Hoping for a normal life: Decision-making on hematopoietic stem cell transplantation by patients with a hemoglobinopathy and their caregivers.

BACKGROUND: To provide insight into the perspectives of children and young adults with transfusion-dependent thalassemia and sickle cell disease and their caregivers regarding the decision for hematopoietic stem cell transplantation (HSCT). PROCEDURE: A qualitative longitudinal multicenter study. Data collection consisted of 40 audio-recorded conversations between physicians and families and 77 interviews with patients and/or caregivers related to 27 unique cases, collected at different time points throughout the decision-making process. RESULTS: Conversations and interviews revealed "hoping for a normal life" as an overarching theme, consisting of four main topics: (i) "Building a frame of reference" refers to a process where patients or families try to obtain comprehensive information on HSCT and translate this to their situation to decide. (ii) "Balancing between loss and benefit" reports the process of considering the advantages and disadvantages of continuing with supportive care to treat their disease versus choosing HSCT. (iii) "Experiencing the impact of HSCT" describes the impactfull experience of the HSCT period by those who chose HSCT. (iv) "Balancing again" refers to reflecting on the decision made. CONCLUSIONS: The hope for a normal life guided the decision-making process, described as a constant balance between the impact of the disease and HSCT. A structured approach to explore patients' and caregivers' perspectives on HSCT decision-making is needed, where specifically discussing the impact of the disease and hope for a normal life need to be integrated in the process.

The value of using patient-reported outcomes for health screening during long-term follow-up after paediatric stem cell transplantation for nonmalignant diseases.

INTRODUCTION: The assessment of using patient-reported outcomes (PROs) within comprehensive care follow-up programmes, specifically focused on health screening, remains largely unexplored. PROs were implemented in our late effects and comprehensive care programme after paediatric hematopoietic stem cell transplantation (HSCT) for nonmalignant diseases. The programme focuses solely on screening of physical and mental health and on discussing PROs during the consultation. METHODS: The primary method of this study was semistructured interviews to explore the perspective of both patients and healthcare providers' (HCP) on the use of PROs, which were thematically analyzed. Additionally, an explorative quantitative approach with patient-reported experience measures (PREMS) was used, with a pretest-posttest design, to assess whether the use of PROs was accompanied by more patient-centred care. RESULTS: From the patient-interviews (N = 15) four themes were extracted: use of PROs (1) help to discuss topics; (2) make the patients feel understood; (3) create a moment of self-reflection; and (4) make consultations more efficient. Pre- and postimplementation analysis of PREMs (N = 40) did not show significant differences in terms of patient-centeredness. CONCLUSION: Our results demonstrate the added value of integrating PROs for health screening purposes within the long-term follow-up programme after paediatric HSCT, as perceived by both patient and HCP. With the active use of PROs, patients are stimulated to consciously assess their health status. PATIENT CONTRIBUTION: This study included patients as participants. Caregivers were approached if patients were below a certain age. Additionally, preliminary results were shared with all patients (including nonparticipants) during a patient conference day.

Physical, Mental, and Social Health of Adult Patients with Sickle Cell Disease after Allogeneic Hematopoietic Stem Cell Transplantation: A Mixed-Methods Study.

Patients with sickle cell disease (SCD) experience a considerable physical and psychosocial disease burden. In recent years, the application of allogeneic hematopoietic stem cell transplantation (HSCT) to treat adults with SCD has increased. A thorough understanding of patients' physical, mental, and social health before and after cure is needed to meet the needs of this growing group of patients. We aimed to explore the perspectives of adult SCD patients on the changes in their experienced health and personal life goals after being cured. A mixed-methods approach was used, comprising a semistructured interview and a set of 9 Patient Reported Outcomes Measurement Information System (PROMIS) measures. Adult SCD patients who underwent HSCT at least 1 year earlier were eligible to participate in the study. Interviews were thematically analyzed using MAXQDA software. PROMIS T scores were compared with reference scores of the general population using SPSS Statistics. Ten patients participated in the study; their median age was 29.5 years (range, 19 to 49 years), and their median time since HSCT was 2.7 years (range, 1.0 to 3.5 years). Themes from the interviews were (1) pain/living pain free, (2) physical well-being, (3) mental well-being, (4) perspective/ outlook, (5) education/work, (6) family/friends, and (7) activities/participation. Following the PROMIS framework, we described these themes in a narrative synthesis according to health domain and categorized in 4 chronological time phases: before HSCT, first year post-transplantation, current situation, and future expectations. Physical health improved greatly, but transplantation-related toxicity, ongoing pain from avascular osteonecrosis, and fatigue negatively impacted quality of life in some patients. Furthermore, emotional struggles during the post-transplantation period were common, and patients expressed a need for psychological help. Patients reported improvements in social health and the ability to pursue personal life goals. The mean T scores of all PROMIS measures fell within the normal symptom limits compared with reference data of the general population, although, large variations were observed among the participants, matching our qualitative findings. In general, adult SCD patients experienced improved physical, mental, and social health after cure by HSCT and were able to pursue personal life goals. Yet they found themselves confronted with a new and unfamiliar reality that brought different challenges. Pain due to irreversible avascular osteonecrosis continued to have a negative impact. Clinicians should aim to help patients have realistic expectations before transplantation and offer timely psychological care.

Exploring the long-term psychosocial impact of paediatric haematopoietic stem cell transplantation for nonmalignant diseases.

INTRODUCTION: An understanding of the long-term psychosocial impact of paediatric haematopoietic stem cell transplantation (HSCT) for nonmalignant diseases is needed to optimize pre-HSCT counselling, supportive care and long-term follow-up programmes after HSCT for this group of patients and caregivers. METHODS: This qualitative study included 14 patients who underwent transplantation for a nonmalignant disease during childhood. In-depth interviews were held online to explore patients' perspectives on the long-term psychosocial impact of HSCT on their lives. The results were analysed based on the Grounded Theory approach. RESULTS: Patients' median age at the time of the interview was 19 years (range: 14-49), and the median years after HSCT was 12 years (range: 3-33). Four main themes were identified: (1) doing okay, (2) experiencing persistent involvement with healthcare services, (3) influence on relationships with loved ones and (4) impact on the patient's life course. Subthemes extracted were doing okay, feeling of being cured, health limitations, sense of vulnerability, ongoing connection to the hospital, acceptance, friendship, family relations, development of own identity, not taking life for granted, social development, impact on (school) career and thinking about the future. CONCLUSIONS: Patients reported active coping strategies and resilience after this high-impact treatment. The data highlight the need for patient-adjusted supportive care, indicating more need for supportive care in the long-term outpatient clinic. PATIENT CONTRIBUTION: This study included patients as participants. Caregivers were approached if patients were below a certain age. Additionally, preliminary results were presented during a patient conference day.

Safe transfer of pediatric patients from hematopoietic stem cell transplant unit into the pediatric intensive care unit: views of nurses and physicians.

Sufficient communication between hematopoietic stem cell transplantation (HSCT) and pediatric intensive care unit (PICU) teams is pivotal for a successful advanced support in the PICU for HSCT-related complications. We evaluated perceived communication and barriers between both teams with the aim of providing recommendations for improvement. In this cross-sectional survey, a self-designed online questionnaire was distributed among ESPNIC and EBMT members. Data were analyzed using descriptive statistics. Over half of HSCT respondents employed a transfer indication protocol and patient assessment tool, but less structured checklist prior to patient transfer. Nearly all PICU respondents perceived this checklist as improvement for communication. Most HSCT and PICU physicians have daily rounds upon patient transfer while this is mostly missing between nursing teams. Half of both HSCT and PICU nurses indicated that HSCT training for PICU nurses could improve communication and patient transfer. Most respondents indicated that structured meetings between HSCT and PICU nurses could improve communication. Overall there is good communication between HSCT and PICU units, although barriers were noted between members of both teams. Based on our findings, we recommend use of a structured and specific checklist by HSCT teams, HSCT training for PICU personnel, and structured meetings between HSCT and PICU nurses.

Discussing Sexual Health with Adolescent and Young Adults with Cancer: a Qualitative Study Among Healthcare Providers.

Sexual health is an important aspect of quality-of-life for adolescent and young adult (AYA) patients with cancer. AYAs report a need for information about sexual health but experience a lack of communication with their healthcare provider. It is known that sexual health is a difficult theme for healthcare providers to discuss. This study aims to gain insight into perspectives of healthcare providers in facilitating AYAs' needs regarding sexual health. A qualitative study was performed. Six doctors and eight nurses from eight Dutch hospitals, specialized in AYA care, participated in semi-structured interviews. All interviews were audio-recorded, transcribed, and analyzed using a thematic analysis approach. Interviews with the healthcare providers revealed five themes concerning the discussion of sexual health: (1) being responsible for bringing up the topic of sexual health, (2) finding optimal timing to discuss sexual health, (3) acquiring knowledge to enable discussion of sexual health, (4) facilitating communication about sexual health, and (5) providing informative material for AYAs. To facilitate discussing sexual health, clear defined responsibilities within the team and sufficient knowledge are important. Self-report questionnaires, material to hand out, a checklist for healthcare providers, and knowledge of different communication strategies are helpful when discussing sexual health. By applying such means, communication with AYAs might be improved.

How to Facilitate Decision-Making for Hematopoietic Stem Cell Transplantation in Patients With Hemoglobinopathies. The Perspectives of Healthcare Professionals.

Hematopoietic stem cell transplantation decision-making for hemoglobinopathy patients is a complex process, and it remains difficult for health care professionals to decide whether and when a hematopoietic stem cell transplantation should be offered. Gaining insight into health care professionals' considerations is required to understand and optimize this decision-making process. A qualitative interview study using semi-structured interviews with eighteen health care professionals. Data were thematically analyzed. Two main themes emerged from the data: (1) Experiencing the influence of a frame of reference and (2) Feeling responsible for a guided decision-making. The frame of reference, meaning the health care professionals' knowledge and experiences regarding hematopoietic stem cell transplantation, influenced the guided decision-making process. Subsequently, three subthemes evolved from the second theme: (a) weighing up disease severity against possible complications, (b) making an effort to inform, and (c) supporting the best fitting decision for the individual patient. The health care professionals' frame of reference determined the hematopoietic stem cell transplantation decision-making process. This demands reflection on the health care professionals' own frame of reference and its influence on decision-making. Furthermore, reflection on the frame of reference is needed by exchange of knowledge and experiences between referring and referred-to healthcare professionals in an open and two-way direction. The transplantation teams have a responsibility of keeping the frame of reference of their referring colleagues up to date and referring health care professionals should share their feelings regarding hematopoietic stem cell transplantation. To guide patients, a shared decision-making approach is supportive, in which eliciting the patients' preferences is highly important. Health care professionals can refine the decision-making process by guiding patients in eliciting their preferences and including these in the decision.

Specialized Pediatric Palliative Care Services in Pediatric Hematopoietic Stem Cell Transplant Centers.

Hematopoietic stem cell transplantation (HSCT) is widely used in pediatric patients as a successful curative therapy for life-threatening conditions. The treatment is intensive, with risks of serious complications and lethal outcomes. This study aimed to provide insight into current data on the place and cause of death of transplanted children, the available specialized pediatric palliative care services (SPPCS), and what services HSCT professionals feel the SPPCS team should provide. First, a retrospective database analysis on the place and cause of death of transplanted pediatric HSCT patients was performed. Second, a survey was performed addressing the availability of and views on SPPCS among HSCT professionals. Database analysis included 233 patients of whom the majority died in-hospital: 38% in the pediatric intensive care unit, 20% in HSCT units, 17% in other hospitals, and 14% at home or in a hospice (11% unknown). For the survey, 98 HSCT professionals from 54 centers participated. Nearly all professionals indicated that HSCT patients should have access to SPPCS, especially for pain management, but less than half routinely referred to this service at an early stage. We, therefore, advise HSCT teams to integrate advance care planning for pediatric HSCT patients actively, ideally from diagnosis, to ensure timely SPPCS involvement and maximize end-of-life preparation.

Decision making for hematopoietic stem cell transplantation in pediatric, adolescent, and young adult patients with a hemoglobinopathy-Shared or not?

BACKGROUND: Hematopoietic stem cell transplantation (HSCT) offers an established curative option for sickle cell disease (SCD) and thalassemia patients but is associated with significant risks. Decision making is a complex process and shared decision making (SDM) could be a fitting approach in case of such preference-sensitive decisions. This study investigated what level of SDM is used in conversations with hemoglobinopathy patients and/or their caregivers considering HSCT as a curative treatment option. METHODS: Longitudinal, descriptive study using the Observing-Patient-Involvement-in-Decision-Making scale (OPTION5 ) scale to determine the level of SDM in conversations with 26 hemoglobinopathy patients and/or their caregivers. RESULTS: The total mean OPTION5 score was 43%, which is a moderate SDM approach. There was no difference between conversations with thalassemia patients and SCD patients. Conversations needing an interpreter scored worse than nontranslated conversations. The best scoring OPTION5 item was item 3: "informing about the various treatment options" (mean score 2.3 on scale 0-4). For OPTION5 item 4: "eliciting patients' preferences" a more skilled effort was measured for SCD patients compared to thalassemia patients. CONCLUSIONS: The mean OPTION5 score of "moderate" was achieved mainly by giving information on available options, which is primarily a one-way communication. The SDM process can be improved by actively inviting patients to deliberate about options and including their elicited preferences in decision making.

Parental experiences in end-of-life decision-making in allogeneic pediatric stem cell transplantation: "Have I been a good parent?"

BACKGROUND: In pediatric hematopoietic stem cell transplantation (HSCT), the end-of-life (EOL) phase and the loss of the child is often characterized by a sudden deterioration of the child following a period of intensive curative treatment. This demands a fast transition for parents. Therefore, an understanding of the parents' perspective on decision-making in such a complex situation is needed. This study aims to gain insight in parental experiences in EOL decision-making in allogeneic pediatric HSCT. METHODS: A qualitative descriptive study was performed among parents of eight families. Data were thematically analyzed. RESULTS: All parents were aware of their child's deterioration. Six families were confronted with a rapid deterioration, while two families experienced a gradual realization that their child would not survive. Parental EOL decision-making in pediatric HSCT shows a reflective perspective on the meaning of parenthood in EOL decision-making. Two central themes were identified: "survival-oriented decision-making" and "struggling with doubts in hindsight." Six subthemes within the first theme described the parents' goal of doing everything to achieve survival. DISCUSSION: Parents experienced EOL decision-making mainly as a process guided by health care professionals (HCPs) based on the child's condition and treatment possibilities. The decision-making is characterized by following opportunities and focusing on hope for cure. In hindsight parents experienced doubts about treatment steps and their child's suffering. HCPs can strengthen the parental role by an early integration of palliative care, providing timely support to parents in the process of imminent loss. Advance care planning can be used to support communication processes, defining preferences for future care.