Prognostic Understanding for Older, Medically Infirm, and/or Frail Recipients of Allogeneic Hematopoietic Cell Transplantation in an Ongoing Seamless Phase 2/3 Randomized Interventional Trial (ACE-BMT)
Authors: Loggers Elizabeth T. , Fausto James , Givens Matthew , Yeow Mei-Ean , Jayani Reena V. , Alkhateeb Hassan B , Schoenherr Laura A , Rabow Michael W. , Juckett Mark B , Carrum George , Uberti Joseph , Neale Kyle , Lopez Santiago , Martins-Welch Diana , Ferrer Jeanette G , Rhee Laura S , Rosielle Drew A , McInnes Susan , Webb Jason A , Cook Rachel J. , Newman Elisheva , Sobecks Ronald , Johnston Laura , Sorror Mohamed 
Published: December 2024

INTRODUCTION

While prognostic understanding (PU) is important for all patients (pts) considering hematopoietic stem cell transplantation (HCT), it may be particularly important for older (&gt;65 years), medically infirm, and/or frail pts deciding whether to undergo allogeneic HCT (allo-HCT). This abstract reports on PU and concordance with transplant physician (hereafter MD) PU for pts participating in an ongoing seamless, phase II-III randomized clinical trial (ACE-BMT) comparing 4-arms: a pt-administered comorbidity management intervention (CM), specialist-administered palliative care (PC), a combined arm of CM and PC to standard of care. Here we report preliminary data on the concordance between pt and MD PU on the chance of cure 1-year (yr) post allo-HCT.

METHODS

Pt PU was compared to MD PU (gold standard) to determine concordance. MD and pt answers were compared on a single validated question asked at enrollment to the study (typically 2 weeks prior to HCT). Both pts and MD's estimated the chance of cure 1 yr after allo-HCT by selecting one of six possible response options: very good (more than 90%), good (75-90%), better than 50% (50-74%), worse than 50% (25-49%), bad (10-24%) or very bad (less than 10%). PU was “concordant” or “accurate” if MD and pt selected the same response option. Pts with discordant PU were labeled “pessimistic” if the pt selected a response option with a lower chance of cure than the MD (and “optimistic” if a higher chance). Response options were further simplified to greater than or less than 50% chance of cure and MD and pt PU were compared again. Descriptive statistics, chi-square for categorical and t-tests for continuous variables were used. Logistic regression was used to determine the association between pt prognostic concordance and pt socio-demographic and clinical characteristics.

RESULTS

All pts consented to date were included (n=358), except those who did not answer the prognosis question (n=61). 54.2% (n=194) had HCT-Comorbidity Index (HCT-CI) of ≥3, 67.9% (n=243) were older than 65 yrs and 4.5% (n=16) were frail, defined by walk speed &lt;0.8 m/s. Median age was 68 yrs (range 20-80). 39.7% (n=141) were female. 89.9% (n=322) identified as White, 4.2% (n=15) Asian, and 1% each as Black/African American and Native Hawaiian/Other Pacific Islander. 5.3% (n=19) identified Hispanic. 11.5% (n=44) had high school education or less. 54.1% (n=183) made less than $100,000/yr. 40.1% (n=136) had acute myelogenous leukemia, 25.4% (n=86) had high-risk myelodysplastic syndrome and 10.6% (n=36) had a myeloproliferative cancer. 72.1% (n=245) had matched, 82.5% (n=283) unrelated and 95.0% (n=36) peripheral blood allo-HCT. 9% (n=31) had KPS of 100%, 35% (n=121) KPS 90%, and 47.5% (n=147) KPS 80%.

Pts reported having a very good, good, better than 50%, worse than 50% and bad/very bad chance of cure 1 yr post allo-HCTL 34.4% (n=123), 30.7% (n=110), 26.0% (n=93), 7.3% (n=26) and 1.7% (n=6), respectively. 91.1% (n=326) of pts felt they had a &gt;50% chance of being cured 1 yr post allo-HCT.

MD answers were missing for 159/358 (44.4%) pts. MDs reported pt had a very good, good, better than 50%, worse than 50% and bad/very bad chance of being cured 1 yr post allo-HCT, 3.0% (n=6), 6.5% (n=13), 52.8% (n=105), 35.7% (n=71) and 2% (n=4) respectively. 62.3% (n=124) of MDs felt the pt had a &gt;50% chance of being cured 1 yr post allo-HCT.

For the subset of pts with MD prognostic data (n=199), pt's PU was concordant 20.6% (n=41) of the time when compared across all 6 response options. Overall, 71.9% (n=143) of pts erred toward optimism, while 7.5% (n=15) of pts were pessimistic. 60.8% of MD and pts agreed on prognosis when dichotomized to better or worse than 50% chance of being cured 1 yr post allo-HCT.

Prognostic concordance was not significantly associated with any pt socio-demographic factors, HCT-CI or KPS via descriptive statistics or regression analysis.

CONCLUSIONS

In this prospective randomized trial focusing on a population of older, medically infirm, and/or frail pts receiving allo-HCT, pts' PU was not concordant with MD prognosis approximately 80% of the time. While some pts were pessimistic, the majority overestimated the likelihood they would be cured of disease compared to MD estimates. Future studies should investigate the true accuracy of both MD and pt prognosis, as well as how to improve communication about prognosis and pt PU (if inaccurate based on best available information) prior to HCT.