Bone marrow transplantation in sickle cell anaemia.

Abstract

Sickle celi anaemia is still responsible for severe crippling and death in young patients living in developing countries. Apart from prophylaxis and treatment of infections, no active treatment can be safely proposed in such areas of the world. Therefore a bone marrow transplantation was performed in 12 patients staying in Belgium and planning to return to Africa.Twelve patients, aged between 11 months and 23 years (median 4 years), underwent a HLA identical bone marrow transplantation. The conditioning regimen included oral busulphan for four consecutive days (4 mg/kg) followed by four days of intravenous cyclophosphamide (50 mg/kg). In 10 patients the engraftment was rapid and sustained. A further patient suffered transient red cell hypoplasia and another underwent a second bone marrow transplantation from the same donor at day 62 because of graft rejection. All patients are alive and well with a follow up ranging from 9-51 months (median 27 months). In all cases a complete cessation of vaso-occlusive episodes and haemolysis was observed as was a change in the haemoglobin pattern in accordance with the donor's electrophoretic pattern.

Transplantation of unrelated placental blood cells in children with high-risk sickle cell disease

“…25,26,[29][30][31] In contrast, the incidence of acute GVHD in children with high-risk SCD who received BMTs from HLA-identical sibs ranges from 18-38% but is mostly mild to moderate (grades I and II). 14,15,18,19 The incidence of extensive chronic GVHD in children undergoing UPBCT (9%) 31 is similar to that reported after BMT from HLA-identical sibs for high-risk SCD (3-8%). 13,14,15,18,19 The degree of HLA disparity (greater than one antigen difference between donor and recipient vs zero to one antigen difference) has been suggested as a risk factor for development of GVHD, 26 but other studies have not confirmed this observation.…”

“…14,15,18,19 The incidence of extensive chronic GVHD in children undergoing UPBCT (9%) 31 is similar to that reported after BMT from HLA-identical sibs for high-risk SCD (3-8%). 13,14,15,18,19 The degree of HLA disparity (greater than one antigen difference between donor and recipient vs zero to one antigen difference) has been suggested as a risk factor for development of GVHD, 26 but other studies have not confirmed this observation. 28,29,31 Aggressive approaches to treat or prevent acute GVHD may be needed in UPBCT for high-risk SCD.…”

“…29,31 Overall survival in children and young adults with high-risk SCD who underwent allogeneic BMT [13][14][15][16]18 or placental blood cell transplantation 21 from HLA-matched healthy sibs is between 90 and 100%.…”

“…[10][11][12] Transplantation of allogeneic hematopoietic cells (bone marrow or placental blood cells) from HLA-identical healthy sibs can establish normal erythropoiesis, abrogate symptoms and eliminate the need for chronic transfusions and chelation in children with high-risk SCD. More than 200 children and young adults have undergone bone marrow transplantation (BMT) from HLA-matched sibs [13][14][15][16][17][18][19] for complications of SCD that included CVAs, recurrent acute chest syndrome 20 or recurrent vasoocclusive crises. These studies have shown overall survival and event-free survival of approximately 90 and 80%, respectively.…”

Evolution of sickle cell disease from a life‐threatening disease of children to a chronic disease of adults: The last 40 years

“…Her leukemia was cured and she was converted to sickle cell trait [132]. In the following decade, several pilot studies of bone marrow transplantation for the treatment of young patients with symptomatic SCD demonstrated eradication of the underlying disease with low transplantation-related mortality [133][134][135][136]. In 1996, Walters et al reported the results of HLAidentical allogeneic transplantation in 22 individuals with SCD (all <16 years old), of which 20 survived and 16 had stable engraftment of donor cells [137].…”