Engaging hematopoietic cell transplantation patients and caregivers in the design of print and mobile application individualized survivorship care plan tools.

PURPOSE: INSPIRE (INteractive Survivorship Program with Information and REsources) is an online health program that includes a mobile app, website, health action plan, and individualized survivorship care plans for adult hematopoietic cell transplant (HCT) survivors. The INSPIRE program integrates two previously effective randomized control trials that tested an internet-based program and patient-centered survivorship care plans for HCT survivors. METHODS: Three focus groups were conducted with a total of 22 participants (20 patients, 2 caregivers/patient advocates) to explore patient and caregiver preferences and to optimize the patient-centered emphasis of INSPIRE. Adult (age > 18 years at the time of study entry) HCT recipients had to be at least 1-year post-HCT to participate; caregivers/patient advocates were also eligible. Participants had to be able to communicate in English, could have any diagnosis, transplant type, or donor source, and could have had multiple transplants. RESULTS: All patient participants received an allogeneic HCT; average time since HCT was 8 years (range 2-22 years). The majority of participants were female (77.3%). Overall, the tools were well received by participants in this study, particularly the personalized features of all the tools. Major themes included interest in having the ability to tailor features to individual needs, and an interest in tracking information over time. DISCUSSION: Engaging patients and caregivers is invaluable to optimize tools designed to improve HCT survivorship care. Print, online, and mobile-based tools, tailored to individual patients' treatment history and requisite follow-up care, can provide otherwise unavailable expertise and guidelines for care.

Tailoring a Survivorship Care Plan: Patient and Provider Preferences for Recipients of Hematopoietic Cell Transplantation.

This study aimed to develop a survivorship care plan (SCP) that can be individualized to facilitate long-term follow-up care of hematopoietic cell transplantation (HCT) survivors. A sample SCP was developed that included 2 documents: a treatment summary and preventive care recommendations that combined data on treatment exposures routinely submitted by HCT centers to the Center for International Blood and Marrow Transplant Research (CIBMTR) with long-term follow-up guidelines. Focus groups were conducted by phone to characterize the critical patient-centered elements of the SCP. Focus group eligibility criteria included (1) adult patients >1 year post-HCT and their caregivers (3 groups; n = 22), (2) HCT physicians and advanced practice providers (APPs) (2 groups; n = 14), (3) HCT nurses and social workers (4 groups; n = 17), and (4) community health care professionals (3 groups; n = 24). Transcripts were analyzed for saturation of key themes using NVivo 10 software. Patients and caregivers suggested combining the treatment summary and care guidelines into a single document. They also requested sections on sexual and emotional health and the immune system. Providers wanted the treatment summary to focus only on what they absolutely must know. Themes were similar across healthcare professionals, although screening for psychosocial issues was emphasized more by the nurses and social workers. All preferred to receive the SCP electronically; however, hardcopy was considered necessary for some patients. All felt that the SCP would facilitate appropriate post-HCT care. This study highlights the need for an SCP instrument to facilitate HCT survivorship care. Furthermore, it demonstrates the feasibility and value of engaging HCT recipients, caregivers, and providers in developing an SCP. Their feedback was incorporated into a final SCP that was subsequently tested in a randomized trial.

Randomized controlled trial of individualized treatment summary and survivorship care plans for hematopoietic cell transplantation survivors.

Survivorship Care Plans (SCPs) may facilitate long-term care for cancer survivors, but their effectiveness has not been established in hematopoietic cell transplantation recipients. We evaluated the impact of individualized SCPs on patient-reported outcomes among transplant survivors. Adult (≥18 years at transplant) survivors who were 1-5 years post transplantation, proficient in English, and without relapse or secondary cancers were eligible for this multicenter randomized trial. SCPs were developed based on risk-factors and treatment exposures using patient data routinely submitted by transplant centers to the Center for International Blood and Marrow Transplant Research and published guidelines for long-term follow up of transplant survivors. Phone surveys assessing patient-reported outcomes were conducted at baseline and at 6 months. The primary end point was confidence in survivorship information, and secondary end points included cancer and treatment distress, knowledge of transplant exposures, health care utilization, and health-related quality of life. Of 495 patients enrolled, 458 completed a baseline survey and were randomized (care plan=231, standard care=227); 200 (87%) and 199 (88%) completed the 6-month assessments, respectively. Patients' characteristics were similar in the two arms. Participants on the care plan arm reported significantly lower distress scores at 6 months and an increase in the Mental Component Summary quality of life score assessed by the Short Form 12 (SF-12) instrument. No effect was observed on the end point of confidence in survivorship information or other secondary outcomes. Provision of individualized SCPs generated using registry data was associated with reduced distress and improved mental domain of quality of life among 1-5 year hematopoietic cell transplantation survivors. Trial registered at clinicaltrials.gov 02200133.

Addressing Knowledge Gaps in Acute Myeloid Leukemia to Improve Referral for Hematopoietic Cell Transplantation Consultation.

BACKGROUND: Outcomes after hematopoietic cell transplantation (HCT) for acute myeloid leukemia (AML) are better when HCT is performed during first complete remission (CR1). This study aimed to identify and address knowledge gaps that affect the timely referral of patients for HCT consultation. METHODS: A mixed-methods educational needs assessment included a national survey and focus groups consisting of hematologists/oncologists. An educational intervention of 3 webinars addressed identified knowledge gaps. RESULTS: A total of 150 hematologists/oncologists were recruited for the survey, of whom 20 participated in focus groups. Physicians in practice 0 to 10 years were 4.2 times more likely to refer for HCT consultation in CR1 than those with >10 years in practice (P=.0027). Physicians seeing ≤10 patients with AML in the past year were 3.7 times more likely to refer for HCT consultation in CR1 than those seeing >10 patients (P=.0028). Knowledge gaps included (1) improper classification of molecular/cytogenetic results for risk stratification, (2) lack of understanding that disease stage impacts outcomes, and (3) use of chronologic age alone for referral decision-making. Combined attendance for the webinars was 1,098 clinicians; >74% of participants indicated that they would apply the knowledge they gained in clinical practice. Trends were observed toward improvement in identifying favorable-risk AML, from 48% to 60% (n=85; P=.12); improvement in identifying 2 poor-risk cytogenetic/molecular abnormalities, with the percentage of respondents indicating chromosome 7 deletion increasing from 51% to 70% (n=53; P=.05) and that of respondents indicating TP53 mutation increasing from 42% to 62% (n=62; P=.03); and improvement in identifying which patients with AML aged >60 years were most likely to benefit from HCT based on cytogenetic/molecular features, with the percentage of correct responses increasing from 66% to 81% (n=62; P=.07). CONCLUSIONS: The webinars met the educational needs of learners and improved knowledge gaps. This study provided novel insights into the learning needs of clinicians who care for patients with AML and a roadmap for future educational interventions.

Caregiver availability and patient access to hematopoietic cell transplantation: social worker perspectives inform practice.

PURPOSE: Hematopoietic cell transplantation (HCT) often involves a long hospitalization and recovery period, with patients generally required to have a caregiver. This study aimed to identify transplant center (TC) requirements for a caregiver, describe challenges that impact caregiver availability, and identify potential solutions. METHODS: An exploratory sequential mixed-methods approach was used. Qualitative data was obtained from focus groups of TC social workers in the United States (US) (three focus groups; n = 15 total participants). Results informed the development of a national, web-based survey that was administered to the primary social worker contact at TCs in the National Marrow Donor Program (NMDP)/Be The Match Network (n = 133). RESULTS: Respondents included social workers from adult (n = 47) and pediatric (n = 19) TCs (response rate = 49%). The majority (89%) of both adult and pediatric TCs required a caregiver for a patient to proceed to transplant, but requirements varied in length of time, formality, transplant type, and HCT setting. Regardless of transplant type or patient population, social workers identified loss of caregiver income as the greatest challenge to caregiver availability, with the most common solution being allowing patients to have multiple caregivers throughout the transplant course. DISCUSSION: Caregiver availability is an important concern for patients considering and receiving HCT, and may be a barrier proceeding to HCT when a caregiver is unavailable. Results from this study highlight caregiver availability barriers and solutions of TCs across the US. These results can inform TCs about other center experiences with caregiver availability and identify potential practice changes for individual TCs.

Perceived Workforce Challenges among Clinical Social Workers in Hematopoietic Cell Transplantation Programs.

Clinical social workers are psychosocial care experts who provide interventions that aim to address the emotional, relational, financial, and logistical challenges that arise throughout the hematopoietic cell transplantation (HCT) treatment and recovery process. Interventions that contribute to better patient outcomes can include cognitive behavioral therapy and counseling for adaptation to illness, family planning for 24/7 caregiver availability and strategies to support patient activities of daily living, instruction on guided imagery and relaxation techniques for symptom management and to decrease anxiety, psychoeducation on the treatment trajectory, and linkage with financial resources. A Social Work Workforce Group (SWG) was established through the System Capacity Initiative, led by the National Marrow Donor Program/Be The Match, to characterize the current social work workforce capacity and challenges. The SWG conducted a web-based survey of HCT clinical social workers in the United States. The response rate was 57% (n = 90), representing 76 transplant centers. Survey results indicated that the clinical social worker role and scope of practice varies significantly between centers; less than half of respondents reported that their clinical social work expertise was used to its fullest potential. With an estimated 3-fold increase in HCT patient volume by 2020, the need for specialized psychosocial health services will increase. The SWG makes recommendations to build capacity for the psychosocial care of HCT patients and to more fully integrate the social worker as a core member of the HCT team. The SWG created a Blood and Marrow Transplant (BMT) Clinical Social Worker role description that can be used by transplant centers to educate healthcare professionals, benchmark utilization of clinical social workers, and improve comprehensive psychosocial health programs.

Translation of Clinical Research into Practice: An Impact Assessment of the Results from the Blood and Marrow Transplant Clinical Trials Network Protocol 0201 on Unrelated Graft Source Utilization.

Barriers and facilitators to adoption of results of clinical trials are substantial and poorly understood. We sought to examine whether the results of the randomized, multicenter Blood and Marrow Transplant Clinical Trials Network (BMT CTN) 0201 study comparing peripheral blood (PB) with bone marrow (BM) stem cells for unrelated donor (URD) hematopoietic cell transplantation (HCT) changed practice from PB to BM graft utilization and explored factors that impact graft selection and translation of research results into practice. The difference between use of URD BM and PB in the 2 years before and after publication of results in 2012 was examined using observational data collected by the Center for Blood and Marrow Transplant Research. A web-based survey of transplant physicians was conducted to understand the change in physician-reported personal and center preferred URD graft. No significant change in use of BM versus PB grafts occurred after 2012. Both BMT CTN participating and nonparticipating centers continued to use PB. Ninety-two percent of respondents were aware of the study results; 18% reported a change in personal and 16% reported a change in their center's practice of requesting BM instead of PB for URD HCT. Patient characteristics and the perception that engaging local champions to increase the evidence uptake were factors associated with personal or center change in practice. Despite awareness of the trial results, fewer than one-fifth of HCT physicians reported practice change in response to the BMT CTN 0201 results. Observational data confirmed no discernible change in practice.

Burnout, Moral Distress, Work-Life Balance, and Career Satisfaction among Hematopoietic Cell Transplantation Professionals.

A projected shortage of hematopoietic cell transplantation (HCT) health professionals was identified as a major issue during the National Marrow Donor Program/Be The Match System Capacity Initiative. Work-related distress and work-life balance were noted to be potential barriers to recruitment/retention. This study examined these barriers and their association with career satisfaction across HCT disciplines. A cross-sectional, 90-item, web-based survey was administered to advanced practice providers, nurses, physicians, pharmacists, and social workers in 2015. Participants were recruited from membership lists of 6 professional groups. Burnout (measured with the Maslach Burnout Inventory subscales of emotional exhaustion and depersonalization) and moral distress (measured by Moral Distress Scale-Revised) were examined to identify work-related distress. Additional questions addressed demographics, work-life balance, and career satisfaction. Of 5759 HCT providers who received an individualized invitation to participate, 914 (16%) responded; 627 additional participants responded to an open link survey. Significant differences in demographic and practice characteristics existed across disciplines (P < .05). The prevalence of burnout differed across disciplines (P < .05) with an overall prevalence of 40%. Over one-half of pharmacists had burnout, whereas social workers had the lowest prevalence at less than one-third. Moral distress scores ranged from 0 to 336 and varied by discipline (P < .05); pharmacists had the highest mean score (62.9 ± 34.8) and social workers the lowest (42.7 ± 24.4). In multivariate and univariate analyses, variables contributing to burnout varied by discipline; however, moral distress was a significant contributing factor for all providers. Those with burnout were more likely to report inadequate work-life balance and a low level of career satisfaction; however, overall there was a high level of career satisfaction across disciplines. Burnout, moral distress, and inadequate work-life balance existed at a variable rate in all HCT disciplines, yet career satisfaction was high. These results suggest specific areas to address in the work environment for HCT health professionals, especially the need for relief of moral distress and a greater degree of personal time. As the creation of healthy work environments is increasingly emphasized to improve quality care and decrease costs, these findings should be used by HCT leadership to develop interventions that mitigate work-related distress and in turn foster recruitment and retention of HCT providers.

Engaging Patients in Setting a Patient-Centered Outcomes Research Agenda in Hematopoietic Cell Transplantation.

The goal of patient-centered outcomes research (PCOR) is to help patients and those who care for them make informed decisions about healthcare. However, the clinical research enterprise has not involved patients, caregivers, and other nonproviders routinely in the process of prioritizing, designing, and conducting research in hematopoietic cell transplantation (HCT). To address this need the National Marrow Donor Program/Be The Match engaged patients, caregivers, researchers, and other key stakeholders in a 2-year project with the goal of setting a PCOR agenda for the HCT community. Through a collaborative process we identified 6 major areas of interest: (1) patient, caregiver, and family education and support; (2) emotional, cognitive, and social health; (3) physical health and fatigue; (4) sexual health and relationships; (5) financial burden; and (6) models of survivorship care delivery. We then organized into multistakeholder working groups to identify gaps in knowledge and make priority recommendations for critical research to fill those gaps. Gaps varied by working group, but all noted that a historical lack of consistency in measures use and patient populations made it difficult to compare outcomes across studies and urged investigators to incorporate uniform measures and homogenous patient groups in future research. Some groups advised that additional pre-emptory work is needed before conducting prospective interventional trials, whereas others were ready to proceed with comparative clinical effectiveness research studies. This report presents the results of this major initiative and makes recommendations by working group on priority questions for PCOR in HCT.

Healthcare Costs and Utilization for Patients Age 50 to 64 Years with Acute Myeloid Leukemia Treated with Chemotherapy or with Chemotherapy and Allogeneic Hematopoietic Cell Transplantation.

The primary aim of this study was to describe healthcare costs and utilization during the first year after a diagnosis of acute myeloid leukemia (AML) for privately insured non-Medicare patients in the United States aged 50 to 64 years who were treated with either chemotherapy or chemotherapy and allogeneic hematopoietic cell transplantation (alloHCT). MarketScan (Truven Health Analytics) adjudicated total payments for inpatient, outpatient, and prescription drug claims from 2007 to 2011 were used to estimate costs from the health system perspective. Stabilized inverse propensity score weights were constructed using logistic regression to account for differential selection of alloHCT over chemotherapy. Weighted generalized linear models adjusted costs and utilization (hospitalizations, inpatient days, and outpatient visit-days) for differences in age, sex, diagnosis year, region, insurance plan type, Elixhauser Comorbidity Index), and 60-day prediagnosis costs. Because mortality data were not available, models could not be adjusted for survival times. Among 29,915 patients with a primary diagnosis of AML, 985 patients met inclusion criteria (774 [79%] receiving chemotherapy alone and 211 [21%] alloHCT). Adjusted mean 1-year costs were $280,788 for chemotherapy and $544,178 for alloHCT. Patients receiving chemotherapy alone had a mean of 4 hospitalizations, 52.9 inpatient days, and 52.4 outpatient visits in the year after AML diagnosis; patients receiving alloHCT had 5 hospitalizations, 92.5 inpatient days, and 74.5 outpatient visits. Treating AML in the first year after diagnosis incurs substantial healthcare costs and utilization with chemotherapy alone and with alloHCT. Our analysis informs healthcare providers, policymakers, and payers so they can better understand treatment costs and utilization for privately insured patients aged 50 to 64 with AML.

National Institutes of Health Hematopoietic Cell Transplantation Late Effects Initiative: The Patient-Centered Outcomes Working Group Report.

In 2015, the National Institutes of Health convened six working groups to address the research needs and best practices for late effects of hematopoietic stem cell transplantation survivors. The Patient-Centered Outcomes Working Group, charged with summarizing the HRQOL evidence base, used a scoping review approach to efficiently survey the large body of literature in adult and pediatric HCT survivors over 1 year after transplantation. The goals of this paper are to (1) summarize the current literature describing patient-centered outcomes in survivors, including the various dimensions of health-related quality of life affected by HCT, and describe interventions tested to improve these outcomes; (2) highlight areas with sufficient evidence allowing for integration into standard practice; (3) address methodological issues that restrict progress in this field; (4) identify major gaps to guide future research; and (5) specify priority research recommendations. Patient-centered outcomes were summarized within physical, psychological, social, and environmental domains, as well as for adherence to treatment, and health behaviors. Interventions to improve outcomes were evaluated for evidence of efficacy, although few interventions have been tested in long-term HCT survivors. Methodologic issues defined included lack of consistency in the selection of patient-centered outcome measures, along with the absence of a standard for timing, frequency, and mode of administration. Recommendations for HCT survivorship care included integration of annual screening of patient-centered outcomes, use of evidence-based practice guidelines, and provision of treatment summaries and survivorship care plans after HCT. Three priority research recommendations included the following: (1) design and test risk-targeted interventions with dose-intensity modulation matching the needs of HCT survivors with priority domains, including sexual dysfunction, fatigue, sleep disruption, nonadherence to medications and recommended health care, health behaviors including physical inactivity and healthy eating, and psychological dysfunction, with particular consideration of novel technologies to reach HCT survivors distant from their transplantation centers; (2) design a consensus-based methodologic framework for outcomes evaluation; and (3) evaluate and compare existing practices for integrating patient-centered outcome screening and interventions across HCT survivorship programs.

An Innovative Educational Program for International Hematopoietic Cell Transplant Pediatric Patients.

Pediatric patients and their families confront a difficult journey with hematopoietic cell transplantation. Understanding complex information, navigation, and coping with emotional, physical, and social changes in the patient, parents and family nucleus life could be quite challenging for all. In Spanish-speaking countries where transplant is emerging as an available treatment option, healthcare professionals encounter the lack or limited quality resources to educate pediatric patients and their families on a complex treatment process. Also, the level of literacy of each family varies widely, making transplant education very difficult to address in a simple and engaging way that could benefit all levels of literacy and foster well-being in the family as a whole, so they are prepared to make better informed decisions and decrease stress in all members. Super Sam versus the Marrow Monsters is a DVD resource made by the National Marrow Donor Program® (NMDP)/Be The Match® intended as an educational tool, to be shared with our U.S. network transplant centers to use when educating their pediatric patients. Hospital Angeles Lomas in the State of Mexico-member of the NMDP international network-has adopted the DVD with their pediatric audience, with very successful results, and demonstrates the benefits of expanding the initial intended use.

Administrative Claims Data for Economic Analyses in Hematopoietic Cell Transplantation: Challenges and Opportunities.

There is an increasing need for the development of approaches to measure quality, costs, and resource utilization patterns among allogeneic hematopoietic cell transplantation (HCT) patients. Administrative claims data provide an opportunity to examine service utilization and costs, particularly from the payer's perspective. However, because administrative claims data are primarily designed for reimbursement purposes, challenges arise when using it for research. We use a case study with data derived from the 2007 to 2011 Truven Health MarketScan Research database to discuss opportunities and challenges for the use of administrative claims data to examine the costs and service utilization of allogeneic HCT and chemotherapy alone for patients with acute myeloid leukemia (AML). Starting with a cohort of 29,915 potentially eligible patients with a diagnosis of AML, we were able to identify 211 patients treated with HCT and 774 treated with chemotherapy alone where we were sufficiently confident of the diagnosis and treatment path to allow analysis. Administrative claims data provide an avenue to meet the need for health care costs, resource utilization, and outcome information. However, when using these data, a balance between clinical knowledge and applied methods is critical to identifying a valid study cohort and accurate measures of costs and resource utilization.

Patient housing barriers to hematopoietic cell transplantation: results from a mixed-methods study of transplant center social workers.

PURPOSE: Hematopoietic cell transplantation (HCT) is performed in select centers in the United States (U.S.), and patients are often required to temporarily relocate to receive care. The purpose of this study was to identify housing barriers impacting access to HCT and potential solutions. METHODS: A mixed-methods primary study of HCT social workers was conducted to learn about patient housing challenges and solutions in place that help address those barriers. Three telephone focus groups were conducted with adult and pediatric transplant social workers (n = 15). Focus group results informed the design of a national survey. The online survey was e-mailed to a primary social worker contact at 133 adult and pediatric transplant centers in the U.S. Transplant centers were classified based on the patient population cared for by the social worker. RESULTS: The survey response rate was 49%. Among adult programs (n = 45), 93% of centers had patients that had to relocate closer to the transplant center to proceed with HCT. The most common type of housing option offered was discounted hotel rates. Among pediatric programs (n = 20), 90% of centers had patients that had to relocate closer to the transplant center to proceed with HCT. Ronald McDonald House was the most common option available. CONCLUSIONS: This study is the first to explore housing challenges faced by patients undergoing HCT in the U.S. from the perspective of social workers and to highlight solutions that centers use. Transplant centers will benefit from this knowledge by learning about options for addressing housing barriers for their patients.

National Survey of Hematopoietic Cell Transplantation Center Personnel, Infrastructure, and Models of Care Delivery.

Hematopoietic cell transplantation (HCT) is a complex procedure that requires availability of adequate infrastructure, personnel, and resources at transplantation centers. We conducted a national survey of transplantation centers in the United States to obtain data on their personnel, infrastructure, and care delivery models. A 42-item web-based survey was administered to medical directors of transplantation centers in the United States that reported any allogeneic HCT to the Center for International Blood and Marrow Transplant Research in 2011. The response rate for the survey was 79% for adult programs (85 of 108 centers) and 82% for pediatric programs (54 of 66 centers). For describing results, we categorized centers into groups with similar volumes based on 2010 total HCT activity (adult centers, 9 categories; pediatric centers, 6 categories). We observed considerable variation in available resources, infrastructure, personnel, and care delivery models among adult and pediatric transplantation centers. Characteristics varied substantially among centers with comparable transplantation volumes. Transplantation centers may find these data helpful in assessing their present capacity and use them to evaluate potential resource needs for personnel, infrastructure, and care delivery and in planning for growth.

Designing and operationalizing a customized internal evaluation model for cancer treatment support programs.

Be The Match® Patient and Health Professional Services (PHPS) supports patients undergoing hematopoietic cell transplant (HCT) and caregivers by providing educational programs and resources. HCT is a potentially curative therapy for blood cancers such as leukemia and lymphoma. To help meet the increasing demand for support services, PHPS implemented a multipronged plan to build and sustain the organization's capacity to conduct evaluation of its programs and resources. To do so, PHPS created and operationalized an internal evaluation model, developed customized resources to help stakeholders incorporate evaluation in program planning, and implemented utilization-focused evaluation for quality improvement. Formal mentorship was also critical in the development of an evidence-based, customized model and navigating inherent challenges throughout the process. Our model can serve as a guide for evaluators on establishing and operationalizing an internal evaluation program. Ultimately, we seek to improve support and education services from the time of diagnosis through survivorship.

Hematopoietic cell transplantation in 2020: summary of year 2 recommendations of the National Marrow Donor Program's System Capacity Initiative.

The National Marrow Donor Program, in partnership with the American Society for Blood and Marrow Transplantation, sponsored and organized a series of symposia to identify complex issues affecting the delivery of hematopoietic cell transplantation (HCT) and to collaboratively develop options for solutions. "Hematopoietic Cell Transplantation in 2020: A System Capacity Initiative" used a deliberative process model to engage professional organizations, experts, transplant centers, and stakeholders in a national collaborative effort. Year 2 efforts emphasized data analysis and identification of innovative ideas to increase HCT system efficiency, address future capacity requirements, and ensure adequate reimbursement for HCT programs to meet the projected need for HCT. This report highlights the deliberations and recommendations of Year 2 and the associated symposium held in September 2011.

Barriers to hematopoietic cell transplantation clinical trial participation of african american and black youth with sickle cell disease and their parents.

African Americans and Blacks have low participation rates in clinical trials and reduced access to aggressive medical therapies. Hematopoietic cell transplantation (HCT) is a high-risk but potentially curative therapy for sickle cell disease (SCD), a disorder predominantly seen in African Americans. We conducted focus groups to better understand participation barriers to HCT clinical trials for SCD. Nine focus groups of youth with SCD (n=10) and parents (n=41) were conducted at 3 sites representing the Midwest, South Atlantic, and West South Central US. Main barriers to clinical trial participation included gaps in knowledge about SCD, limited access to SCD/HCT trial information, and mistrust of medical professionals. For education about SCD/HCT trials, participants highly preferred one-on-one interactions with medical professionals and electronic media as a supplement. Providers can engage with sickle cell camps to provide information on SCD/HCT clinical trials to youth and local health fairs for parents/families. Youth reported learning about SCD through computer games; investigators may find this medium useful for clinical trial/HCT education. African Americans affected by SCD face unique barriers to clinical trial participation and have unmet HCT clinical studies education needs. Greater recognition of these barriers will allow targeted interventions in this community to increase their access to HCT.

Costs and cost-effectiveness of hematopoietic cell transplantation.

Interest is growing in economic and comparative effectiveness analyses, with increasing emphasis on optimizing healthcare resources and costs. Limited information is available on the economic aspects of hematopoietic cell transplantation (HCT). We review contemporary literature on the costs and cost-effectiveness of HCT in the United States and worldwide. Published studies confirm the high costs associated with HCT, although the reported costs are highly variable, related to the differing methodologies used across studies. We examine the challenges in reviewing costs and cost-effectiveness across studies specific to HCT and highlight factors identified as associated with higher costs of HCT. We also discuss opportunities for future research in this area.

Easy-to-read informed consent forms for hematopoietic cell transplantation clinical trials.

Informed consent is essential to ethical research and is requisite to participation in clinical research. Yet most hematopoietic cell transplantation (HCT) informed consent forms (ICFs) are written at reading levels that are above the ability of the average person in the United States (U.S.). The recent development of ICF templates by the National Cancer Institute, National Institutes of Health, and the National Heart Blood and Lung Institute have not resulted in increased patient comprehension of information. Barriers to creating Easy-to-Read ICFs that meet U.S. federal requirements and pass institutional review board (IRB) review are the result of multiple interconnected factors. The Blood and Marrow Transplant Clinical Trials Network (BMT CTN) formed an ad hoc review team to address concerns regarding the overall readability and length of ICFs used for BMT CTN trials. This paper summarizes recommendations of the review team for the development and formatting of Easy-to-Read ICFs for HCT multicenter clinical trials, the most novel of which is the use of a 2-column format. These recommendations intend to guide the ICF writing process, simplify local IRB review of the ICF, enhance patient comprehension, and improve patient satisfaction. The BMT CTN plans to evaluate the impact of the Easy-to-Read format compared with the traditional format on the informed consent process.