Stem cell transplantation in pediatrics

Abstract

Pediatric diseases are unique in that there is a preponderance of genetic and metabolic conditions which afflict this age group. There are agressive malignancies not amenable to or which have failed conventional therapy. Stem cell therapy technology has evolved to make treatment possible for these difficult disorders. Stem cell treatment can be broadly divided into two groups, Hematopoietic stem transplantation (HSCT) and Stem cell therapy. Bone marrow- or peripheral blood-derived allogeneic hematopoietic stem cell transplantation from an HLA-identical sibling or matched unrelated donor can cure children with thalassemia major, severe aplastic anemia and leukemias. There are several centers which perform HSCT in India, they have been performed for both malignant and non malignant diseases. Post transplant complications occur in both the acute setting with acute graft versus host disease, sinusoidal obstructive syndrome and infections causing early mortality and morbidity. Late complications include chronic graft versus host disease and infections as well as the sequelae of treatment. Advancements in understanding transplant biology and the newer immunosuppressive drugs, hale helped to reduce and treat graft versus host disease, routine prophylaxis, better early diagnostic markers for viral infections and immunizations have helped in prompt identification, treatment and prevention of infections. Better chimerism techniques have enhanced post transplant monitoring for early identification of relapse and rejection so that intervention can be delivered. For diseases like thalassemia major and aplastic anemia if transplants are planned, the patient and donor workup needs to be performed early as the transplant outcome is better prior to receiving multiple transfusions. Stem cell therapy is the forefront of regenerative medicine and has been utilized for degenerative disorders. Newer applications of stem cell therapy in congenital and developmental pediatric diseases holds promise for many incurable disorders.