Hafsa Rais

Date of Award

10-25-2023

Degree Type

Thesis

Degree Name

Master of Health Policy and Management

First Advisor

Zahra Hoodbhoy

Second Advisor

Lena Jafri

Third Advisor

Aysha Habib Khan

Department

Community Health Sciences

Abstract

Background: Congenital Hypothyroidism is an endocrine disorder, identified on newborn screening (NBS) and requires long-term monitoring to evaluate the progress or outcome. Registry is a good tool for recording and monitoring clinical progress or patient outcome data. However, there is no standardized dataset parameters defined for establishing CHT registry. This project aimed to identify clinical parameters for establishing a CHT outcome registry s in Pakistan.
Methods: The operational definitions encompassed primary CHT identified through screening and clinical dataset parameters covering various aspects. For identification of clinical parameters, a Delphi survey involving newborn screening experts was done. The study utilized a purposive sampling approach, targeting at least 20 experts involved in screening (n=9) or managing (n=11) CHT patients. The survey covered demographic details, screening tests, diagnostic tests, treatment parameters, growth monitoring, and developmental aspects. A consensus was sought with a minimum agreement level of ≥75%. Parameters with agreement between 50 to 75% underwent a second Delphi survey for consensus building.
Results: The literature search identified 2688 papers, of those 23 articles fulfilling the criteria were included in final analysis (reporting a total of 51,728 CHT cases. Clinical parameters (n=41) for the CHT outcome assessment registry related to four areas i.e., newborn birth or family history, diagnostic information, treatment related and developmental monitoring parameters, were identified. The Delphi survey engaged 20 NBS experts (11 engaged in management and 9 in diagnosis of CHT patients) to select the clinical parameters for the final registry. In round 1 survey 66% (n=27) clinical parameters were included, 12% (n=5) were excluded while 22% (n=9) were shared again in second round of survey, based on the predefined criteria. In the second round of Delphi survey 55.5% (n=5/9) clinical parameters, including birth weight, delivery type, visual congenital conditions, serum FT4, and thyroid ultrasound/scintigraphy, tests to determine etiology of CHT were included while Renal and musculoskeletal congenital conditions, thyroid antibodies (Anti[1]TPO and TSHR antibodies) were excluded. The exclusion reasons ranged from perceived lack of relevance to CHT to the impracticality of certain parameters in early assessments.
Conclusion: The study addressed a gap in literature for establishing CHT registry, including the absence of standardized data sets. The registry framework can be applied for the outcome 5 monitoring of CHT patients and can be gradually scaled-up to include other institutes and form a national level database of CHT.

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1

Last Page

48

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