis experience of HP is defined as patients with chronic HP were previously diagnosed with other diseases due to related symptoms or hypocalcemia.	0 day （cross-sectional）
Median medical costs	Medical costs contains of direct medical costs and indirect medical costs	0 day （cross-sectional）
The mean score of Short-Form Six-Dimension version 2 (SF-6Dv2)	The SF-6Dv2 score ranges from 0.29 to 1, with higher scores indicating better performance.	0 day （cross-sectional）
The mean score of Hypoparathyroidism Patient Experience Scale (HPES)	The HPES score ranges from 0 to 100, with lower scores indicating better performance.	0 day （cross-sectional）
The proportion of patients with different unmet medical needs	Unmet medical needs for HP treatment and quality of life by patients and physicians	0 day （cross-sectional）

Collaborators and Investigators

• Sponsor: Peking University
• Collaborators: Peking Union Medical College Hospital; First Affiliated Hospital, Sun Yat-Sen University; West China Hospital; Central South University; First Hospital of China Medical University; First Affiliated Hospital of Xinjiang Medical University; Shanghai 6th People's Hospital
• Investigators: Principal Investigator: Weibo Xia, Peking Union Medical College Hospital

Study record dates

Study Major Dates

• Study Start (Actual): January 10, 2022
• Primary Completion (Actual): December 22, 2022
• Study Completion (Actual): December 22, 2022

Study Registration Dates

• First Submitted: December 24, 2021
• First Submitted That Met QC Criteria: January 17, 2022
• First Posted (Actual): January 28, 2022

Study Record Updates

• Last Update Posted (Actual): May 24, 2023
• Last Update Submitted That Met QC Criteria: May 22, 2023
• Last Verified: May 1, 2023

More Information

Terms related to this study

• Keywords: Registry study; Chronic hypoparathyroidism
• Additional Relevant MeSH Terms: Endocrine System Diseases; Parathyroid Diseases; Hypoparathyroidism
• Other Study ID Numbers: 2021-PaTHwayR

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No