Allogeneic stem cell transplantation for sickle cell disease. A study of patients' decisions

Abstract

Summary:Allogeneic stem cell transplantation is increasingly considered as a curative though risky treatment option for adults with sickle cell disease. Little is known about attitudes of adult patients and their health care providers regarding the risks and benefits of transplantation. A survey of 100 patients and their health care providers was undertaken. Assessment of risk was by a reference gamble paradigm. Comparison was made of the characteristics of those accepting substantial risk vs those not accepting risk, as well as assessment of agreement on risks recommended by health care providers and accepted by patients. Sixty-three of 100 patients were willing to accept some short-term risk of mortality in exchange for the certainty of cure. Fifteen patients were willing to accept more than 35% mortality risk. No differences in patient or disease-related variables were identified between those accepting risk and those not accepting risk. There was no agreement between the recommendations of health care providers and the risk accepted by patients. A substantial proportion of adults with sickle cell disease are interested in curative treatment, at the expense of considerable risk. The decision to accept risk is influenced by individual patient values that cannot be easily quantified and that do not correlate with the assessment of the health care provider. Given the substantial interest in curative therapy, education about and consultation for allogeneic stem cell transplantation in sickle cell patients should be encouraged. Bone Marrow Transplantation (2001) 28, 545-549. Keywords: sickle cell disease; stem cell transplantation; risk; survey Sickle cell disease is a common congenital disorder with major implications for the health and longevity of affected 1 Several mature studies of allogeneic transplantation in children with sickle cell disease indicate a cure rate of 80%, a transplant-related mortality of less than 10% and a risk for serious complications (ie graft failure, grade III-IV acute GVHD and extensive chronic GVHD) of less than 10%.2,3 In one study, survival and cure rates were even better in children with early stage sickle cell disease.2 In adults, sickle cell disease causes considerably more morbidity and mortality than in children, and no satisfactory medical treatment exists.

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

“…The high acceptance rate of this procedure is not surprising, given the similar response to the survey in the United States which explored the attitude of patients with SCD towards the mortality associated with conventional allografting. 14 Apparently, there is little impact of the health-care system on patient response if one compares these studies. One might expect a different attitude in private health insurance systems compared with a state-funded health economy.…”

“…Two issues, namely the acceptance of mortality associated with the procedure (transplant related mortality (TRM)) and the acceptance of failure of this procedure (graft-failure (GF)) without mortality, that is autologous recovery with a return to previous disease state, were explored in a gambleparadigm as in two previous studies. 13,14 This was done as follows. In the first situation, allo-BMT/SCT was described as offering 100% cure of SCD without any mortality or GF.…”

Hydroxyurea treatment for sickle cell disease: impact on haematopoietic stem cell transplantation's outcome

“…Following the observation of a high incidence of convulsive episodes post transplantation, 25 the last 10 patients received clonazepam as an anticonvulsant prophylaxis from -12 to þ 100 days post transplant. Two patients received the same prophylaxis but for a shorter period (8 and 50 days post transplant).…”

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.…”