Early September 2016, on a late evening I received a call from the E.R (Emergency Room) that a young 18 year old boy’s family was seeking an emergency opinion from a hematologist for their son.
He was found to have severe pancytopenia [severe reduction of all three cell lines –Hemoglobin (Hb), white blood cells (WBCs) and platelets] noted on a blood count (CBC) done for progressive fatigue over a month, and new onset rashes all over his body since a day.
His evaluation revealed a related rare disorder of the bone marrow, called severe aplastic anemia (SAA), that occurs due to near complete dysfunction of the entire hematopoietic functioning(involving Hb, WBC and platelets).
If left untreated, such patients rapidly detioriate and die either due to the severe thrombocytopenia (low platelets) related massive bleeding, or severe opportunistic infections (fungal, bacterial or viral) due to their extreme low blood counts.
In addition, these patients remain transfusion dependent (requiring blood and platelet transfusions almost every 2 weeks) till any definitive therapy. Fortunately, none of that happened and he lives today, healthy and upbeat as any young teenager would, though still under a close supervision. He underwent a procedure called as a bone marrow transplant (BMT), with his younger sibling brother being the stem cell donor (…)
Bone marrow transplants (BMT) are complicated procedures that involve replacing the entire hematopoietic(blood) system in an affected person through guided chemotherapy and subsequently, substituting it with a healthy donor hematopoietic stem cells (HSC). It is these donor HSCs, which eventually replenishes the patients hematopoietic system over time, and gives rise to all living blood cells like white blood cells (WBC), red blood cells(RBC) and platelets, giving a new lease of life to the patient and his/her family.
Several disorders benefit from such procedures. In addition to the described case above of a classical patient with aplastic anemia, BMTs are indicated in several other congenital bone marrow failure syndromes, hereditary diseases caused by faulty hemoglobin synthesis like thalassemia’s, sickle cell anemias, immunodeficiency disorders with an impaired ability of their immune system to fight infections, life threatening blood cancers like multiple myelomas, leukemia and several other types of advanced tumors like lymphomas, neuroblastomas, etc.
Approximately 1500 BMTs were recorded from 11 centers in a major publication from CMC Vellore, in Aug 2008. Since then many more centers have been developed and an estimated 8,000 more patients have undergone hematopoietic stem cell transplants (HSCT) in India. These are average numbers, as many centers do not participate in reporting and registering their transplant data (…)
Autologous HSCT is done by collecting the patient’s own HSC, before giving them high dose chemotherapy, and then re-infusing these preserved HSC back to the patients after chemotherapy. In certain diseases, only stem cells from a normal healthy donor can be given to the patient to replace the damaged or the diseased bone marrow. This is called an allogeneic (donor other than the patient himself, i.e. either a sibling donor or an unrelated donor searched through stem cell registries)hematopoietic stem cell transplant (HSCT) (…)