ALBERT

Description: Background Pain is the hallmark of sickle cell disease (SCD) but burden of pain is underestimated when measured using health care visits for vaso-occlusive crisis. In the PiSCES study adult patients reported pain on 〉 50% of diary days but sought care on only 3.5 % of diary days. Accurate assessment of the burden of pain and related morbidity is crucial in clinical care and research studies in SCD. Paper based pain diaries for assessing daily pain are limited by recall bias, errors, inflated retrospective reports and falsely high compliance due to backfilling of entries. Electronic pain diaries facilitate real-time data capture, are convenient, prevent backfilling, maximize compliance and facilitate data management. They have been used in children with arthritis, cancer, abdominal and musculoskeletal pain but no validated instrument is available for use in children with SCD. Objective To develop, establish the face and content validity, and usability of a novel web-based multidimensional electronic pain diary for children and adolescents with SCD. Methods Needs assessment: Pediatric subjects in a pilot SCD pain intensity diary study participated in qualitative interviews to assess their preferences regarding an electronic pain diary. Instrument development: Items for the pain diary were adapted for SCD from “e-Ouch”(c), an electronic pain diary validated for use in children with arthritis. Items assess pain intensity, duration, interference with daily tasks, sleep, fatigue, precipitating factors, pain relieving treatments and response to treatments using the Numerical Rating Scale (0-10). We created a web-based pain diary that can be accessed via a secure website using a smartphone or computer. Face validity: Experts in SCD, pain and psychometrics rated the items on a 5 point Likert scale for content, language, clinical relevance, comprehensiveness of answer choices and likely feasibility and acceptability in children with SCD. Two iterative cycles of expert review were conducted and were used for modification of items. Content validity: Using items with established face validity, two iterative cycles of testing (n=5 each) with paper screenshots of questions using semi-structured cognitive interviewing techniques were done in pediatric patients age 15-22 with SCD. Preliminary usability testing: Participants age 9-21(n=5) pilot tested the web-based electronic pain diary on a computer, smartphone and tablet. They were asked to recall their current pain and pain in the prior 12 hours while answering the diary questions. The usability testing was followed by semi-structured interviews. Results Needs assessment: Patients indicated that electronic monitoring of pain could facilitate coordination of care, communication with providers and early intervention and that twice daily electronic documentation of pain would not pose an unacceptable burden. Face validity: Items were reviewed by 15 experts in the first iterative cycle and 12 experts in the second iterative cycle and were modified for language, content and relevance; 2 items were deleted and 1 item was added. Content Validity: During the first iterative cycle, participants identified items that were difficult to understand, ambiguous or irrelevant. Number of items was reduced from 18 to 13. During the second iterative cycle, one repetitive item was removed and others minimally modified. To minimize user burden items were redistributed so pain intensity, location, quality and precipitating factors were asked twice daily; effect of pain on sleep was asked in the morning and pain interference with daily activities, mood, school and interactions and clinical management items were asked in the evening. Usability testing: Participants were easily able to navigate between questions, use the 0-10 NRS slider, select affected areas on the body image and select checkbox options and provided positive feedback on the question content and, layout of the diary, ease of its use and preference for accessing it from a smartphone. Conclusions This study established the face and content validity and usability of a web-based multidimensional electronic pain diary developed for use in children with SCD. This instrument can be used to assess pain as a patient reported outcome in clinical trials, to enhance communication in clinical care and as a comprehensive measure of pain phenotype in mechanistic studies. Disclosures: No relevant conflicts of interest to declare.

Description: Background The decision-making process regarding therapeutic options in Sickle Cell Disease (SCD) can be complex. Disease modifying therapies such as hydroxyurea (HU), bone marrow transplantation (BMT) or chronic blood transfusion (CBT) are heterogeneous in their therapeutic intent, efficacy in preventing progression of disease, costs, acute complications, patient burden and long-term side effects. Both healthcare provider and patient-related factors may influence decision-making regarding these treatment options. That a majority of patients with SCD belong to minority underserved populations adds to the complexity of the decision-making in this group of patients. Shared decision making (SDM), a collaborative process that allows patients and their providers to make health care decisions together is preferred since it takes into account the best clinical evidence available, as well as the patient's values and preferences. There is a paucity of data on how physicians approach their conversations with patients regarding therapeutic options. The objective of this study was to determine the perspective of physicians with expertise in SCD and BMT regarding decision making about disease modifying therapy. Methods We enrolled a geographically diverse and nationally representative sample of physicians with expertise in the clinical management of SCD in this mixed methods study. We conducted and analyzed qualitative interviews focused on the physician perspective of patients' values, preferences and decisional needs in decision making regarding disease-modifying therapeutic options in SCD. These interviews were part of a larger study to understand decisional needs of patients with SCD in order to develop a decision aid for the therapeutic options in SCD. Open-ended semi-structured interviews were used to collect data. All interviews were recorded and transcribed verbatim. We used NVivo10 for analysis of qualitative data. We sought to understand the framework of discussion used by physicians for the various therapeutic options. Our analysis concentrated on how physicians engaged in discussions related to therapeutic options to reveal whether physicians facilitate the patient's decision process or intend to convince the patient of a treatment plan. Results We interviewed 37 physicians who were experts in pediatric or adult SCD or BMT. Twenty of the 37 were female. Physician narratives reflected high concern for the well-being of the patient and caregivers. Using content analysis, we categorized the physicians' narratives on a spectrum based on their degree of involvement in guiding decision making regarding disease modifying therapies. Narratives placed on one end of the spectrum were those where the physician frequently discussed patient involvement in the decision-making process. On the other end of the spectrum, we categorized physicians who were explicitly advocating for a particular treatment plan for the patient. Thus, the assumption was that this latter group of physicians entered into physician-patient conversations with a recommended therapeutic plan. We further analyzed the content of this spectrum to reveal what patient attributes influenced the physician's narrative. While all of the physicians in our study were advocates of patient education and active involvement, more than half of the narratives were explicitly promoting a particular therapeutic plan. Our analysis revealed that the degree of physician involvement was influenced by the perception of patient compliance, socio-economic barriers, and the patient's current clinical condition. Physicians who advocated a treatment option were more likely to discuss patient non-compliance and barriers as influencing their treatment decision. Moreover, many of these physicians were less likely to discuss all available treatment options, such as BMT. Conclusions Decision making regarding disease modifying therapy in SCD is complex and the approach of physicians ranges from SDM model to that of a physician advocating for a particular treatment. These data suggest the need for greater awareness of and education about the SDM model for patients and physicians. They provide the rationale for creating of decision support systems regarding disease-modifying therapies for SCD. Disclosures No relevant conflicts of interest to declare.

Description: Background:Bone marrow transplantation (BMT) is currently the only curative treatment option available for patients with sickle cell disease (SCD). There are very limited data available on the patients and caregivers perspective on decision making for BMT and their overall experiences with the BMT process. The objective of this study was to determine patients and caregivers knowledge of BMT, the factors that influenced families to consider BMT as a treatment option and their experiences during and after transplantation. Methods:At Children's Healthcare of Atlanta, we conducted 22 qualitative interviews involving patient-caregiver dyads using a semi-structured interview guide. The questions focused on patient and caregiver decision-making and their experience with bone marrow transplant for SCD. All interviews were recorded and transcribed verbatim. Transcripts were then double-coded. Content analysis was performed using NVivo 10. Results: Eleven patients (n=6; Females) and 11 caregivers (n=10, Mothers) participated. At the time of interview, patient's age range was 11-32 years with a median value of 20 years; at the time of transplant, patient's age range was 6 to 17 years with a median value of 11 years. Time from transplant ranged from 0-17 years with a median value of 2 years. Analysis revealed that most patients' and caregivers' were presented with BMT as a treatment option based on the severity of patient's clinical situations. Six participants, patients and caregivers combined, described minimal knowledge and understanding of the BMT process and/or the associated risks/benefits involved with transplant before consultation with the BMT team. Both patients and caregivers reported that the decision to move forward with BMT was strongly influenced by the progression of sickle cell related complications. All patients were either suffering from chronic pain or had been on chronic transfusions for several years. Given the effects of BMT on the entire family, 7 patients and 5 caregivers reported the need to involve the entire family in the decision-making process. Other factors that played a significant role in the decision-making process for patients and caregivers included: information and education received from the BMT team, having an HLA-matched sibling or unrelated donor, and spirituality. A few patients and caregivers stated that hearing from other families would have provided them a more realistic understanding of the transplant process and further aided in their decision-making. Of the 7 patients and caregivers asked about fertility issues and its impact on transplant decision making, 3 patients, and 3 caregivers conveyed that it was the "least of their concerns" and did not feel the need to proceed with fertility preservation. One caregiver voiced concerns about fertility issues, but after consultation with a fertility specialist decided to proceed with BMT. About 95% of patients and caregivers described transplant day as a "re-birth day" or "new birthday". Most patients conveyed that having to deal with chemotherapy-related side effects, such as hair loss, the need for nasogastric tubes, and being isolated from family and friends was all "worth it" because they no longer have sickle cell disease. For 8 caregivers, prayers and family support played a prominent role in helping to cope with the fears and stress involved with BMT. Eight patients indicated that prayers and distractions such as watching movies, playing video games and doing school work helped with coping. Despite having to deal with the arduous transplant process and transplant-related complications, patients and caregivers alike were satisfied with their decision and expressed no decisional regret.While this study included some patients who had severe complications including chronic GVHD, we did not interview caregivers of any patient who had died following BMT. Conclusion: Decision making for bone marrow transplant as a treatment option for those with sickle cell disease is a complex process. Most patients and caregivers have limited knowledge about bone marrow transplant being a curative treatment for sickle cell disease, and the need to educate patients and families is crucial. Gaining an understanding of factors that influence decision making for this population and their experiences with the transplant process will generate crucial insights that will impact the care of future patients and research studies. Disclosures No relevant conflicts of interest to declare.

Description: Background Medical decisions regarding disease modifying therapy for sickle cell disease (SCD) such as chronic blood transfusion, hydroxyurea, and bone marrow transplantation are complex and have many short and long term implications for the patient's health. Shared decision-making, a collaborative process that allows patients and healthcare providers to make health care decisions collaboratively taking into account the best clinical evidence available as well as the patient's values and preferences, is considered the preferred process in complex medical decisions. There is a paucity of information regarding perspectives of patients with SCD and their caregivers regarding shared decision-making. The objective of this study was to determine the perspective of patients and their caregivers regarding shared decision making in disease modifying therapy for SCD. Methods We conducted qualitative interviews of patients with SCD as well as their caregivers from a geographically diverse population recruited from attendees at regional and national conferences for SCD. A semi-structured open-ended interview guide was used to collect data. Interview guide focussed on determining the expectations of patients and caregivers in their interaction with their physician and their involvement in decision making regarding disease modifying therapy. Interviews lasted 30-60 minutes and were recorded. Audio recordings were transcribed verbatim. Transcripts were coded using qualitative content analysis with NVivo 10. Results Participants were African American, average age was 40 (SD 10.8), 18 of 19 were female, one had a high school degree, eight had some college, and 10 had either a bachelor's or graduate degree.Qualitative analyses yielded the following themes: 1. Patients prefer decision making to occur as a collaborative process between a patient or caregiver and the physician. They indicated that physician and patient each brings their own expertise to the table when discussing treatment. They strongly endorsed that patients should make an informed decision based on ability to comply and personal goals, preferences, and beliefs. 2. Patients prefer that the physician provide information about SCD, including complications and expected long-term outcomes based on the literature as well as personal knowledge of the patient. Patients indicated a desire to receive information in an unbiased fashion. Participants felt that it is the physician's responsibility to provide verbal and written information as well as referral to websites of value in learning. 3. Participants felt that physicians should be aware of their patient's medical history and personal preferences for care. 3. Patients prefer to receive detailed information on side effects and expectations of the impact of treatment on the patient based on the literature and physician experience. 4. Patients would like for the physician to listen to the patient and attempt to understand the patient perspective regarding self-care. 5. Patients have the responsibility to understand SCD and its complications, including expected long-term outcomes of SCD. Participants also underlined the importance of patients making an effort to understand the treatment being offered, including intended purpose, expected outcome, risks, and benefits. Participants felt that patients need time to do their own research and learning before making a decision. 6. Multiple sources for learning about SCD and treatment options. These include education by the physician and asking questions, talking to other patients, doing research on the internet, and personal knowledge of their medical history to including the way their body responded to past treatment. 7. Nurses, and social workers can have a supportive role in decision-making, and can address past medical history and provide guidance regarding socioeconomic barriers to care. 8. Extended family, unless living with the patient, plays a supportive role but is not part of the decision-making team. Conclusion The perspective of patients and their caregivers suggests strong support for shared decision making collaboratively with the physician, supported by high quality information and based on the patient's personal values and preferences. These data provide a rationale for developing and implementing measures to facilitate shared decision making for disease modifying therapy for SCD. Disclosures No relevant conflicts of interest to declare.

Description: Background Sickle cell disease (SCD) is associated with substantial morbidity, and premature mortality. Hydroxyurea (Hu), chronic blood transfusion and hematopoietic stem cell transplantation (HCT) can modify the course of this disease, reduce complications, and improve survival. These interventions are diverse in their therapeutic intent, but are all associated with substantial burden of care, signfiicant side effects and the potential for late complications. The complexity inherent in making decisons about these disease modifying therapies is compounded by the socioeconomic disadvantage experienced by this largely minority, underserved population. That despite proven efficacy in clinical trials, uptake and adherence with HU remains poor, and that only a small proportion of eligible patients undergo HCT underscores the difficulty in making these decisions. The objective of this study was to use a theory based systematic approach to develop, implement and test a web based decision aid to support patients and caregivers in shared decision making regarding disease modifying therapies for SCD.The theoretical basis for this project is the Ottawa decision support framework( ODSF), an evidence-based, practical, mid-range theory for guiding patients making health or social decisions which uses a three-step process to assess client and practitioner determinants of decisions to identify decision support needs; provide decision support tailored to client needs and evaluate the decision making process and outcomes. Methods Following the ODSF, we conducted qualitative interviews of patients with SCD, their caregivers, policymakers, community advocates and healthcare providers and other stakeholders. Subjects were recruited at multiple regional and national SCD conferences which draw a mix of patients and professional audience. Interviews lasted 30-40 minutes and were transcribed verbatim, transcripts were coded using QSR NVivo 10 and analyzed using qualitative mixed methods. Themes from qualitative interviews were incorporated into the each of the following phases of the project: Phase 1: Qualitative interviews for needs assessment to facilitate the description of the participants' experiences in seeking information about and making decisions related to SCD and utilizing both open and closed-ended questions. Phase 2: Data synthesis, and construction of a storyboard, draft content, draft design and format for the decision aid. Phase 3: Alpha testing for quality and error correction. Extensive modifications were carried out systematically incorporating the recommendations received. Phase 4: Iterative cylces of Beta testing for feasibility, comprehensibility and usability with qualitative interviews and observations and incorporated recommendations. Phase5: Peer-review of the finalized decision aid by stakeholders who had not participated in the development of the instrument and had not previously examined the website. Results Needs assessment qualitative interviews with 205 individuals yielded information on decisional needs, preferences regarding content, presentation and ease of use as well as a distinct preference to learn from the experience of other patients and guided the devlopemnt of the decision aid. Alpha testing for quality and error correction was conducted with 51 patients / family members, clinicians, health educators and policy makers. Beta testing for feasiblity, comprehensibilty, and usability was completed by a total 111 participants. Peer review by 60 providers, patients and stakeholders of the final decision aid (www.sickleoptions.org) indicated a high level of satisfaction with the content, presentation, ease of use, use of graphics and the use of patient testimonials. We have enrolled and are gathering data on 120 subjects in a randomized clinical trial to evaluate the impact of the decion aid on clinical decision making. Conclusions This stiudy provides empirical evidence about the successful process of creating, implementing and testing a web based decision aid for patients to guide shared decision making in disease modifying therapy for SCD. It provides evidence of favorable patient and physician perceptions about the comprehensivility and usability of the decision aid. Results of an ongoing randomized clinical trial will generate additional information about the impact of the decision on clinical decision making. Disclosures No relevant conflicts of interest to declare.