Allogeneic hematopoietic stem-cell transplantation (HSCT) is a potentially curative treatment option for selected individuals with sickle cell disease or thalassemia major. In practice, children are only candidates before they accumulate too much end organ damage to be transplant candidates. There have not and are not likely to be randomized studies but the NMDP (2008) lists hemoglobinopathies, including sickle cell disease (SCD) and thalassemia major, as diseases which are treatable by allogeneic HSCT.
The NHLBI (2007) notes that bone marrow transplantation can be a very effective treatment for sickle cell anemia, but because of its risks, only some individuals can or should have this procedure. The NHLBI says that HSCT for SCD should be considered for children who experience significant, noninfectious complications caused by vaso-occlusion. For severely affected children who have human leukocyte antigen- (HLA) identical sibling donors, families should be informed about benefits, risks and treatment alternatives. Regarding thalassemia, the NHLBI (2008) notes that stem cell transplantation is the only treatment that can cure thalassemia; it is most successful in children.
The published peer-reviewed scientific literature supports the safety and effectiveness of myeloablative allogeneic hematopoietic stem-cell transplantation (HSCT) for the treatment of selected children and young adults with sickle cell disease (SCD) and thalassemia major. There is expanding use of this treatment in selected patients.
However, haplodientical donrs are only now being studied for this procedure. Mismatched related (haploidentical) HCT with post-transplant cyclophosphamide (PTCY) allows expansion of the donor pool but is complicated by high rates of graft failure. Therei i an ongoing trial: Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMT CTN 1507), ClinicalTrials.gov Identifier: NCT03263559. This is a Phase II, single arm, multi-center trial, designed to estimate the efficacy and toxicity of haploidentical bone marrow transplantation (BMT) in patients with sickle cell disease (SCD). Based on their age and entry criteria patients are stratified into two groups: (1) children with SCD with strokes; and (2) adults with severe SCD.
Non-myeloablative transplantation can be perforemed without a specific SUN protocol and has been well reported. Saraf et al conlclded: ” These results underscore the successful use of a chemotherapy-free regimen (using alemtuzumab) in MRD HSCT for high-risk adult SCD patients and demonstrates a high cure rate, absence of GVHD or mortality, and improvement in QoL including the applicability of this regimen in ABO mismatched cases (NCT number 01499888).
Mohsen Al-Zahrani, Ahmed Alaskar, Moussab Damlaj, Khadega Abuelgasim, Giamal Gmati, Hind ABDIN Salama, Samer Ghazi, Osama Ali, Bader Alahmari and Ayman Hejaz, Non-Myeloablative Transplant in Severe Sickle Cell Disease Is Safe & Effective. Single Center Experience from Saudi ArabiaBlood 2017 130:5549;
Saraf SL et al, Nonmyeloablative Stem Cell Transplantation with Alemtuzumab/Low-Dose Irradiation to Cure and Improve the Quality of Life of Adults with Sickle Cell Disease.Biol Blood Marrow Transplant. 2016 Mar;22(3):441-8.
Navneet S. Majhail et al, Indications for Autologous and Allogeneic Hematopoietic Cell Transplantation: Guidelines from the American Society for Blood and Marrow Transplantation. Biology of Blood and Marrow Transplantation Volume 21, Issue 11, November 2015, Pages 1863–1869
Hsieh MM, Fitzhugh CD, Tisdale JF. Allogeneic hematopoietic stem cell transplantation for sickle cell disease: the time is now. Blood 2011; 118:1197.
Bernaudin F, Socie G, Kuentz M, Chevret S, Duvall M, Bertrand Y, et al. Long-term results of related myeloablative stem-cell transplantation to cure sickle cell disease. Blood. 2007 Oct 1;110(7):2749-56. Epub 2007 Jul 2.
Bhatia M, Walters MC. Hematopoietic stem cell transplantation for thalassemia and sickle cell disease: past, present and future. Bone Marrow Transplant. 2008 Jan;42(2):109-17.
Angelucci E, Matthes-Martin S, Baronciani D, et al. Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: Indications and management recommendations from an international expert panel. Haematol. 2014; 99(5): 811-820.
Pawlowska, Anna B. et al..HLA Haploidentical Stem Cell Transplant with Pretransplant Immunosuppression for Patients with Sickle Cell Disease.
Biology of Blood and Marrow Transplantation ,2014 Volume 24 , Issue 1 , 185 – 189