Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel

Single-centre experience of allogeneic haemopoietic stem cell transplant in paediatric patients in Cape Town, South Africa

“…This is in keeping with the morbidity described in an adult cohort from GSH [27] and is also below the 30% suggested by Azevedo et al [28] Successful Allo-HSCT can significantly improve quality of life for patients requiring regular transfusion. [9] It reduces the cumulative financial burden of blood products, chelation and treatment of iron overload-related morbidity, which place an enormous load on an already overburdened healthcare system. Current monthly cost estimates for a child include admission to hospital (ZAR1 900/day), filtered packed red cells (ZAR1 500/unit) and chelation (deferasirox ZAR2 300 -5 000/month).…”

“…[8] Haemoglobinopathies such as homozygous betathalassaemia and sickle cell disease (SCD) are cured with successful Allo-HSCT. [9] At Red Cross War Memorial Children's Hospital (RCWMCH) in Cape Town, South Africa, approximately four new patients are diagnosed annually with bone marrow failure syndromes. The number of patients with SCD who attend the haematology clinic has expanded to nearly 150.…”

Reduced-intensity conditioning and stem cell transplantation in infants with Diamond Blackfan anemia

“…However, successful results in terms of safety, tolerability and efficacy have been reported in other non-malignant diseases, such as thalassemia and metabolic disorders, with transplant related mortality as low as 5% in young low-risk children transplanted from an HLA-matched sibling donor. 10 One major concern is the conditioning regimenrelated acute toxicities and the toxic late effects of busulfan, mainly secondary malignancies and infertility. [12][13][14] In fact, the North American DBAR registry described two DBA patients who developed post-transplant neoplasms after conditioning with a total body irradiation (TBI)-containing regimen, and one patient after a busulfan-including regimen.…”

Traffic Light: prognosis-based eligibility for clinical trials of hematopoietic SCT in adults with sickle cell anemia

“…Because of limited data in adults with SCA and because of published reports of patient concerns regarding GVHD in SCA, [32][33][34] we have stratified MUD apart from MSD despite promising data in other conditions, as MUD transplants have been associated with increased chronic GVHD. 35 Further, unlike other Hb gene disorders such as beta-thalassemia, 10 there is limited real-world availability of MUDs in SCA. Published experience in adults with SCA to date primarily has been of MSD or haploidentical donor transplants utilizing reduced intensity preparatory regimens.…”

“…There is a consensus that HSCT via clinical trial should be offered to adult SCA subjects who have a poor prognosis despite maximal medical therapy, although the definition of 'poor prognosis' remains imprecise. [9][10][11] In 2011, investigators at the NIH published eligibility criteria that served as the basis for non-myeloablative MSD transplant in adults with SCA. 12 Subsequently, Krishnamurti 13 published more conservative criteria for the MSD and matched unrelated donor (MUD) trial: 'Bone Marrow Transplantation in Young Adults with Severe Sickle Cell Disease' (STRIDE, Table 1).…”