Bone Marrow Transplant for Sickle Cell Disease

No. Sickle cell disease is a gene defect — there are sickle cell carriers and sickle cell disease patients. Disease means both parents were carriers; a carrier has only one gene affected and can pass it to their children if their partner is also a carrier. Other than gene therapy, there is no treatment for asymptomatic carriers — no cure or treatment is needed, because they reach adulthood and live a normal life. Since there is no phenotypic expression of the genotypic disorder, no treatment is warranted, because every treatment carries a risk of morbidity and mortality.

We advise the youngest age should be more than two years — doing a transplant very early, at 6 months to 1 year after only one crisis episode, children cannot tolerate the required immunosuppression properly, though on an emergency basis it is sometimes done for leukaemia. For sickle cell disease and thalassemia, transplant should be offered after 2 years of age. There is no upper age cutoff — every patient has the right to live, whatever the outcome percentage; the decision to go for transplant depends on the patient and family, weighed against quality of life.

Race does not affect the outcome. African sickle cell patients present with more severe sickling compared to Indian sickle cell patients, but the transplant outcome is the same — leukaemia treated in Africa versus leukaemia treated in India has the same outcome. Transplant outcome is mainly affected by patient factors (disease stage and comorbidity), donor factors (full match, half match, or mismatch), and treatment factors (right conditioning, right immunosuppression, right supportive care). Get those right and outcomes across the globe are similar, whether the transplant is done in America, Tokyo, or India.

We encourage good nutrition — patients can eat whatever they like, there is no prohibition, other than avoiding street food. Any hygienic food eaten at home, with good nutrition and a good amount of protein, is good for transplant outcome — the same as for any ordinary healthy human being.

I will circulate full information from my lab on the prerequisites for an HLA sample — what temperature it should be kept at, how many hours it can take to transport, and how many ml of blood sample or a buccal swab is required, so the sample can safely reach India for testing. Laboratory networks such as Metropolis and Lancet already exist in Kenya, Ghana and Uganda, though coverage is patchier in countries such as Nigeria and the DRC.

It replaces the patient's defective haematopoietic stem cells with healthy donor stem cells. Medicines can modify what defective cells produce, but only transplant replaces them entirely, so after successful engraftment the patient's blood is produced entirely from donor cells, free of sickle cell disease.

Conditioning, where chemotherapy destroys the defective marrow and creates space for donor cells; stem cell infusion, where donor cells from bone marrow, peripheral blood, or cord blood are infused intravenously; and engraftment and recovery, with close monitoring for the first 30 to 100 days and follow-up for about five years.

Six months to one year, unlike kidney or liver transplant, which require lifelong immunosuppression.

No. Existing organ damage does not reverse after transplant, which is why early referral matters before irreversible damage accumulates.