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NCT Number: NCT07289087

Patient-Reported Outcomes in Allogeneic Stem Cell Transplantation 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).

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Key information

Conditions

Quality of Life Allogeneic Blood and Marrow Transplantation (BMT) Blood Protein Disorders CAR T Cell Therapy Cardiovascular Diseases Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders Hemostatic Disorders Immune System Diseases Immunoproliferative Disorders Leukemia Lymphatic Diseases Lymphoma Lymphoproliferative Disorders Multiple Myeloma Neoplasms Neoplasms by Histologic Type Neoplasms, Plasma Cell Paraproteinemias Patient Reported Outcome (PRO) Vascular Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Primary outcomes

  1. 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

    Time frame: 3 MONTHS

    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.

Secondary outcomes

  1. To describe PRO trajectories during hospitalization

    Time frame: 3 MONTHS

    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.

  2. To assess differences in PROs based on disease type (e.g., Leukemia, lymphoma, myeloma)

    Time frame: 3 MONTHS

    Since patients with different hematologic diseases are involved, the quality-of-life and symptom outcomes may vary for each condition.

  3. To assess differences in PROs based on disease status (remission vs progression)

    Time frame: 3 MONTHS

    Patients at enrollment may be in remission or in disease progression. the quality-of-life and symptom outcomes may vary for each condition.

  4. To assess differences in PROs based on treatment line (first-line vs multiple lines)

    Time frame: 3 MONTHS

    At enrollment, patients may have received first vs multiple lines of treatment. the quality-of-life and symptom outcomes may vary for each condition.

  5. To validate the use of the HM-PRO in the context of inpatient care for advanced therapies.

    Time frame: 3 MONTHS

    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.

Sponsors and collaborators

Lead sponsor

Associazione Qol-one

Other

Registry information

Official study title

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

Acronym: QOL-ONE PRO-CT

Important dates

Study start
2026
Primary completion
2027
Study completion
2027
First posted
Dec 17, 2025
Registry last updated
Dec 17, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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