Related Experiment Video
Updated: Feb 26, 2026
A Precision Medicine Tool for Measurement and Monitoring of Hemoglobin S in Sickle Cell Disease Patients Receiving Transfusion Therapy
Proponent or collaborative: Physician perspectives and approaches to disease modifying therapies in sickle cell
Nitya Bakshi1,2,3,4, Cynthia B Sinha4, Diana Ross1,2,4
1Division of Pediatric Hematology-Oncology, Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, United States of America.
Insights
Physician decision-making for sickle cell disease (SCD) therapies involves two approaches: collaborative, emphasizing informed patient choice, and proponent, advocating a specific treatment plan. Understanding these physician perspectives can improve shared decision-making for SCD patients.
Area of Science:
- Hematology
- Medical Ethics
- Qualitative Research
Background:
- Sickle cell disease (SCD) disproportionately affects minority populations.
- Existing disease-modifying therapies include hydroxyurea (HU), bone marrow transplantation (BMT), and chronic blood transfusion (CBT).
- Treatment decisions are complex due to varying efficacy, side effects, costs, and patient burden, with limited research on physician perspectives.
Purpose of the Study:
- To explore physician decision-making approaches for disease-modifying therapies in SCD.
- To understand physician perceptions of patient needs in treatment decision-making.
- To identify factors influencing physician treatment recommendations and decision-making processes.
Main Methods:
- Qualitative study involving 36 in-depth interviews with SCD expert physicians.
- Analysis of physician interviews focusing on their approaches to discussing treatment options with patients.
- Identification and characterization of distinct physician narrative approaches to decision-making.
Main Results:
- Two primary physician approaches were identified: Collaborative Approach (CA) and Proponent Approach (PA).
- CA emphasizes comprehensive discussion of all options for informed patient decisions.
- PA involves advocating a predetermined plan to guide patient acceptance.
- Physician approaches are influenced by patient, disease, decision, physician, and institutional factors.
Conclusions:
- Physician decision-making styles in SCD vary, impacting patient engagement.
- The Collaborative Approach aligns with fostering informed patient choice and shared decision-making.
- Developing systems to enhance patient engagement is crucial for effective shared decision-making in SCD therapy selection.
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.
More Related Videos
08:23Characterization of Sickling During Controlled Automated Deoxygenation with Oxygen Gradient Ektacytometry
Published on: November 5, 2019
05:23Continuous Manual Exchange Transfusion for Patients with Sickle Cell Disease: An Efficient Method to Avoid Iron Overload
Published on: March 14, 2017
Related Concept Videos
01:49Multiple Allele Traits
01:22iPS Cell Differentiation
01:15Methods of Documentation VI: Case Management Model
For example, a patient with a chronic...
01:29Therapeutic Drug Monitoring: Affecting Factors
01:29Cardiomyopathy V: Interprofessional Care
01:29Pharmacogenetic Phenotypes: Alterations in Pharmacokinetics, Drug Targets and Biologic Milieu