Real-World HbF Status in Sickle Cell Disease from an Endemic Zone

Author(s): Dinesh Pendharkar*, Garima Nirmal, Neha Gupta, Abhishek Raj

Introduction: Sickle cell disease is debilitating hereditary disorder affecting large tribal population in certain parts of India. Complications of sickle cell disease are veno-occlusive crisis, gall stones, leg ulcers, stroke, anemia requiring transfusions adding to the decreasing quality of life. Usage of hydroxyurea increase value of  HbF and thereby  decreases the complications. To assess the real world scenario of SCD patients, particularly, there levels of HbF, a random clinical examination with blood sampling was  initiated and compared to baseline available data.  

Methods: Confirmed cases of sickle cell disease attended special clinical camps. Patients of all age group were invited to attend the camp. To assess the real-world scenario of SCD patients, particularly the levels of HbF, a questionnaire and a random clinical examination with blood sampling was initiated and compared to baseline available data.

Results: One hundred fourteen patients attended the camp. There were 68 males and 46 female patients with a median age group of 19 years (2-70 years). HU was prescribed on average considering age of the patient and average weight. 104 children were taking hydroxyurea. Exact dose calculations were not used. The dose averages between 10 mg/kg to15 mg/kg. As there was only one formulation available, the required daily dosing was changed to fixed dose scheduling There was significant change in the levels of HbF in the patients on hydroxyurea in all age groups using fixed dose combination.

Conclusions: The achievement of desired HbF target levels using “real-world” scenario, compels us to think about HU strategies. The practice of using fixed dose schedules, in real-time clinical practice, with minimal follow up deserves a s