PUBLICATION ON “DEVELOPMENT OF A CORE SET OF PATIENT – AND CAREGIVER – REPORTED SIGNS AND SYMPTOMS TO FACILITATE EARLY RECOGNITION OF ACUTE CHIMERIC ANTIGEN RECEPTOR T-CELL THERAPY TOXICITIES”

ABSTRACT

Prompt recognition of acute chimeric antigen receptor T (CAR T)-cell–mediated toxicities is crucial

because adequate and timely management can prevent or reverse potential life-threatening complications. In the

outpatient setting, patients and informal caregivers have to recognize and report signs and symptoms marking

these acute toxicities. This study provides a core set of patient- and caregiver-reported signs and symptoms

(outcomes, P/CROs) and definitions of red flags warranting immediate action to include in a daily checklist for

support at home, with the goal to make outpatient post–CAR T-cell care safer, optimize patient and caregiver

support, and thereby facilitating an early discharge/hospital visit reduction strategy.

We performed a systematic review of phase II/III trials of US Food and Drug Administration–approved

CAR T-cell products and selected all common and severe adverse events that could be translated into a P/CRO

for inclusion in a two-round modified Delphi procedure. Eleven CAR T-cell–dedicated hematologists from the

Dutch CAR T-cell tumorboard representing all treating centers selected P/CROs for inclusion in the core set and

defined red flags. The final core set was evaluated with patients and caregivers.

From nine clinical trials, 457 adverse events were identified of which 42 could be used as P/CRO. The

final core set contains 28 items, including five signs for measurement via wearables and two signs for caregiverperformed

assessments.

This study provides a core set of P/CROs that can serve as a framework for (eHealth) tools that aim

to enable patients and caregivers to more effectively recognize and report signs and symptoms of acute toxicities

after CAR T-cell therapy, which will enhance safe outpatient treatment monitoring.