Real-world HbF status in sickle cell disease from an endemic zone.
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 serious discussion
and could be of great use in low resource countries.