D. Kathryn Tierney

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.

Mobilization of Hematopoietic Stem Cells for Use in Autologous Transplantation

Autologous hematopoietic stem cell transplantation (HSCT) is a potentially curative therapeutic approach for various malignant hematologic and lymphoid diseases. Hematopoietic stem cells (HSCs) may be collected from the blood or the bone marrow. HSCs are capable of self-renewal and give rise to progenitor cells, multipotent cells that differentiate and proliferate into the mature cells of the blood and immune system. HSCs and progenitor cells are released from the bone marrow into the peripheral blood through a process called mobilization. HSCs then are collected from the blood in a process called apheresis and cryopreserved for administration following the high-dose preparative regimen. This article reviews stem cell biology, current mobilization strategies, use of novel mobilization agents, and nursing care of patients during the mobilization phase of autologous HSCT. Understanding the biology and process of HSC mobilization is critical for transplantation nurses to deliver and coordinate care during this complex phase of autologous HSCT.

Exploring long-term cancer survivors’ experiences in the career and financial domains: Interviews with hematopoietic stem cell transplantation recipients

Abstract Using semi-structured interviews with 50 hematopoietic stem cell transplantation (HSCT) recipients who were 2 to 22 years post-transplant, this study investigates cancer survivors’ interpretations of their economic and work-related experiences during and after treatment. Survivors described a variety of challenges in these areas, including job insecurity, discrimination, career derailment, the lack of career direction, delayed goals, financial losses, insurance difficulties, constraints on job mobility, and physical/mental limitations. Survivors described the ways these challenges were offset by external factors that helped them to navigate these difficulties and buffered the negative financial and career-related impacts. Good health insurance, favorable job characteristics, job accommodations, and financial buffers were prominent offsetting factors. Most survivors, however, were also forced to rely on individual behavioral and interpretative strategies to cope with challenges. Behavioral strategies included purposeful job moves, retraining, striving harder, and retiring. Some strategies were potentially problematic, such as acquiring large debt. Interpretive strategies included reprioritizing and value shifts, downplaying the magnitude of cancer impact on ones life, denying the causal role of cancer in negative events, making favorable social comparisons, and benefit finding. Post-treatment counseling and support services may assist survivors in identifying available resources and useful strategies to improve long-term adaptation in the career and financial realms.

PALLIATIVE CARE OF HEMATOPOIETIC CELL TRANSPLANT RECIPIENTS AND FAMILIES

OBJECTIVES To provide support for the early integration of palliative care into the care of hematopoietic cell transplant (HCT) recipients and families with the goal of improving care. DATA SOURCES Journal articles and on-line databases. CONCLUSION Early integration of palliative care for HCT recipients at high risk for complex symptom clusters, non-relapse mortality, or relapse offers an opportunity to clarify goals of care, advanced care planning, and improving the quality of care for both recipients and families. IMPLICATIONS FOR NURSING PRACTICE The palliative care service can support the HCT nurse in providing complex care to HCT recipients who are faced with significant side effects, toxicities, and complications of transplant.

Sexuality, Menopausal Symptoms, and Quality of Life in Premenopausal Women in the First Year Following Hematopoietic Cell Transplantation

PURPOSE/OBJECTIVES To describe sexuality, menopausal symptoms, and quality of life (QOL) in premenopausal women in the first year following hematopoietic cell transplantation (HCT)
. DESIGN One-year prospective longitudinal study. SETTING Stanford University Medical Center in California.
. SAMPLE 63 premenopausal female recipients of HCT with a mean age of 34.5 years. METHODS Three instruments were used. MAIN RESEARCH VARIABLES Sexuality, menopausal symptoms, and QOL
. FINDINGS At one year post-HCT, women reported absent to low desire and arousal, adequate lubrication less than half of the time, absent or rare orgasm, pain during vaginal penetration more than half the time, and dissatisfaction with overall sex life. Women also reported moderate to severe vasomotor symptoms, including hot flashes, night sweats, and sweating. Twenty-one women were avoiding sexual activity, and 25 women were not sexually active. Mean QOL scores significantly increased (p = 0.028) in the first year, signifying an improvement in QOL. Variables predictive of improved QOL at one year post-HCT include decreased psychosocial and physical symptoms, sexual satisfaction, and pre-HCT QOL score
. CONCLUSIONS One year post-HCT, women reported sexual dysfunction, sexual dissatisfaction, and menopausal symptoms, which negatively affect QOL. IMPLICATIONS FOR NURSING Nurses and other healthcare providers working with recipients of HCT can provide anticipatory guidance on potential changes in sexuality and menopausal symptoms to facilitate adaptation by reducing discordance between expectations and new realities
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Intensive Care Utilization for Hematopoietic Cell Transplant Recipients.

Blood and marrow transplantation (BMT) is a potentially curative therapy for a number of malignant and nonmalignant diseases. Multiple variables, including age, comorbid conditions, disease, disease stage, prior therapies, degree of donor-recipient matching, type of transplantation, and dose intensity of the preparative regimen, affect both morbidity and mortality. Despite tremendous gains in supportive care, BMT remains a high-risk medical therapy. A critically ill BMT recipient may require transfer to an intensive care unit (ICU) and the specialized medical and nursing care that can be provided, such as mechanical ventilation and vasopressor support. Mortality for BMT recipients requiring care in an ICU is high. This paper will describe the experience of the Stanford Blood and Marrow Transplant Program in developing and implementing guidelines to maximize the benefit of intensive care for critically ill BMT recipients.

Long-term implications of cancer for work and financial wellbeing: Evidence from autologous hematopoietic cell transplantation (HCT) survivors

OBJECTIVE Little is known about how long-term cancer survivors adapt in the realm of work and finances, and whether there are differences in these adaptations based on overall health status. We hypothesize that survivors with better health-related quality of life (HQL) have better work and financial outcomes. STUDY DESIGN Cross-sectional study with 200 adult recipients of autologous hematopoietic cell transplantation (HCT) 3-26 years after transplant using self-administered questionnaires and medical records extraction. MAIN OUTCOME MEASURES Questionnaires assessed work status, financial satisfaction, and perceived improvements in financial status since transplant. RESULTS Nearly half the survivors were employed (37.2% full-time, 8.7% part-time); 37.2% had retired. Higher scores on the functional HQL were linked to a lower relative risk of having retired (RRR 0.85, CI 0.75-0.98) and of being neither in the workforce nor retired (RRR 0.84, CI 0.72-0.99) compared with working full-time. Higher functional HQL also related to higher financial satisfaction (b 0.06, CI 0.01-0.10) and increased odds of perceived improvements in ones financial situation since transplant (OR 1.15, CI 1.04-1.17). Patients receiving HCT at age ≥60 were more likely than counterparts receiving HCT at age 18-39 to work part-time (RRR 18.24, 95% CI 1.19-280.24) and to have retired (Model 1 RRR 579.14, 95% CI 49.53-6771.54) than to be working full-time. CONCLUSIONS Survivors with poorer HQL may be at risk for overall poorer work and financial adaptation. Interventions targeting this group and specifically focusing on re-integration into the world of paid work should be considered.

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.