A survey on patient perception of reduced-intensity transplantation in adults with sickle cell disease

Abstract

The development of reduced-intensity conditioning (RIC) and the success of BMT for paediatric sickle cell disease (SCD) have raised the possibility of revisiting this prospect in adults as well. In a chronic debilitating disorder managed with supportive therapy, the patients' perception is critical in the advancement of any potential curative therapy. To explore this aspect, we undertook a questionnaire-based survey on 30 adults with SCD. Sixty two per cent of the patients were ready to accept a transplant-related mortality (TRM) 410%; 30% of them a TRM 430%. A risk of graft failure (GF) 410% was acceptable to 64%, with a risk 430% acceptable to 41%. Infertility was acceptable to only 50%. Chronic graftversus-host disease (GVHD) was unacceptable to the majority (80%). Seventy six per cent% of patients had a full sibling and 60% were willing to participate in a clinical trial of RIC transplantation. This survey suggests that the majority of adults with SCD might be willing to consider a curative option such as RIC transplantation even with a high TRM or GF. The major concerns relate to chronic GVHD and infertility. There is an urgent need to explore RIC transplants in SCD patients within the framework of a clinical trial, considering patient perception regarding cure and complications.

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

“…94 With the use of ATG, cyclosporine A and MTX an incidence of over grade II aGvHD between 11% and 44% and an incidence of cGvHD between 6% and 44% has been reported. 34,72,74 Two small series have been published on the combination of RIC and alemtuzumab and no aGvHD or cGVHD was reported.…”

Hematopoietic Stem Cell Transplantation for Sickle Cell Disease: Current Practice and Emerging Trends

“…In 2 adult surveys, 63% of adult SCD patients were willing to accept a mortality risk of Ͼ 10% and 60% were willing to accept a RIT transplantation trial, but only 20% would accept chronic GVHD and only 50% would accept infertility. 26,27 Donor sources determine ability to offer HSCT Fewer than 14% of SCD patients have MSDs. Patients with sibling donors can consider transplantation early in the disease process due to good outcomes.…”

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

“…At present, we stratify haploidentical transplants more stringently than MSD owing to the high risk of graft loss. 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.…”