Patient-Reported Outcomes in Allogeneic Stem Cell Transplantation and CAR-T Therapy (QOL-ONE PRO-CT)

A Real-World Observational Study on Patient-Reported Outcomes in Allogeneic Stem Cell Transplantation (Allo-SCT) and CAR-T Therapy

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).

Study Overview

• Status: Not yet recruiting
• Conditions: Quality of Life; Allogeneic Blood and Marrow Transplantation (BMT); Patient Reported Outcome (PRO); CAR T Cell Therapy

• Study Type: Observational
• Enrollment (Estimated): 162

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

Adults (≥18 years) with a hematologic malignancy admitted for either (a) allo-SCT or (b) CAR-T cell therapy.

Description

Inclusion Criteria:

• 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
• Signed informed consent

Exclusion Criteria:

• Cognitive or physical impairments that preclude the ability to complete questionnaires
• Estimated life expectancy < 7 days at the time of admission

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort
CAR-T
ALLO-SCT

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	3 MONTHS

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	3 MONTHS
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.	3 MONTHS
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.	3 MONTHS
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.	3 MONTHS
To validate the use of the HM-PRO in the context of inpatient care for advanced therapies.	This outcome will evaluate the psychometric properties of the Hematological Malignancy Patient-Reported Outcome (HM-PRO) questionnaire in the inpatient setting of advanced cellular therapies (allo-SCT and CAR-T). HM-PRO assessments are collected at five time points during hospitalization-T1 (admission), T2 (day of infusion), T3 (day 3 post-infusion), T4 (day 10 post-infusion), and T5 (discharge). Psychometric analyses will assess: Internal consistency reliability of the Impact and Symptoms Scales (Cronbach's alpha). Construct validity, evaluated through known-groups comparisons (allo-SCT vs CAR-T; remission vs progression). Responsiveness to clinical change, assessed by changes in HM-PRO scores over time in relation to clinical status and adverse events. These analyses will determine whether HM-PRO performs adequately for use in routine inpatient clinical practice for advanced therapies.	3 MONTHS

Collaborators and Investigators

• Sponsor: Associazione Qol-one

Study record dates

Study Major Dates

• Study Start (Estimated): February 15, 2026
• Primary Completion (Estimated): March 1, 2027
• Study Completion (Estimated): April 1, 2027

Study Registration Dates

• First Submitted: November 17, 2025
• First Submitted That Met QC Criteria: December 4, 2025
• First Posted (Actual): December 17, 2025

Study Record Updates

• Last Update Posted (Actual): December 17, 2025
• Last Update Submitted That Met QC Criteria: December 4, 2025
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Keywords: Multiple Myeloma; Quality of Life; Lymphoma; Leukemia; CAR-T cell therapy; Patient Reported Outcome; Allogenic BMT
• Additional Relevant MeSH Terms: Vascular Diseases; Cardiovascular Diseases; Neoplasms; Immune System Diseases; Neoplasms by Histologic Type; Hematologic Diseases; Lymphatic Diseases; Lymphoproliferative Disorders; Immunoproliferative Disorders; Neoplasms, Plasma Cell; Hemostatic Disorders; Paraproteinemias; Blood Protein Disorders; Hemorrhagic Disorders; Hemic and Lymphatic Diseases; Leukemia; Lymphoma; Multiple Myeloma
• Other Study ID Numbers: QOL-ONE PRO-CT

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No