A systematic review on hydroxyurea therapy for sickle cell disease in India

Background & objectives: Sickle cell disease (SCD) constitutes frequently inherited haemoglobin disorders and poses a significant health burden in India. Hydroxyurea (HU), the most commonly used drug, has shown promising results in the clinical management of SCD. The present systematic review was undertaken to assess the efficacy and toxicity of HU in Indian sickle cell patients. Methods: A systematic review of studies on HU therapy was conducted to identify the application of HU and its outcome(s) across India. PubMed, Scopus and Cochrane Library was used as data sources for various studies on the efficacy and toxicity of HU therapy for treatment for SCD in India published between January 2001 and October 2021. Two authors independently extracted the data on study design, patient characteristics and therapeutic outcomes of HU in order to determine the study quality of the present review. Results: Overall, 14 studies were included for a systematic analysis. Of these 11 were prospective, two cross-sectional and one double-blind randomized controlled trial. Low-dose HU (10 mg/kg/day) was found to reduce the rates of vaso-occlusive crisis and hospitalization as well as decreased the requirement of blood transfusion in SCD patients. The foetal haemoglobin (HbF) level was recorded in 13 (80%) studies all of whom reported an elevation in the HbF levels, with a mean increase in per cent HbF from 15.8 to 21.4 per cent across studies. The common adverse events were reversible, mild-to-moderate cytopenia and anaemia. Interpretation & conclusions: The findings of the present review suggest that there is still insufficient information presently to determine the long-term or major adverse effects on organ damage, fertility as well as pregnancy on the use of HU therapy for SCD. Long-term multi-centric studies are thus required to address these problems.

Guidelines for screening, diagnosis and management of hemoglobinopathies.

The b‑thalassemias and sickle cell disorders are a major health burden in India. Diagnosis and management of these disorders both in adults and in newborns using appropriate approaches and uniform technology are important in different regions of a vast and diverse country as India. In view of a National Thalassemia Control Program to be launched soon, a need was felt for guidelines on whom to screen, cost‑effective technologies that are to be used as well as for establishing prenatal diagnosis programs in regional centers. Newborn screening for sickle cell disorders is in its infancy in India and uniform approaches need to be followed. Also, included are guidelines for monitoring and managing patients who are now growing older and need comprehensive care as well as management of complications of the disease.

Morbidity pattern in hospitalized under five children with sickle cell disease.

Background & objectives: Children with sickle cell disease require more frequent hospital care and younger children (<5 yr of age) are more vulnerable to mortality. There are limited data on the events leading to hospitalizations and death in younger children with sickle cell disease from India. This study was, therefore, undertaken to evaluate the morbidity pattern in hospitalized under five children with sickle cell disease in a tertiary care hospital in Maharashtra, India. Methods: This was a prospective observational study carried out from July 2007 to June 2009. Hospitalized children below five years of age with sickle cell disease were enrolled for the study and evaluated for morbid event/s leading to hospitalization. Haematological indices were noted at baseline (most recent past when patient was not acutely sick) and at the time of hospitalization. Results: Eighty five children with sickle cell disease were hospitalized during the study period. Hospitalization with acute febrile illness (31%) was the most common morbid event followed by severe anaemia (30%) and acute painful events (20%). Majority (62%) of the events occurred between August and October. Forty five patients had foetal haemoglobin (HbF) more than 20 per cent (26.80±4.81%) and morbidity was significantly less in these patients. Interpretation & conclusion: Acute febrile illness was the most common morbid event followed by severe anaemia and acute painful event hospitalized children with sickle cell disease. There was significant seasonal variation with maximum events occurring in the monsoon season.

Sickle Cell Anemia from Central India: A Retrospective Analysis.

Although sickle cell anemia in India is believed to have a mild clinical presentation, few studies report severe disease in many patients from central India. Hence, we have retrospectively studied 316 children with SCA who were followed up for a period of 5.8±5.7 years. There were 55.4 blood transfusions, 43.3 episodes of vaso-occlusive crises requiring hospitalization, and 108.9 hospitalizations per 100 person years. Ninety six (30%) patients had severe disease whereas 74 patients also fulfilled the criteria for hydroxyurea therapy. Significant proportion of children with sickle cell anemia from central India present with severe clinical presentation and require regular medical attention.