Pseudohypoparathyroidism type 1a
Conditions
Interventions
Sponsors
Eligibility
Inclusion criteria
Inclusion criteria: 1) Age >= 3.5 years at the initiation of GH therapy; 2) Height at screening below -2 standard deviation score (SD) of the height of normal children of the same chronological age, gender, and ethnicity; or below the 3rd percentile (P3, -1.88 SD); 3) Epiphyses confirmed to be unclosed by bone age radiographs before treatment; 4) Children diagnosed with PHP1A through clinical phenotype and genetic testing; 5) Complete case records (including baseline data, GH treatment records of >= 1 year, and follow-up data).
Exclusion criteria
Exclusion criteria: 1) Retrospective cases with growth hormone (GH) treatment duration of less than 1 year; 2) Patients with diabetes mellitus, impaired liver or kidney function, epilepsy, intellectual disability, skeletal deformities, slipped capital femoral epiphysis, severe cardiopulmonary diseases, hematological system diseases, malignant tumors, systemic infections, immunodeficiency, or mental illnesses; 3) Patients allergic to GH or its protective agents; 4) Cases with missing key data (fewer than 3 bone age radiographs per year or missing data from 2 consecutive follow-ups); 5) Other conditions deemed unsuitable for inclusion in this study by the researchers.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| ?PAH;body composition; | — |
Secondary
| Measure | Time frame |
|---|---|
| ?HtSDSCA;?HtSDSBA;?BA/?CA;GV;IGF-1;BMI;Carbohydrate Metabolism; | — |
Countries
China
Contacts
Children's Hospital, Zhejiang University School of Medicine