Healthcare providers may struggle with the decision to recommend hematopoietic cell transplantation (HCT) for children with sickle cell disease (SCD). A recent report published in JMIR Formative Research reviewed the acceptability and usability of a newly developed decision-support tool to help guide treatment decisions in this patient population.
“Although the reported HCT survival outcomes for patients with SCD are generally favorable, it may be difficult for healthcare providers to personalize risk factors for an individual patient or have expert knowledge of the field,” wrote the authors, led by Anirudh Veludhandi, of the School of Medicine at Emory University in Atlanta, Georgia. “In addition, although age and type of donor are the most important predictive factors of HCT outcomes, there is no available tool to individualize the risk factors for age, type of donor, type of conditioning, or other factors to provide estimates for an individual patient.”
To address that gap, the researchers developed and tested the Sickle Options Decision Support Tool for Children, a mobile app that calculates survival and risk estimates for pediatric patients undergoing HCT. The tool allows healthcare providers to input certain data about an individual patient. Incorporating published data from a patient registry, the tool then provides personalized estimates on a person’s transplant survival and risks. The researchers hope the tool will eventually help healthcare providers and families make better-informed decisions about treatment. “The tool may help patients weigh the risks and pros and cons in the context of their own values and preferences,” the authors wrote.
To develop the tool, the researchers analyzed registry-based studies from data reported to the Center for International Blood and Marrow Transplant Research, Eurocord, and European Society for Blood and Marrow Transplantation databases. To be included, a study had to have a large, diverse patient sample with respect to age, sex, type of donor, stem cell source, and type of pre-transplant conditioning regimen. The researchers selected three studies to represent the four donor types: HLA antigen–matched sibling, HLA antigen–matched unrelated, haploidentical, and HLA antigen–mismatched unrelated. Analyses examined for overall survival, event-free survival, graft failure, and graft-versus-host disease (GVHD, both acute and chronic).
Incorporating input from healthcare providers, the researchers developed the tool and made changes as needed. Then they recruited 18 physicians with a background in SCD to use the tool on their own phones with a hypothetical patient in mind. The physicians then gave feedback via interviews and surveys, specifically the Ottawa Decision Support Framework and Mobile Health App Usability Questionnaire.
Overall, the physicians rated acceptability and usability of the tool as high. Most physicians indicated that they would use the tool in their own practice. They recommended that the tool be amended to include more background on HCT and to simplify medical terminology to help facilitate physician-patient communication and decision-making. The authors also said they plan to finalize the tool by validating its predictive capabilities against a larger dataset. They also intend to explore incorporating patient-specific measures, such as the HCT-Specific Comorbidity Index and a measurement of quality of life after transplant.
“These data provide a proof-of-concept of the potential acceptability and utility of such a decision support tool for use by hematologists and HCT physicians,” the authors concluded. “To the best of our knowledge, this is the first decision-support tool to provide individualized and age-specific risk and survival estimates for pediatric patients considering HCT.”
