ALBERT

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 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.