Abstract
The success in minimizing sickle cell disease (SCD) morbidity in pediatrics has often been difficult to maintain upon moving to adult care. Research to improve young adult (YA) transitions primarily focuses on pre-transition (pediatric) preparedness and/or increased patient focus on SCD complication monitoring, producing mixed results and unclear sustainability on the adult side. Three years ago, the University of Minnesota (UMN) adult SCD team began to prioritize a person-first approach for YAs, ostensibly focusing less on keeping SCD as the mental focal point and more on future non-healthcare-related aspirations [“life goals” (LG)] as hematologists' standard practice. This aligned with non-SCD psychology data supporting goal-directed cognitive behavioral therapy (CBT) in transitions of YAs with complex diseases. A 45% reduction in acute care utilization (ACU) was seen after 1 year (range of improvement 0-81%; none worse than prior to LG initiation) using this strategy while maintaining good clinic adherence, offering pilot data for studying this approach more rigorously.
An IRB-approved randomized controlled trial using block randomization (STUDY00018692 on www.studyfinder.umn.edu) is ongoing in the UMN adult SCD program where YAs (18-28 years) new to clinic, regardless of genotype or SCD complication history, are approached for participation after the second visit. The hypothesis is that LG-focused care can reduce ACU versus standard complication-focused clinical visits (historical approach), with added benefit from CBT. The control group involves UMN standard evidence-based care with added LG prioritization and documentation each visit. Visits questionnaires include PHQ-9, Working Alliance Inventory (WAI) for therapeutic relationship evaluation (WAI are done by participants, hematologists, and the psychologist if applicable), Hill-Bone Medication Adherence Scales (HBMAS) and LG Scale (modified from HBMAS), and the WHO Quality of Life-BREF evaluation. The experimental arm receives the same approach plus monthly CBT focused on acceptance and commitment therapy. An exploratory arm matches the experimental arm but is for those undergoing transplant or gene therapy regardless of age. Clinical outcomes include ACU, medication use and refill frequency, and missed visit counts using an intention-to-treat approach.
Goal recruitment is 10 each for the control and experimental arms and 6 for the exploratory arm. Study participation is for 15 months. Participants see any 1 of 3 hematologists who see YAs with SCD. One psychologist conducts experimental and exploratory arm CBT visits. Outcomes will be compared to historical institutional data from 2019-2021 prior to institution of the LG approach from which the pilot data originated. Inclusion is limited to English speakers and those with English literacy and having had no more than 2 clinic visits prior to enrollment, except for those in the exploratory arm, where no clinic visit limit was in place.
With 20 participants (10 per arm) in the control vs experimental prospective cohort, we will have 80% power to be able to detect an effect size of 1.57 between these study arms at the 0.0167 significance level. Historical data (2019-2021) will be selected using coarsened exact matching for age, sex, and race to the prospective randomized cohort (2:1 ratio). Exploratory arm data will be matched 1:1. Participant demographics and clinical variables will be summarized, and balance between arms and cohorts will be assessed using standardized mean differences. Differences between study arms for the primary outcomes of ACU, medication adherence, and visit attendance will assessed overall using generalized linear models for continuous or count data. Each primary outcome will be compared using the 0.0167 significance level to account for multiple testing via Bonferroni correction. The WAI has a validated scoring element, so these data will be used in aggregate to evaluate the therapeutic alliances. Larger thematic elements from CBT will be collected and qualitatively analyzed using constant comparison analyses.
Enrollment currently stands at 14 for the RCT portion (7 each, 3 males, 11 females, median age 23) and 1 male in the exploratory arm. Enrollment age range initially began at 18-24 years but was expanded to 28 to improve recruitment. One has been lost to follow up and 1 had major health complications limiting further engagement. One has completed the study with 2 more finishing soon.
