Recently, ICMR raised the issue of jointly developing a low-dose or pediatric oral form of hydroxyurea for treating sickle cell disease in India.

About Hydroxyurea:

  • Hydroxyurea is an effective treatment for sickle cell anaemia (SCA) due to its multifaceted therapeutic benefits. 
  • As a myelosuppressive agent, it reduces the frequency of painful episodes associated with SCA by increasing the levels of fetal hemoglobin (HbF) and overall hemoglobin. 
  • By promoting the production of larger and rounder red blood cells, it enhances their flexibility and thus reduces the likelihood of them assuming a sickle shape.

About Sickle Cell Disease: 

Sickle cell disease impacts the hemoglobin in red blood cells. It changes the shape of red blood cells, which are responsible for carrying oxygen throughout the body.

  • In sickle cell disease, some cells become shaped like sickles or crescent moons. 
  • These altered cells are rigid and sticky, which can impede the flow of blood, causing blockages or slow circulation.

Cure: Bone marrow transplant is effective only for patients under 16, and the gene-editing cure using CRISPR and gene therapy is currently accessible only in the US.

Current Challenges in Hydroxyurea Treatment for pediatric patients:

  • Pharmaceutical companies in India primarily offer hydroxyurea in high-dosage forms of capsules and tablets (500mg and 200mg), making it challenging to administer appropriate doses for pediatric patients (Children). 
  • In children over two years old, the prescribed dosage ranges from 10 to 15 milligrams per kilogram of body weight. However, titrating this dosage accurately is challenging and currently involves using a fraction of broken capsules.
  • The lack of a suspension form of Hydroxyurea poses difficulties in determining accurate dosage according to body weight.

Need for Pediatric Formulation:

  • In South Asia, India bears the highest burden of sickle cell disease (SCD), with a population of over 20 million individuals affected by the condition.
  • With the launch of the National Mission to eliminate SCD by 2047, there’s a pressing need for a pediatric formulation of hydroxyurea. 

Importance of Accurate Dosage:

  • Healthcare providers typically begin hydroxyurea therapy only for symptomatic children due to both the limited availability of pediatric doses and concerns regarding potential toxicity due to lack of suitable dosage.
  • A pediatric formulation would enable precise dosing, reducing the risk of side effects and improving the effectiveness of the treatment.

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