# Clinico-epidemiological Profile of Transfusion-dependent Thalassemia Patients in a Tertiary Care Children’s Hospital in Nepal: An Observational Study

## TL;DR

This study examines the clinical and epidemiological characteristics of children with transfusion-dependent thalassemia in Nepal, highlighting treatment practices and complications.

## Contribution

The study provides a detailed clinico-epidemiological profile of transfusion-dependent thalassemia in a Nepalese pediatric hospital, contributing to a data-scarce region.

## Key findings

- β-thalassemia major was the most common type, affecting 83.42% of patients.
- Over half of patients had iron overload, with 49.19% having serum ferritin levels above 2500 ng/mL.
- Only 19.87% of patients receiving chelation therapy had good compliance.

## Abstract

Transfusion-dependent thalassemia is a major public health concern in Nepal, with limited access to comprehensive care and paucity of national data. The main objectives of this study were to explore clinico-epidemiological profile, management practices and complications among pediatric transfusion-dependent thalassemia patients at a tertiary pediatric hospital in Nepal.

This is a retrospective observational study conducted at the Thalassemia Day Care Unit, Kanti Children’s Hospital, Kathmandu. Transfusion-dependent thalassemia cases registered from January 2020 to December 2024, aged less than 15 years were included. Data on demographics, clinical features, transfusion and chelation profiles, complications, and nutritional status were acquired from hospital registry and analyzed using descriptive statistics.

Out of 187 patients, 121 (64.71%) were males and 156 (83.42%) had β-thalassemia major. Mean age at last follow-up was 7.52±3.68 years. 83 (44.38%) were Janajatis, particularly Tharus 40 (21.39%). Median age at diagnosis was 10 months (IQR 6-22 months), with mean pre-transfusion hemoglobin level 8.86 g/dL, and average transfusion period 2.64±0.83 weeks. Among 167 (89.30%) patients receiving chelation, 37 (19.87%) had good compliance. Endocrinopathies, hepatic dysfunction and cardiac complications were observed in 104 (55.62%), 65 (34.76%) and 2 (1.07%) patients respectively. Serum ferritin levels were above 2500 ng/mL in 92 (49.19%) patients. Regular follow up was done by 56 (29.95%) patients.

β-thalassemia major was the most common type of transfusion dependent thalassemia in our study. There was male predominance, and over half of the patients had iron overload and some form of complications of thalassemia. Regular follow up and compliance to chelation therapy has to be focused for better care of transfusion dependent thalassemia patients.