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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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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| CAR-T | |||
| ALLO-SCT |
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| 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 |
| 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. |
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Inclusion Criteria:
Exclusion Criteria:
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Adults (≥18 years) with a hematologic malignancy admitted for either (a) allo-SCT or (b) CAR-T cell therapy.
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Oct 6, 2025 | Oct 29, 2025 | Prot_000.pdf |
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| ID | Term |
|---|---|
| D007938 | Leukemia |
| D009101 | Multiple Myeloma |
| D008223 | Lymphoma |
| ID | Term |
|---|---|
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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| 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 |
| D054219 | Neoplasms, Plasma Cell |
| D020141 | Hemostatic Disorders |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D010265 | Paraproteinemias |
| D001796 | Blood Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D008232 | Lymphoproliferative Disorders |
| D007160 | Immunoproliferative Disorders |
| D007154 | Immune System Diseases |
| D008206 | Lymphatic Diseases |