Introduction: Sickle cell disease (SCD) is one of the most common hemoglobinopathies, with nearly 40000 children born annually to the tribal population of India. Hematopoietic stem cell transplantation is currently the only curative option available. Patients eligible for transplantation according to the ASH and other guidelines are those with multiple episodes of veno-occlusive crisis, despite disease-modifying treatment, recurrent episodes of acute chest syndrome, osteonecrosis, patients who experienced overt stroke, or abnormal transcranial Doppler ultrasound, requiring frequent admissions to the hospital. Although HSCT is the sole curative treatment, only a fraction of eligible patients receive it in resource-limited settings. To understand the requirements for bone marrow transplantation, we analyzed the number of eligible patients using clinical data.
Methods: A cross-sectional prospective research design was used that involved clinical examination and personal interviews with patients and caregivers, with the completion of questionnaires on clinical characteristics and treatment options. Two hundred fifty individuals with S ,invited to outreach clinics, were analyzed
Results: Among the 251 surveyed children, 122 (48.6%) were male and 129(51.4%) were female. Among them, 160 (63.7%) children had recurrent episodes of VOC in the past 1 year, despite 116 (72.5%) receiving regular hydroxyurea. Among them, 98 (62%) required multiple admissions for VOC. Episodes of fever were observed in 51 % of the patients. The requirement for admission to a healthcare facility was recorded in 57 % of the patients. Nearly 13 % of the patients required more than three transfusions in the preceding year.
Conclusions: There is an urgent need to organize SCD care pathways, as the whooping number of patients (64%) falls within the criteria of eligibility for HSCT, a curative option. The transplant community, along with healthcare providers and non-governmental organizations, should decide on a strategy to offer this important modality of curable alternatives.
Disclosures
No relevant conflicts of interest to declare.
