Shared Decision Making or Physician Advocate for a Particular Treatment Option: A Spectrum of Approaches to Decision Making about Disease Modifying Therapies in Sickle Cell Disease

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.