Barriers to Hematopoietic Cell Transplantation Clinical Trial Participation of African American and Black Youth With Sickle Cell Disease and Their Parents

Omondi, Nancy; Ferguson, S E; Majhail, Navneet S.; Denzen, Ellen M.; Buchanan, George R.; Haight, Ann E.; Labotka, Richard; Rizzo, J. Douglas; Murphy, Elizabeth A.

doi:10.1097/mph.0b013e31828d5e6a

Cited by 28 publications

(43 citation statements)

References 23 publications

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“…Transplant teams may also consider factors not assessed here for making decisions to offer transplantation to patients, such as the resources available to families for decision making and patient factors such as medication adherence, social support systems, and reluctance to participate in clinical trials involving transplantation. 35 …”

Section: Discussionmentioning

confidence: 99%

Pediatric Hematology Providers on Referral for Transplant Evaluation for Sickle Cell Disease

Mikles

Bhatia

Oyeku

et al. 2014

Journal of Pediatric Hematology/Oncology

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Summary Hematology referral for evaluation is a key step for hematopoietic stem cell transplantation for sickle cell disease (SCD). Pediatric SCD providers in the US Northeast (New York-Mid-Atlantic and New England regions) were surveyed anony-mously for perspectives and practices regarding transplant referral and compared by whether they practiced at SCD transplant centers. Data were analyzed using the Fisher exact test, χ2 test, and logistic regression. Half of the respondents practiced primarily at transplant sites. Most (79%) were enthusiastic about transplant for SCD and 78% had recently referred ≥1 child for evaluation. Overall, 77% limited referral to certain sickle hemoglobinopathies and 44% preferred referral for β-thalassemia to SCD. Indications selected for referral resembled current transplant criteria, plus family request or poor response to therapy. Referral for children on chronic transfusions predicted enthusiasm and prior referral. Many (66%) referred children with multiple SCD complications, even without matched sibling donors, 37% with sibling donors despite limited disease. Practitioners at transplant centers more commonly accepted event-free survival rates of ≤90% (P = 0.002). North-eastern providers expressed varying enthusiasm for referral for evaluation based on eligibility, donor availability, and acceptable risk, with modestly more interest from practitioners at transplant centers. Differing provider perspectives may affect patient referral for transplant consideration.

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Section: Discussionmentioning

confidence: 99%

Pediatric Hematology Providers on Referral for Transplant Evaluation for Sickle Cell Disease

Mikles

Bhatia

Oyeku

et al. 2014

Journal of Pediatric Hematology/Oncology

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“…Yet, far less than 1% of the SCD population in the US has received a transplant. Many barriers to transplant exist, and these are detailed in other reports 6, 78 . A key barrier is a prevailing assumption that HCT is risky and carries a mortality rate that exceeds mortality experienced with a supportive care approach.…”

Section: Introductionmentioning

confidence: 99%

Indications and Results of HLA-Identical Sibling Hematopoietic Cell Transplantation for Sickle Cell Disease

Walters

Castro

Sullivan

et al. 2016

Biology of Blood and Marrow Transplantation

106

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Although there are a number of published trials of human leukocyte antigen (HLA) – identical sibling hematopoietic cell transplantation (HCT) for sickle cell disease (SCD) that span 2 decades, when and for whom this therapy should be pursued is a subject of debate. Assessments of the risks of transplant-related complications that include infertility and debilitating graft-versus-host disease and long-term quality of life after successful HCT are difficult to perform without prospective trials in transplant and non-transplant cohorts. However, it is possible to assess the risk of mortality and to compare published rates of survival in individuals with sickle cell disease treated and not treated by HCT. In this brief review, projections about mortality risk based upon recent published reports are reviewed and summarized. The published data show overall survival and event-survival rates of 95% and 92%, respectively, in children treated by HLA-identical sibling HCT. The overall survival in the Center for International Blood and Marrow Transplant Research (CIBMTR) (N=412) and European Blood and Marrow Transplant (EBMT) (N=487) registries was 91% and 95%, respectively. These results provide broad support for the therapeutic value of HLA-identical sibling HCT for children with sickle cell disease and serve as the basis for a strong recommendation in favor of the option of HCT when a suitable donor is available. The experience of HLA-identical sibling HCT in adults with SCD is limited but appears similar to results in children, and these preliminary observations warrant further investigation.

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“…The few studies addressing this topic have focused on the factors that affect the decisions of children/adolescents with SCD or their parents. 8–13 Yet a large body of literature exists that examines the determinants of African-American participation in research generally. This research has found an important role for the attitudes that individuals have about clinical research.…”

Section: Introductionmentioning

confidence: 99%

Attitudes toward clinical trials among patients with sickle cell disease

et al. 2014

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Background A substantial number of planned clinical trials for sickle cell disease (SCD) have terminated early due to insufficient patient enrollment. Purpose To describe attitudes towards clinical trials among a sample of adults with SCD and identify patient-level factors associated with these attitudes. Methods Our data came from a sample [N = 291] of primarily adults with SCD participating in the Improving Patient Outcomes with Respect and Trust (IMPORT) study, which is a federally-funded observational study of SCD patient experiences in seeking health care. Attitudes towards clinical trials were assessed using items from the Perceptions of Participation in Clinical Research instrument. Patient factors examined as potential correlates of clinical trial attitudes were demographics, disease severity, engagement in self-care, trust, healthcare experience ratings, and prior history of participation in clinical trials. Multiple regression analyses were used to identify patient-level correlates of clinical trial attitudes. Results Our sample of SCD patients expressed overwhelmingly favorable attitudes about clinical trials, with 77% to 92% of our sample expressing agreement with a series of positive statements about clinical trials in general. Demographics, engagement in self-care, healthcare experience ratings, and prior trial participation each explained significant portions of the variability in clinical trial attitudes. Limitations The generalizability of our results to the entire SCD population may be of concern as the study participants were all receiving care at comprehensive sickle cell centers and already participating in clinical research. Conclusions Our results suggest that, in principle, adults with SCD enrolled in an observational study express very positive general attitudes about clinical trial participation and that specific factors attached to particular clinical trial opportunities may play a greater role in a SCD patient’s decision to participate than a general unwillingness to participate.

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Barriers to Hematopoietic Cell Transplantation Clinical Trial Participation of African American and Black Youth With Sickle Cell Disease and Their Parents

Cited by 28 publications

References 23 publications

Pediatric Hematology Providers on Referral for Transplant Evaluation for Sickle Cell Disease

Pediatric Hematology Providers on Referral for Transplant Evaluation for Sickle Cell Disease

Indications and Results of HLA-Identical Sibling Hematopoietic Cell Transplantation for Sickle Cell Disease

Attitudes toward clinical trials among patients with sickle cell disease

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