Proponent or collaborative: Physician perspectives and approaches to disease modifying therapies in sickle cell disease

Abstract

Sickle cell disease (SCD) is an inherited blood disorder that primarily affects African-American and other ethnic minority populations. There are three available disease-modifying therapies for sickle cell disease: hydroxyurea (HU), bone marrow transplantation (BMT), and chronic blood transfusion (CBT). Since these treatments vary in their therapeutic intent, efficacy in preventing progression of the disease, short and long-term adverse effects, costs and patient burden, the decision-making process regarding these therapies is complicated for both the patient and healthcare provider. While previous research has focused on the patient perspective of treatment-related decision making, there is a paucity of research investigating the physician perspective of treatment-related decision making. We conducted a qualitative study with physicians who were experts in the field of SCD. Interviews focused on physician perceptions of patient decisional needs as well as physicians’ approach to decision making regarding disease-modifying therapies in SCD. Thirty-six physician interviews were analyzed, with a focus on their perspectives regarding available treatment options and on how they approach decision making with patients. We identified two narrative approaches. The Collaborative approach (CA) was characterized by emphasizing the need to discuss all possible treatment options to ensure that the patient and/or family was equipped to make an informed decision. The Proponent approach (PA) was characterized by strongly advocating a pre-determined treatment plan and providing patients/families with information, with the objective of convincing them to accept the treatment. An interplay of patient-related and disease-related factors, decision type and physician-related factors, as well as institutional frameworks, influenced physician perspectives on treatment options and decision making regarding these therapies. These findings point to the potential value of developing systems to foster patient engagement as a way of facilitating shared decision making.

Paediatric haematologists’ attitudes regarding haematopoietic cell transplantation as treatment for sickle cell disease

“…Another reason may be the increased availability of clinical drug trials and other investigational therapies for SCD at University‐based practices. In some cases, rather than discussing HCT as a treatment option, in a child with minimal SCD‐related complications, traditional therapy with hydroxyurea or enrolment in clinical drug trials may be preferred over referral for HCT (Bakshi et al , ). Additionally, with gene editing therapies also on the horizon, University‐based providers, who tend to have greater access to advanced therapies, may prefer to await the opportunity to potentially refer a patient for gene editing in the future, rather than to have them undergo HCT (Ribeil et al , ; Bueren et al , ; Ikawa et al , ).…”

“…Sociocultural factors, both patient-and provider-related, may also contribute to this phenomenon (Gluckman et al, 2017). Studies assessing medical decision-makers' role in this trend show that parents of children with SCD and adult patients affected by the disease are willing to accept relatively high risk of mortality to achieve cure of the disease (Van Besien et al, 2001;Hansbury et al, 2012;Meier et al, 2015). Research on clinicians' perspectives of HCT as treatment for SCD suggests a lower tolerance of risk in order to achieve cure among the healthcare providers who treat this population (Van Besien et al, 2001;Bakshi et al, 2017).…”

The Construction of Sickle Cell Disorder in Saudi Arabia: The Invisible Disease

“…Individuals with haemoglobin disorders, such as SCD, experience a wide range of complications that have severe implications, not only for the individuals themselves, but also for their families (Anie, 2005;Bakshi et al, 2017;Brown, Weisberg, & Sledge, 2016). Thus, multidisciplinary and comprehensive interventions are considered vital in order to effectively manage SCD and its complications.…”

“…It has also been found that physicians have varied approaches to decision making regarding disease modifying therapy for SCD (Bakshi et al, 2017). The research of these authors identified two approaches the first of which was termed collaborative where all treatment options were discussed and patient and family were equipped to make informed decisions.…”

“…Doctor preferences and institutional practices were key determining factors in approach (Bakshi et al, 2017). Elsewhere it is argued that more research with people with SCD should positively address misperceptions and enhance opportunities to ask questions and to consider treatment benefits and risks (Strong et al, 2017).…”