The National Marrow Donor Program's Symposium on Patient Advocacy in Cellular Transplantation Therapy: Addressing Barriers to Hematopoietic Cell Transplantation

Article Outline

• Abstract
• Introduction
• Symposium Organization
• Pretransplantation Working Group
  • Key Barriers and Recommended Initiatives
    • Timing of Referral for Transplantation
    • Availability of Unrelated Donors and UCB Units
    • Financial Clearance during the Pretransplantation Phase
• Early Posttransplantation Working Group
  • Key Barriers and Recommended Initiatives
    • Inadequate Knowledge of Specific Patient and Caregiver Needs
    • Lack of Information for Patients and Caregivers
    • Inadequate Resources and Support for Caregivers
• Survivorship Working Group
  • Key Barriers and Recommended Initiatives
    • Psychosocial Support
    • Medical Care Concerns
    • Financial Concerns
• Financial Working Group
  • Key Barriers and Recommended Initiatives
    • There is a lack of patient education regarding insurance, finances, and employment rights
    • Coverage Restrictions
    • Fundraising Barriers
    • Limited Legislative Advocacy
• Special Populations Working Group
  • Key Barriers and Recommended Initiatives
• Summary of Recommendations
• Acknowledgments
• References
• Copyright

Although hematopoietic cell transplantation (HCT) is an effective treatment option for patients with life-threatening blood, immune system, or genetic disorders, many barriers besides a lack of suitably matched donors exist and can have an adverse impact on access and outcomes of HCT. In 2008, the National Marrow Donor Program, through its Office of Patient Advocacy, convened a diverse group of experts and transplantation survivors to identify persistent patient barriers throughout the transplantation process and to make recommendations for programs and initiatives to address these barriers, including new research opportunities. This group included transplantation physicians and other health care providers, relevant subject experts, and representatives from transplantation centers and patient advocacy organizations. Working groups were formed to identify patient barriers to HCT and to recommend and prioritize initiatives as they relate to the pretransplantation period, the early posttransplantation period, long-term survivorship, financial issues, and special populations. This report summarizes the symposium's deliberations and recommendations to address persistent patient barriers throughout the transplantation process.

Key Words: Hematopoietic stem cell transplantation, Quality of life, Barriers to transplantation, Patient advocacy, Health disparities, Survivorship, Financial barriers, National Marrow Donor Program

Back to Article Outline

Introduction 

Despite the tremendous progress in transplantation over the past 4 decades, persistent barriers to successful transplantation still exist. Traditionally, lack of a suitable donor has been considered among the key barriers to unrelated donor hematopoietic cell transplantation (HCT). Efforts to increase the number and availability of adult volunteer unrelated donors in donor registries such as the Be The Match Registry^SM of the National Marrow Donor Program® (NMDP), the use of haploidentical family donors, and the introduction of unrelated umbilical cord blood (UCB) as a graft source has partially overcome this problem and substantially increased the probability that patients who need HCT will be able to find a suitable donor [1]. But, other factors besides the availability of a suitable donor can significantly affect a patient's suitability for HCT. Consequently, the NMDP commissioned a symposium to identify persistent non-HLA patient barriers throughout the unrelated donor transplantation process and to make recommendations for programs and initiatives to address those barriers, including new research opportunities.

Back to Article Outline

Symposium Organization 

The NMDP, through its Office of Patient Advocacy (OPA), convened a diverse group of experts including transplant physicians and other medical providers, relevant subject experts, transplantation survivors, and representatives from transplant centers and patient advocacy organizations. Based on their areas of expertise, participants were assigned to 1 of 5 working groups: pretransplantation, early posttransplantation, survivorship, financial, and special populations. Although the charter of each of these working groups focused on unrelated donor transplantation, the participants were encouraged to also include and address barriers specific to allogeneic-related and autologous transplantation in their deliberations. Each group identified specific barriers to HCT and gave recommendations for new initiatives and programs designed to address these barriers (Table 1). The 5 groups conducted their work via conference call meetings over the summer of 2008. Their recommendations were presented, discussed and prioritized at a symposium, held in September 2008, in Minneapolis, Minnesota. Although each working group presented a substantial list of recommendations, what follows is a discussion of the recommendations regarding the high-priority items identified by each group. In many cases, there is scant scientific evidence on which to determine the magnitude or prevalence of the barriers identified, and the high-priority items were so designated based on their perceived importance by panelists.

Table 1. Important Non-HLA Barriers to Successful HCT and High-Priority Initiatives Needed to Address These Barriers, Along with Ongoing NMDP Initiatives to Address These Barriers

HCT, indicated hematopoietic cell transplantation; NMDP, National Marrow Donor Program.

Back to Article Outline

Pretransplantation Working Group 

The pretransplantion working group addressed barriers encountered by patients, physicians, and transplantation center health care professionals from the time of diagnosis to workup for HCT. This phase is characterized by important steps, including timely referral, consultation at a transplantation center, access to a suitably matched donor source, and financial clearance to proceed to workup for HCT.

Key Barriers and Recommended Initiatives 

Referring physicians may not always inform patients of transplantation as an option for treating their disease or refer patients to transplantation centers at an optimal time in the disease course. Transplantation late in the disease process leads to suboptimal outcomes and fewer patients able to proceed to transplantation because of disease progression, complications, and/or lack of time to identify a suitable donor 2, 3, 4, 5.

To improve physician/provider-targeted education, the working group recommended the following:

The working group also recognized the need for patient education. To improve patient-focused education, the working group recommended the following:

Donor unavailability can be a significant barrier to successful transplantation 8, 9, 10, 11. Reasons for donor attrition include deferment for medical reasons, temporary unavailability, loss of interest in participating in the program, and inability to locate donors. After initial confirmation, continued availability of the donor is also critical when workup is requested. For patients being considered for unrelated UCB transplantation, confirmatory typing of units by non-US CB banks can sometimes have a long turnaround time and hinder selection of optimal units by transplantation centers.

The working group recommended the following initiatives:

Lack of insurance coverage for donor search and graft procurement can be a significant barrier to HCT [12]. Lack of funds for patient housing and travel costs related to the transplantation evaluation process also can be a barrier to timely transplantation. Increasing the number of donors on the registry with high-resolution typing can help eliminate some steps during the search process and their related costs, as well as allow for more rapid transplantation planning.

To address financial barriers to the search and evaluation process, the working group recommended the following:

Back to Article Outline

Early Posttransplantation Working Group 

This working group addressed issues faced by patients in the early posttransplantation period (up to 6 months to 1 year after HCT), which occurs between the pretransplantation and survivorship phases and is the most intensive and dynamic phase of the transplantation experience. This phase is characterized by important transitions: travel to the transplantation center before the transplantation, setting up a temporary residence, entry into the transplantation therapy, transition between the hospital and outpatient clinic setting at the transplantation center, and discharge from the transplantation center back to home and local health care. Patients and their caregivers also are prone to financial adversity during this period.

Key Barriers and Recommended Initiatives 

There is a lack of existing data regarding early posttransplantation patient and caregiver needs, specifically the types of information that are useful, the manner in which to present this information, and the barriers to participation in clinical trials. In addition, little is known about the information needs of community physicians regarding this phase of transplantation therapy. Greater understanding of these requirements will aid in the development of new resources and proper utilization of existing materials and resources.

To assess patient and caregiver needs, the working group recommended the following:

Patients and families need more information about the issues that they face during this phase of transplantation. Such information must be available at multiple times and be presented via various modes to accommodate varying language and literacy needs. The NMDP currently provides a number of resources to support patients during the search process and the pretransplantation phase as well as the survivorship phase, but few resources address the acute medical complications and psychosocial issues that arise during the early posttransplantation phase. Even when such resources exist, some patients and caregivers are unaware of their availability. Moreover, individual learning styles differ, and patients may have an especially difficult time assimilating information during periods of stress and crisis. Participation in clinical trials may be daunting because of the lack of patient materials to explain the trial. The utilization and effectiveness of available NMDP patient resources requires further evaluation.

Recommendations to address this barrier include the following:

Caregivers are under enormous stress, especially in the early posttransplantation phase 13, 14. Their support mechanisms often are disrupted if they are far from home. There is considerable variability among transplantation centers in terms of resources to address these issues.

To address caregiver concerns, the working group recommended the following:

Back to Article Outline

Survivorship Working Group 

This working group focused on barriers to successful long-term survivorship for the time period beyond 6 months to 1 year after HCT. Substantial heterogeneity exists in long-term follow-up practices among transplantation centers. The working group noted that several informational resources already exist through the NMDP, its partner organizations, and elsewhere, but that awareness of the available programs among transplantation centers, health care professionals, patients, and caregivers is low and should be a target area for improvement.

Key Barriers and Recommended Initiatives 

Psychosocial concerns faced by survivors and their caregivers continue for months and years after HCT 16, 17, 18. These sequelae can include distress, depression, anxiety, posttraumatic stress disorder, fatigue, cognitive difficulties, sexual difficulties, and caregiver burnout. The distress component can comprise such issues as adjustment to life post-HCT, underlying fear of recurrence and complications, changes in family roles and relationships, return to work, and social integration. Although the NMDP and other patient advocacy organizations provide programs to address these psychosocial sequelae of HCT, awareness of these programs is limited. Other important barriers include a lack of knowledge of the prevalence and impact of psychosocial sequelae by health care providers and survivors, a lack of tools to assess and guidelines to treat psychosocial issues, and the absence of centrally located survivorship information for patients, caregivers, and providers. Caregiver-specific issues related to psychosocial support also are frequently overlooked.

Recommendations to address these barriers include the following:

Many of the medical barriers experienced during the survivorship phase for patients, caregivers, and health care professionals are related to an overall poor understanding of what occurs during the transplantation process and the importance of appropriate follow-up. There also may be poor communication between the transplantation center and the health care provider responsible for ongoing care after the patient leaves the transplantation center. Some health care providers may have little or no experience in caring for patients after transplantation. In addition, the patient or provider may not understand which posttransplantation medical problems call for the expertise of the transplantation center. There also is a general lack of awareness among patients, caregivers, physicians, and other medical providers of existing resources that provide recommendations for posttransplantation care.

Recommendations to address these concerns include the following:

For patients and caregivers, many of the financial concerns during the survivorship period involve insurance and insurability and the ability to return to work. Coping with long-term debt that may have accumulated because of transplantation and related costs can be a significant stressor for many patients and families. Specific educational initiatives to address financial concerns need to be developed through partnering with other organizations. In addition, educating transplantation centers about these patient resources will ensure improved utilization of available resources.

To address financial barriers to successful long-term survivorship, the working group recommended the following:

Back to Article Outline

Financial Working Group 

Financial barriers can be varied and considerable. A significant number of patients undergoing HCT incur medical debt and face ongoing financial challenges throughout the transplantation process. Nonmedical costs and other out-of-pocket expenses (including co-pays for prescription medications) associated with transplantation can add to patient and caregiver financial stress [14]. Some patients are not able to successfully rejoin the workforce because of long-term posttransplantation complications. This working group also recognized that the transplantation process can have a significant financial impact on caregivers as well.

Key Barriers and Recommended Initiatives 

Educating patients, caregivers, and the transplantation community about the financial impact of transplantation is important to allow patients to plan for HCT-related expenses. The OPA currently provides several tools, including the Financial Calculator and educational resources, to help patients and caregivers understand their insurance coverage and prepare for the financial implications of HCT. The OPA also offers financial help through the Patient Assistance Program and can help locate other financial resources; however, these resources are largely underutilized, because not all patients and transplantation centers are aware of their availability.

To increase the dissemination of existing resources that provide information about these issues, the working group recommended the following:

Insurance and Medicare coverage restrictions represent a major barrier to transplantation. Specific limitations can include lack of coverage for specific diagnoses, donor search or procurement limits, insufficient dollar-based transplantation benefit maximums, and denials for cutting-edge treatments (ie, unreasonable, experimental, or investigational policy language). Nationally, the NMDP engages payors through activities that focus on appropriate coverage and reimbursement for transplantation-related expenses and educates them about the indications for, processes in, and outcomes of HCT. The NMDP may need to enhance its payor education efforts to address these barriers.

Specific recommendations directed at the NMDP's payor education efforts include the following:

Fundraising can be an important tool to allow patients to meet some of the financial costs of transplantation. Our experience indicates that the majority of patients do not engage in fundraising efforts, however. Patients may attach a stigma to fundraising or asking for help. In addition, transplantation centers do not have the resources to assist patients in their fundraising efforts.

Recommendations to address this barrier include the following:

Several opportunities exist where government action could address financial barriers to transplantation. Recommended national government advocacy efforts include the following:

Back to Article Outline

Special Populations Working Group 

The transplantation experience can be particularly challenging to patients belonging to medically underserved communities. These patients frequently encounter barriers that substantially limit their access to transplantation as a treatment option and/or issues that affect the quality of life posttransplantation. These “special” populations include racial and ethnic minorities, persons with limited English proficiency or health literacy, residents of certain geographic regions (eg, rural), high-risk age groups (eg, pediatric, adolescent/young adult, over the age of 50 years), the uninsured and underinsured, and non-US citizens and residents. Within the definition of special populations, this working group also included caregivers as a distinctly underserved population, because they have unique care and support needs.

Key Barriers and Recommended Initiatives 

The working group identified 4 domains that are barriers to successful transplantation in medically underserved populations: (1) barriers that prevent access to care; (2) language, cultural, and literacy barriers; (3) environmental and geographic barriers; and (4) genetic and medical barriers. Special populations encounter disproportionate access barriers, including no or limited insurance coverage for transplantation and follow-up care, insufficient financial resources for medical and nonmedical expenses associated with transplantation, and lack of a committed caregiver [19]. Racial and ethnic minority populations are more likely to be uninsured or underinsured. 20, 21. There also is a need for culturally appropriate approaches for outreach and education of special populations. Patients residing in rural areas face many significant challenges in accessing adequate health care compared with those living in urban areas [22]. Issues specific to rural patients include isolation; limited access to health care services, employer-provided health insurance, and public transportation; and lower socioeconomic status. Individuals of certain ethnic backgrounds disproportionately represent both the most commonly matched and the most unique HLA tissue types needed for HCT. High levels of HLA diversity pose a significant challenge for populations at risk, because a patient is most likely to receive a HCT using a donor from his or her own racial or ethnic group.

To address the barriers encountered by underserved populations, this working group recommended a 2-track approach: (1) Ensure that underserved populations are considered in initiatives that address broader access issues, and (2) develop initiatives to address the barriers for populations at risk. Strategies to address these barriers require initiatives within the NMDP and at the local, regional, and national levels.

At the national level, the NMDP should increase advocacy efforts for expanded resources and programs that directly impact access to transplantation for patients and families from underserved populations, as well as those who work on their behalf. Specific key recommendations include the following:

To improve coordination and communication of available programs, resources, and materials among NMDP network centers and those who work on behalf of special populations, the working group recommended the following:

The NMDP should support local and regional initiatives that address barriers to transplantation that have a disproportionate impact on the access to transplantation for special populations. Specific important recommendations for such initiatives include thefollowing:

Back to Article Outline

Summary of Recommendations 

After the Chair of each working group presented the group's recommendations, the symposium attendees voted to prioritize these recommendations. Top priorities for the NMDP to address non-HLA barriers to HCT, in order of importance, include (1) increase awareness of existing HCT resources and services, (2) develop public policy information and education programs to promote access to HCT, (3) conduct a transplantation center needs assessment to determine their level of awareness and use of NMDP resources and services to assist HCT patients and caregivers, (4) conduct a patient needs assessment to determine effective ways to streamline the delivery of HCT information and obtain feedback on NMDP resources and services, (5) expand NMDP efforts to improve retention of donors in the Be The Match Registry, (6) expand the NMDP's Patient Assistance Program to provide financial grants earlier in the pretransplantation phase, (7) increase support for NMDP staff to conduct onsite visits of transplantation centers, and (8) expand promotion of the Referral Outreach Program.

Based on the symposium's prioritized recommendations, the NMDP instituted a special task force to identify metrics to quantify the effectiveness and efficiency of initiatives aimed at resolving or mitigating these barriers. In addition to this task force, the NMDP Board of Directors authorized its Patient-Focused Initiatives Committee to oversee the implementation of specific symposium initiatives and monitor their effectiveness. The NMDP also will continue to collaborate with other organizations to eliminate health disparities; for instance, it is a named partner in the National Institutes of Health's Strategic Plan on Reducing Health Disparities.

Back to Article Outline

Acknowledgments 

Financial disclosure: The authors thank Jeffrey Chell, MD, Dennis Confer, MD, and Dennis Lorentz, PhD, Chair, NMDP Board of Directors, and other members of the NMDP who helped with this effort. They also wish to acknowledge the participation of the following working group members:Pretransplantation Working Group

Back to Article Outline

References 

1. Ballen KK, King RJ, Chitphakdithai P, et al. The National Marrow Donor Program: 20 years of unrelated donor hematopoietic cell transplantation. Biol Blood Marrow Transplant. 2008;14:2–7
   • View In Article
   • Full Text
   • Full-Text PDF (51 KB)
   • CrossRef
2. Karanes C, Nelson GO, Chitphakdithai P, et al. Twenty years of unrelated donor hematopoietic cell transplantation for adult recipients facilitated by the National Marrow Donor Program. Biol Blood Marrow Transplant. 2008;14:8–15
   • View In Article
   • Full Text
   • Full-Text PDF (53 KB)
   • CrossRef
3. Lee SJ, Klein J, Haagenson M, et al. High-resolution donor-recipient HLA matching contributes to the success of unrelated donor marrow transplantation. Blood. 2007;110:4576–4583
   • View In Article
   • CrossRef
4. MacMillan ML, Davies SM, Nelson GO, et al. Twenty years of unrelated donor bone marrow transplantation for pediatric acute leukemia facilitated by the National Marrow Donor Program. Biol Blood Marrow Transplant. 2008;14:16–22
   • View In Article
   • Full Text
   • Full-Text PDF (75 KB)
   • CrossRef
5. Pasquini M, Wang Z. CIBMTR summary slides 2007 (part 2). CIBMTR Newslett. 2008;14:5–13
   • View In Article
6. American Society of Blood and Marrow Transplantation. Guidelines on recommended timing for transplant consultation, 2008. Available from: http://www.asbmt.org/policystat/policy.html. Accessed May 5, 2009.
   • View In Article
7. National Marrow Donor Program. Questions to ask your doctor about treatment options. Available from: http://www.marrow.org/PATIENT/Undrstnd_Disease_Treat/Discuss_Opt_w_Dr/PDFs/questions_about_treatment_options.pdf. Accessed May 5, 2009.
   • View In Article
8. Myaskovsky L, Switzer GE, Dew MA, et al. The association of donor center characteristics with attrition from the National Marrow Donor Registry. Transplantation. 2004;77:874–880
   • View In Article
   • MEDLINE
   • CrossRef
9. Switzer GE, Dew MA, Goycoolea JM, et al. Attrition of potential bone marrow donors at two key decision points leading to donation. Transplantation. 2004;77:1529–1534
   • View In Article
   • MEDLINE
   • CrossRef
10. Switzer GE, Myaskovsky L, Goycoolea JM, et al. Factors associated with ambivalence about bone marrow donation among newly recruited unrelated potential donors. Transplantation. 2003;75:1517–1523
    • View In Article
    • MEDLINE
    • CrossRef
11. Confer DL. The National Marrow Donor Program: meeting the needs of the medically underserved. Cancer. 2001;91:274–278
    • View In Article
    • MEDLINE
    • CrossRef
12. Kollman C, Weis T, Switzer GE, et al. Non-HLA barriers to unrelated donor stem cell transplantation. Bone Marrow Transplant. 2001;27:581–587
    • View In Article
    • MEDLINE
    • CrossRef
13. Frick E, Rieg-Appleson C, Tyroller M, et al. Social support, affectivity, and the quality of life of patients and their support-givers prior to stem cell transplantation. J Psychosoc Oncol. 2005;23:15–34
    • View In Article
    • MEDLINE
    • CrossRef
14. Meehan KR, Fitzmaurice T, Root L, et al. The financial requirements and time commitments of caregivers for autologous stem cell transplant recipients. J Support Oncol. 2006;4:187–190
    • View In Article
    • MEDLINE
15. Caregivers' guide for bone marrow/stem cell transplant. nbmtLINK 2007. Available from: https://nbmtlink.readyhosting.com/resources_support/cg/index.htm. Accessed May 5, 2009.
    • View In Article
16. Andrykowski MA, Bishop MM, Hahn EA, et al. Long-term health-related quality of life, growth, and spiritual well-being after hematopoietic stem cell transplantation. J Clin Oncol. 2005;23:599–608
    • View In Article
    • MEDLINE
    • CrossRef
17. Bishop MM, Beaumont JL, Hahn EA, et al. Late effects of cancer and hematopoietic stem cell transplantation on spouses or partners compared with survivors and survivor-matched controls. J Clin Oncol. 2007;25:1403–1411
    • View In Article
    • CrossRef
18. Fife BL, Huster GA, Cornetta KG, et al. Longitudinal study of adaptation to the stress of bone marrow transplantation. J Clin Oncol. 2000;18:1539–1549
    • View In Article
    • MEDLINE
19. Mandelblatt JS, Yabroff KR, Kerner JF. Equitable access to cancer services: a review of barriers to quality care. Cancer. 1999;86:2378–2390
    • View In Article
    • MEDLINE
    • CrossRef
20. Fiscella K, Franks P, Gold MR, et al. Inequality in quality: addressing socioeconomic, racial, and ethnic disparities in health care. JAMA. 2000;283:2579–2584
    • View In Article
    • MEDLINE
    • CrossRef
21. Navarro V. Race or class versus race and class: mortality differentials in the United States. Lancet. 1990;336:1238–1240
    • View In Article
    • CrossRef
22. Lishner DM, Richardson M, Levine P, et al. Access to primary health care among persons with disabilities in rural areas: a summary of the literature. J Rural Health. 1996;12:45–53
    • View In Article
    • MEDLINE
    • CrossRef