3.1. Overview Prospective, multicenter, observational cohort study comparing short-term PROs measured with the HM-PRO between two exposure groups: patients undergoing allogeneic stem cell transplantation (allo-SCT) and patients receiving CAR-T cell therapy. Patients will be enrolled at hospital admission for the index inpatient procedure and followed through the inpatient stay (admission → discharge). The study is non-randomized and designed to describe trajectories of symptoms and HRQoL and to estimate the between-group difference in deterioration of HM-PRO scores (primary estimand: mean difference in change score, CAR-T vs allo-SCT).
Diagnosed with a hematologic malignancy (e.g., leukemia, lymphoma, multiple myeloma)
Undergoing either allogeneic SCT or CAR-T therapy
Able and willing to provide written informed consent
Sufficient Italian proficiency to complete the HM-PRO
Cognitive or physical impairments that preclude the ability to complete questionnaires
Estimated life expectancy < 7 days at the time of admission
Adults (≥18 years) with a hematologic malignancy admitted for either (a) allo-SCT or (b) CAR-T cell therapy.
Cohort
Prospective
Not listed
To assess and compare short-term patient-reported outcomes [PROs (symptom burden and QoL impact)] between patients receiving allo-SCT and CAR-T therapy using the HM-PRO questionnaire (Impact Scale, Symptoms Scale) from admission to hospital discharge
The primary outcome aims to evaluate the impact of CAR-T therapy and allogeneic transplantation on patients' quality of life using a specific questionnaire (HM-PRO), which assesses PROs (quality of life and symptoms) in hematologic patients, with the goal of determining whether there are differences between the two patient groups. In the HM-PRO questionnaire values range from 0 to 100. Lower values correspond to a better quality of life and a lower symptom burden.
To describe PRO trajectories during hospitalization
Patient-reported outcomes will be assessed using the Hematological Malignancy Patient-Reported Outcome (HM-PRO) instrument, which includes an Impact Scale and a Symptoms Scale. HM-PRO assessments will be performed at five predefined time points during hospitalization: T1 (hospital admission), T2 (day of infusion), T3 (day 3 post-infusion), T4 (day 10 post-infusion), and T5 (day of discharge). The secondary outcome will describe within-patient trajectories of HM-PRO scores over these time points. Data will be summarized using mean scores and mean changes from baseline (T1) for total and domain scores, to characterize changes in symptom burden and QoL impact throughout hospitalization.
To assess differences in PROs based on disease type (e.g., Leukemia, lymphoma, myeloma)
Since patients with different hematologic diseases are involved, the quality-of-life and symptom outcomes may vary for each condition.
To assess differences in PROs based on disease status (remission vs progression)
Patients at enrollment may be in remission or in disease progression. the quality-of-life and symptom outcomes may vary for each condition.
To assess differences in PROs based on treatment line (first-line vs multiple lines)
At enrollment, patients may have received first vs multiple lines of treatment. the quality-of-life and symptom outcomes may vary for each condition.
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