Primary caregiver decision‐making in hematopoietic cell transplantation and gene therapy for sickle cell disease

Abstract

Background: Improved outcomes and the availability of clinical trials of hematopoietic cell transplantation (HCT) from alternate donors and genetically modified autologous hematopoietic progenitor cells have expanded the applicability of HCT for sickle cell disease (SCD). To understand the perspective of primary caregivers exploring HCT in the current milieu, we asked the research question "What motivates primary caregivers to decide to consider HCT and to seek, and to attend, an HCT consultation?" Procedures: We conducted qualitative interviews with primary caregivers within one week after a consultation for HCT for SCD. Data were analyzed using open and axial coding stages of grounded theory methodology. Results: We interviewed 29 primary caregivers (26 females, age 29 to 64 [median 42] years). Primary caregivers report of SCD complications in their child included at least one in the last year by 23 (82%), few or none by 8 (28%), and pain on ≥3 days a week by 13 (46%) primary caregivers. Qualitative analysis revealed that primary caregivers, (i) learn about curative options through social networks, social media, and the news media; (ii) seek consultation because of their child's diminished quality of life, recent complications, an imminent major medical decision, or anxiety about future severe complications; and (iii) see gene therapy as a new, less invasive, and more acceptable treatment. Conclusion: Primary caregivers of children with SCD learn about HCT through social networks, social and news media, and explore HCT as a means to prevent SCD complications and help their child live a normal life.

A Decision Support Tool for Allogeneic Hematopoietic Stem Cell Transplantation for Children With Sickle Cell Disease: Acceptability and Usability Study

“…Some physicians expressed a desire to see QoL pre-HCT and the anticipated impact of HCT on QoL in the risk prediction model of the decision support tool. Studies of decision-making by patients suggest that patients and families consider QoL important in decision-making about HCT [ 18 , 23 - 27 ]. Recent clinical trials have suggested that HCT can improve QoL after HCT [ 34 - 40 ].…”

“…The availability of novel treatments, as well as the increased willingness of physicians and patients to consider HCT, may contribute to the increasing uptake of this treatment [ 20 - 22 ]. Previous studies have investigated the decision-making process and preferences of patients and families considering HCT [ 18 , 23 - 27 ].…”

Assessing Patient Preferences for Treatment Options for Pediatric Sickle Cell Disease: A Critical Review of Quantitative and Qualitative Studies

“…They report that patients learned about curative options from social media and news. 42 Caregivers sought consultation owing to their child’s diminished quality of life, recent complications, anxiety about future difficulty, or an imminent major medical decision. Gene therapy was seen as a less invasive and more acceptable treatment option as compared to HSCT.…”

Evidence-Based Minireview: In young children with severe sickle cell disease, do the benefits of HLA-identical sibling donor HCT outweigh the risks?

“…However, a par ent may also con sider other trade-offs such as the out comes of HCT in child hood vs the poten tial for mor bid ity and mor tality in adult hood, HRQOL with or with out HCT, and cura tive HCT vs chronic GVHD, infer til ity, car dio vas cu lar risk, and sub se quent malig nancy. 28 How fam i lies resolve these deci sional trade-offs is likely to be highly indi vid u al ized and depen dent on sev eral factors unique to the cir cum stances of the patient and fam ily. To assist the hema tol o gist in the con ver sa tion with the fam ily of a young child with SCD who has an HLA-iden ti cal sib ling, we summa rize the avail able evi dence as answers to crit i cal ques tions that may arise in the minds of patients and their care giv ers as they con tem plate HCT.…”