A decision support tool related to use of allogeneic hematopoietic stem cell transplantation (HSCT) in children with sickle cell disease (SCD) was recently developed and described in a report in the journal JMIR Formative Research.
Many disease-modifying therapies exist for SCD, but allogeneic HSCT is an approach with curative intent. However, a patient’s individual risk factors may make the decision of whether to use allogeneic HSCT challenging for the treating physician, according to the researchers who authored the report.
In development of the decision support tool, the researchers examined registry-based studies to develop prediction models for outcomes with allogeneic HSCT. Patient and clinical characteristics and outcomes with HSCT were evaluated from databases of the Center for International Blood and Marrow Transplant Research, Eurocord, and the European Society for Blood and Marrow Transplantation. Included studies each had more than 100 patients involved per donor type and a representation of varied patient demographics and aspects of treatment.
A total of 3 studies were included in model development. These reflected data involving human leukocyte antigen (HLA)-matched siblings, HLA-matched unrelated, haploidentical, and HLA-mismatched unrelated donor types.
Model development resulted in the Sickle Options Decision Support Tool for Children, which is a mobile-based application providing survival and risk estimates for children undergoing allogeneic HSCT. A questionnaire of 5 questions involving patient characteristics is included in the support tool. These relate to patient gender, age, donor type, stem cell source, and conditioning regimen.
Answers on the questionnaire are used by the model to generate survival and risk predictions. A summary provides estimates for outcomes such as overall survival, event-free survival, and graft vs host disease (GVHD) risk.
The researchers evaluated the acceptability and usability of the decision support tool in a study of 18 physicians with SCD expertise. Usability was determined using a 7-point scale, with higher numbers reflecting greater agreement with statements related to usability. Overall usability was reportedly high, with a mean score of 6.15 (SD, 0.79).
Acceptability was determined using a 4-point scale, with 4 reflecting the highest level of acceptability. Acceptability regarding the presentation of information in the tool was considered high, with a mean score of 2.94 (SD, 0.63) regarding bone marrow transplantation and a mean score of 3.29 (SD, 0.48) related to risk factors. However, acceptability related to the amount of information was slightly lower (mean 2.59; SD, 0.5).
The majority, or 87%, of participating physicians indicated they would use the decision support tool in patient consultations, and 89% reported the tool would ease decision making. Physicians also commented on areas of possible improvement with the tool, with some suggesting patient comorbidities should be considered in predictions, in addition to interest in more information on GVHD severity predictions, quality-of-life impacts, and other suggestions.
“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 [HSCT],” the researchers wrote in their report. They also indicated that further modifications of the tool are under consideration.
Veludhandi A, Ross D, Sinha CB, McCracken C, Bakshi N, Krishnamurti L. A decision support tool for allogeneic hematopoietic stem cell transplantation for children with sickle cell disease: acceptability and usability study. JMIR Form Res. 2021;5(10):e30093. doi:10.2196/30093