title: 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) >10%; 30% of them a TRM >30%. A risk of graft failure (GF) >10% was acceptable to 64%, with a risk >30% acceptable to 41%. Infertility was acceptable to only 50%. Chronic graft-versus-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.
authors.affiliation: Department of Haematology and Bone Marrow Transplantation, St George's Hospital, London, UK. suparno@doctors.org.uk
authors.forename: S
authors.initials: S
authors.lastname: Chakrabarti
authors.forename: D
authors.initials: D
authors.lastname: Bareford
mesh_terms: D000293:Adolescent; D000328:Adult; D000755:Anemia, Sickle Cell / Q000523:psychology* / Q000628:therapy*; D016026:Bone Marrow Transplantation / Q000379:methods* / Q000401:mortality / Q000523:psychology*; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D010865:Pilot Projects; D033581:Stem Cell Transplantation; D011795:Surveys and Questionnaires; D019172:Transplantation Conditioning / Q000379:methods*; D014184:Transplantation, Homologous; D017211:Treatment Failure; D016896:Treatment Outcome