A Real-World Observational Study on Patient-Reported Outcomes in Allogeneic Stem Cell Transplantation (Allo-SCT) and CAR-T Therapy
Conditions: Quality of Life, Patient Reported Outcome (PRO), Allogeneic Blood and Marrow Transplantation (BMT), CAR T Cell Therapy
Sponsor: Associazione Qol-one

 QOL-ONE PRO-CT                                Versione 1 del 06.10.2025 
 
P a g .  1 | 16 
 
Clinical Study Protocol 
Study Title: 
A Real-World Observational Study on Patient-Reported Outcomes in Allogeneic 
Stem Cell Transplantation (Allo-SCT) and CAR-T Therapy (QOL-ONE PRO-CT) 
 
Protocol ID: [To be assigned] 
Version: 1.0 
Date: October 6, 2025 
Sponsor/Collaborator: QOL-ONE Research Association 
Study Sites: 
• Policlinico Gemelli, Rome, Italy 
• La Sapienza University, Rome, Italy 
 
Clinical Trial Leader: Esther N Oliva, MD 
 
Coordinating Center: Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, 
Italy 
Principle Investigator: Prof. Simona Sica  
Sponsor: Associazione QOL-ONE 
 
 
 
Steering committee members: Di Rocco Alice, Ionova Tatyana, Iori Anna Paola, Oliva Esther 
Natalie, Salek Sam, Sica Simona, Tripepi Rocco  
 
This protocol has been written and will be conducted according to the Declaration 
of Helsinki, and with the principles established according to Good Clinical 
Practice guidelines 

 QOL-ONE PRO-CT                                Versione 1 del 06.10.2025 
 
P a g .  2 | 16 
 
Approval of the protocol 
 
 
Sponsor/Collaborator: QOL-ONE Research Association 
 
Legal Representative 
Rocco Tripepi 
 
 
Signature ____________________ 
Date ___/___/______ 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 

 QOL-ONE PRO-CT                                Versione 1 del 06.10.2025 
 
P a g .  3 | 16 
 
Principal investigator approval of the protocol 
 
I undersigned___________________________________________________ 
(name and surname) 
 
 
Principal Investigator of the CENTER___________________________ 
 
 
 
Declare to conduct the study QOL-ONE PRO-CT in compliance with the 
protocol. 
 
Acknowledge that I am responsible for overall study conduct. I agree to 
personally conduct or supervise the described clinical study. 
 
Agree to ensure that all associates, colleagues and employees assisting in the 
conduct of the study are informed about their obligations. Mechanisms are in place to 
ensure that site staff receive the appropriate information throughout the study. 
 
 
Investigator name: _____________________________ 
Investigator signature: _______________________ 
 
 
Date: ____/____/___  
 
 
(dd  /  mm /  yy) 
 
 

 QOL-ONE PRO-CT                                Versione 1 del 06.10.2025 
 
P a g .  4 | 16 
 
 
Participating sites 
 
1. Prof. Simona Sica  
(coordinating site) 
Fondazione Policlinico Universitario Agostino Gemelli 
IRCCS, Ematologia e Trapianto di cellule staminali 
emopoietiche, Rome, Italy 
2.  Prof. Anna Paola Iori 
AOU Policlinico Umberto I, UOC Ematologia, Rome, 
Italy 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 

 QOL-ONE PRO-CT                                Versione 1 del 06.10.2025 
 
P a g .  5 | 16 
 
 
 
Summary 
1. Background and Rationale...................................................................................................................................... 6 
2. Study Objectives ........................................................................................................................................................... 7 
3. Study Design................................................................................................................................................................... 7 
3.1. Overview ...................................................................................................................................................................... 7 
3.2 Duration: ....................................................................................................................................................................... 7 
4. Population ....................................................................................................................................................................... 8 
5. Assessment Schedule................................................................................................................................................. 8 
6. Collected Variables ..................................................................................................................................................... 8 
7. Endpoints......................................................................................................................................................................... 9 
8. Statistical Analysis ...................................................................................................................................................... 9 
9. Methods ......................................................................................................................................................................... 12 
9.1 Ethical considerations ......................................................................................................................................... 12 
9.1.1 Data protection ................................................................................................................................................... 12 
10. Schedule of Procedures ...................................................................................................................................... 14 
11. Dissemination of Results ................................................................................................................................... 15 
12. Funding and Conflicts of Interest .................................................................................................................. 15 
13. References ................................................................................................................................................................. 15 
 
 
 
 
 
 
 
 

 QOL-ONE PRO-CT                                Versione 1 del 06.10.2025 
 
P a g .  6 | 16 
 
1. Background and Rationale 
Advancements in cellular therapies for hematologic malignancies (HM)—notably allogeneic 
stem cell transplantation (allo-SCT) and chimeric antigen receptor T-cell (CAR-T) therapies—
have significantly improved clinical outcomes and survival for patients with otherwise 
limited treatment options [1–3]. Allo-SCT remains a curative approach for many malignant 
hematologic conditions through graft-versus-leukemia effects but is associated with 
considerable short- and long-term morbidity, including graft-versus-host disease (GVHD), 
infectious complications, and delayed quality of life recovery [4–6]. CAR-T therapy, by 
contrast, has revolutionized the management of relapsed or refractory B-cell malignancies, 
offering durable responses in heavily pretreated patients. However, CAR-T is characterized 
by unique acute toxicities such as cytokine release syndrome (CRS) and immune effector cell-
associated neurotoxicity syndrome (ICANS), which often occur during the index 
hospitalization and substantially affect symptom burden [7–9]. 
Despite the transformative impact of these therapies, there remains a paucity of real-world 
data describing short-term patient-reported outcomes (PROs) during the acute inpatient 
phase of treatment. While long-term quality of life trajectories have been studied in both allo-
SCT and CAR-T cohorts [10–12], less is known about patients’ immediate experiences 
throughout hospitalization, when symptom burden is highest and when clinical and 
supportive care decisions are most critical. Importantly, a recent prospective longitudinal 
study directly compared PROs across CAR-T, autologous SCT, and allo-SCT recipients. This 
study demonstrated that although all groups experienced a decline in quality of life with a 
nadir around week 2, CAR-T recipients had a significantly less severe decline and a faster 
recovery compared to allo-SCT patients, who experienced the greatest symptom burden and 
slowest recovery [13]. These findings underscore the need for further prospective, real-
world research to better characterize short-term PRO trajectories in these populations and 
to confirm these observations in diverse clinical settings. Understanding these short-term 
experiences has implications for optimizing symptom management, resource utilization, 
discharge readiness, and early survivorship care [14,15]. 
The Hematological Malignancy Patient-Reported Outcome (HM-PRO) is a validated 
instrument specifically developed to assess health-related quality of life (HRQoL) and 
symptom burden in patients with HM [16–20]. It consists of two parts: Part A evaluates the 
impact of disease and treatment on HRQoL, and Part B assesses the severity of symptoms. 
HM-PRO has undergone rigorous psychometric validation, demonstrating strong content 
validity, construct validity, reliability, and sensitivity to change across a broad spectrum of 
hematologic malignancies [16–18]. Its disease-specific design makes it particularly suitable 
for capturing the dynamic and treatment-specific symptom profiles observed in allo-SCT and 
CAR-T recipients. 
Given the distinct toxicity and recovery profiles of allo-SCT and CAR-T therapy, a prospective 
observational study is warranted to compare short-term PRO trajectories between these two 
patient groups during the inpatient treatment course. Observational methodology is 
appropriate, as treatment allocation is determined by clinical indication rather than 

 QOL-ONE PRO-CT                                Versione 1 del 06.10.2025 
 
P a g .  7 | 16 
 
randomization, and such a design enables the capture of real-world patient experiences in a 
non-interventional setting [21,22]. By employing the HM-PRO to collect PRO data at baseline, 
throughout hospitalization, and at discharge, this study will generate high-resolution, real-
world evidence on patient experiences that are currently underrepresented in the literature. 
These findings will help inform supportive care strategies, improve patient-centered 
decision-making, and contribute to comparative effectiveness research in advanced cellular 
therapies. 
2. Study Objectives 
Primary Objective: 
- 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 
from admission to hospital discharge. 
 
Secondary Objectives: 
- To describe PRO trajectories during hospitalization. 
- To assess differences in PROs based on: 
- Treatment modality (allo-SCT vs CAR-T) 
- Disease type (e.g., AML, lymphoma, myeloma) 
- Disease status (remission vs progression) 
- Treatment line (first-line vs multiple lines) 
- To validate the use of the HM-PRO in the context of inpatient care for advanced therapies. 
3. Study Design 
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). 
3.2 Setting:   
Two tertiary care hospitals in Rome, Italy, with active allo-SCT and CAR-T in patient 
programs; multicenter to improve generalizability. 
3.2 Duration:  
1 year enrollment, participation from hospital admission to discharge (maximum 3 months). 

 QOL-ONE PRO-CT                                Versione 1 del 06.10.2025 
 
P a g .  8 | 16 
 
4. Population 
One hundred and sixty-two adults (≥18 years) with a hematologic malignancy admitted for 
either (a) allo-SCT (N=81) or (b) CAR-T cell therapy (N=81).  
4.1 Inclusion Criteria: 
- Age ≥ 18 years 
- 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 
 
4.2 Exclusion Criteria: 
- Cognitive or physical impairments that preclude the ability to complete questionnaires 
- Estimated life expectancy < 7 days at the time of admission 
5. Assessment Schedule 
Patient-reported outcomes will be collected using the HM-PRO instrument at the following 
time points: 
- T1: Hospital admission 
- T2: Day of infusion (CAR-T or stem cell infusion) 
- T3: Day 3 post-infusion 
- T4: Day 10 post-infusion 
- T5: Day of hospital discharge 
6. Collected Variables 
Demographics: 
- Age, Gender, Education level, occupation, civil status 
 
Clinical Variables: 
- Comorbidities, Disease type, Disease status (remission/progression), Line of treatment 
 
Laboratory: 
- Full blood counts 
 
Treatment: 
- CAR-T or allo-SCT treatment details, including conditioning regimen and product specifics 
 
Adverse Events: 
- Documented using CTCAE criteria, including: 
  - Mucositis, Infections, GvHD, CRS, ICANS 
 

 QOL-ONE PRO-CT                                Versione 1 del 06.10.2025 
 
P a g .  9 | 16 
 
Patient-Reported Outcomes: 
- HM-PRO (Impact Scale, Symptoms Scale) 
7. Endpoints 
Primary Endpoint: 
- Difference in HM-PRO total and domain scores between allo-SCT and CAR-T groups at each 
time point (T1–T5) 
 
Secondary Endpoints: 
- Change in HM-PRO scores over time within each group 
- Comparison of PROs by disease status and treatment line 
- Frequency and severity of adverse events and correlation with PRO data 
8. Statistical Analysis 
- All analyses will be performed using two-sided tests with a nominal significance level of α = 
0.05 unless otherwise stated. Estimates will be reported with 95% confidence intervals. 
Primary analysis population include all enrolled participants with at least one post-baseline 
HM-PRO assessment (intention-to-observe principle). 
-Baseline demographic and clinical variables will be summarized by treatment group (allo-
SCT vs CAR-T). 
• 
Continuous variables: mean (SD) and median (IQR) as appropriate. 
• 
Categorical variables: counts and percentages. 
- Between-group baseline comparisons will be descriptive (t-tests/Wilcoxon tests or chi-
square/Fisher exact tests) to display balance; inferential testing of baseline covariates is not 
the primary focus. A baseline table will be produced. 
8.1 Primary longitudinal analysis (HM-PRO) 
• 
Primary modelling approach: linear mixed-effects models (LMM) for repeated 
measures of HM-PRO scores to evaluate within-hospital trajectories and between-
group differences. The base model will include: 
o 
Fixed effects for timepoint (categorical: T1, T2, T3, T4, T5), treatment 
group, and the group × time interaction (to test for differential trajectories). 
o 
A random intercept for participant to account for within-subject correlation; 
random slope for time will be considered if supported by data and model fit. 
o 
Prespecified covariates: baseline HM-PRO score (if modelling discharge score 
via ANCOVA alternatively), age, sex, disease type, disease status, line of 
therapy, major comorbidity index, and site (as fixed effect or as random effect 
in a hierarchical model). 

 QOL-ONE PRO-CT                                Versione 1 del 06.10.2025 
 
P a g .  10 | 16 
 
• 
Parameter of interest: the group × time interaction contrasts (e.g., difference in mean 
change from baseline to discharge between groups). Report estimated marginal 
means (least-squares means), differences, 95% CIs and p-values. 
• 
Model diagnostics: check residuals for normality and heteroscedasticity, influence 
diagnostics, and perform sensitivity analyses using robust standard errors if 
necessary. If severe non-normality persists, consider transformation or a generalized 
linear mixed model (GLMM) or rank-based mixed model. 
Repeated measures ANOVA 
• 
As a supportive analysis (or when assumptions are met and complete data are 
available), repeated-measures ANOVA (with Greenhouse-Geisser correction if 
sphericity violated) may be used to summarize overall time and group effects. 
Primary inference will rely on mixed models which better accommodate missingness 
and unequal intervals. 
Between-group comparisons & ANCOVA 
• 
For the primary endpoint specified as change from baseline to discharge, an ANCOVA 
model will be used (discharge HM-PRO as outcome, baseline HM-PRO as covariate, 
plus the same prespecified covariates). This produces an adjusted estimate of the 
between-group difference in mean change. 
• 
Unadjusted two-sample t-tests (or nonparametric Wilcoxon tests) will be presented 
for transparency. 
Subgroup analyses 
• 
Exploratory subgroup analyses will be performed for: disease type (AML vs 
lymphoma vs myeloma), disease status (remission vs progression), age strata, and 
treatment line. Each subgroup analysis will include a formal interaction test (group 
× subgroup) within the primary model rather than separate stratified unadjusted 
tests. Subgroup results are exploratory and interpreted cautiously. 
Correlation / association between adverse events and PROs 
• 
Correlation analyses: relationship between AE burden (grade or counts) and HM-
PRO scores will be examined using Spearman’s rank correlation (for ordinal AE 
grades) or Pearson correlation if assumptions hold. 
• 
Regression analyses: to examine whether specific AEs (e.g., CRS, ICANS, mucositis, 
infections, GvHD) are associated with concurrent or subsequent HM-PRO scores, 
include AE indicators or graded severity as fixed effects in the mixed model (time-
varying covariates). Logistic or Poisson regression (or negative binomial) may be 
used where appropriate (e.g., modelling probability of clinically meaningful 
deterioration, or AE counts). Report adjusted effect estimates (e.g., mean differences, 
odds ratios, incidence rate ratios) with 95% CIs. 

 QOL-ONE PRO-CT                                Versione 1 del 06.10.2025 
 
P a g .  11 | 16 
 
• 
Pre-specify temporal directionality in the SAP (e.g., whether AEs occurring before an 
HM-PRO assessment are considered predictors). 
Handling of missing data 
• 
Item-level HM-PRO missingness: per the HM-PRO scoring manual, missing item 
responses will not be substituted; scores will be calculated according to the 
instrument’s guidance. 
• 
Missing assessments / visit-level missingness: for missing entire HM-PRO 
assessments (e.g., missed T2, T3, T4 or T5), the primary approach will be multiple 
imputation by chained equations (MICE) under a missing at random (MAR) 
assumption. Imputation models will include baseline and post-baseline HM-PRO 
items/scores, demographic and clinical covariates, laboratory markers, site, and 
indicators predictive of missingness. We will perform at least m = 20 imputations (or 
more if fraction of missing information is high) and combine estimates by Rubin’s 
rules. 
• 
Sensitivity analyses: compare results from: complete-case analysis, multiple 
imputation, and worst-case / tipping-point analyses to assess robustness to missing 
not at random (MNAR) mechanisms. The SAP will detail variables included in 
imputation models and diagnostics for imputation quality. 
Multiplicity and inference 
• 
Multiple secondary endpoints and subgroup tests are planned; these will be 
described as exploratory. Where formal control of family-wise error is desired for 
predefined secondary endpoints, procedures (e.g., Holm or Benjamini-Hochberg false 
discovery rate) will be specified in the SAP. 
Additional analyses 
• 
Proportion with clinically meaningful deterioration: compute proportion of 
participants with ≥6-point worsening from baseline to discharge and compare groups 
using chi-square tests and logistic regression adjusted for covariates; report risk 
differences and adjusted odds ratios with 95% CIs. 
• 
Sensitivity for clustering: if substantial between-site heterogeneity is present, use 
mixed models with random site intercepts or a generalized estimating equations 
(GEE) approach with robust SEs clustered by site. 
• 
Exploratory trajectories: use latent class growth modelling or clustering of 
longitudinal profiles if warranted to identify common PRO trajectory phenotypes 
(reported descriptively). 
Reporting 
• 
All results will be reported according to STROBE guidance for observational studies 
and following best practices for PRO reporting (including instrument scoring details, 
number of missing items, and sensitivity analyses). Statistical code and de-identified 

 QOL-ONE PRO-CT                                Versione 1 del 06.10.2025 
 
P a g .  12 | 16 
 
analytic datasets will be archived as per institutional policies and will be made 
available on reasonable request, consistent with GDPR and ethics approvals. 
 
8.2.  Sample Size: 
The primary objective was to detect a between-group difference in HM-PRO between groups 
based on the difference in HM-PRO total score of Δ = 6 points (the established Minimal 
Important Difference). Assuming a two-sided α = 0.05 and 80% power, the required sample 
size per group was calculated using the two-sample t-test formula: 
n=(2(z_(1-α/2)+z_(1-β) )^2 σ^2)/Δ^2. 
Because the SD of HM-PRO change in our population is uncertain, we present results for a 
range of plausible SDs: for SD = 12 the required sample size is 63 per group (80% power); for 
SD = 15 it is 98 per group. If the primary analysis adjusts for baseline HM-PRO using ANCOVA 
and the correlation between baseline and outcome is r, the residual variance is approximately 
σ^2 (1-r^2) and the required sample size is reduced accordingly (for example, with SD = 15 
and r=0.5, n ≈ 74 per group). We inflated the calculated sample size by 10% to allow for 
anticipated loss to follow-up (final target n per group = 81). All sample-size calculations were 
performed in R using power.t.test. 
 
9. Methods 
9.1 Ethical considerations 
The study will be performed in accordance with the principles of the Declaration of 
Helsinki, Good Clinical Practice (GCP) guidelines, and all applicable national regulations. 
Approval will be obtained from the local Institutional Review Boards (IRB) / Research 
Ethics Committees (REC) of all participating centers prior to study initiation. 
All participants (or their legally authorized representatives) will provide written informed 
consent before enrollment. Patients will be informed about study objectives, procedures, 
potential risks, and their right to withdraw at any time without any impact on their standard 
clinical care. 
9.1.1 Data protection 
Data protection: All study data will be de-identified prior to analysis and processed in 
accordance with the requirements of the European Union General Data Protection 
Regulation (EU GDPR). Pseudonymized identifiers will be used for data entry into the 
REDCap system, and re-identification keys will be stored separately under secure, access-
controlled conditions. Only study personnel will have access to identifiable information. 
9.1.2 Risk assessment:  
There is no anticipated physical risk to participants beyond standard clinical care, as this 
is a non-interventional, observational study limited to collection of PROs and routinely 
available clinical data. 
9.2 Data collection and management 

 QOL-ONE PRO-CT                                Versione 1 del 06.10.2025 
 
P a g .  13 | 16 
 
Data will be collected prospectively and entered into secure electronic case report forms 
(eCRFs) using the REDCap platform hosted on institutional servers. The Contract Research 
Organization (CRO) DIELNET SRL will be responsible for the creation, setup, and 
maintenance of the eCRFs. REDCap access will be role-restricted, password-protected, and 
audited to ensure data integrity.  
9.3 Patient-reported outcomes 
The HM-PRO instrument, a validated PRO tool for patients with hematological malignancies, 
will be used to assess symptom burden and health-related quality of life (HRQoL). HM-PRO 
will be self-administered in paper format by patients at standardized time points during 
hospitalization: 
• 
T1: Hospital admission (baseline) 
• 
T2: Day of infusion (CAR-T or stem cell infusion) 
• 
T3: Day 3 post-infusion 
• 
T4: Day 10 post-infusion 
• 
T5: Day of hospital discharge 
Study staff will facilitate administration of the instrument and verify completeness of 
responses. Where patients are unable to self-complete due to temporary health-related 
limitations, assistance will be provided in accordance with HM-PRO guidance to preserve 
data validity. The completed questionnaires will be scanned and uploaded into the REDCap 
system to ensure secure storage and source documentation. 
9.4 Collected variables 
Demographics 
• 
Age 
• 
Gender 
• 
Education level 
• 
Occupation 
• 
Civil status 
Clinical variables 
• 
Comorbidities (documented at admission) 
• 
Disease type (e.g., AML, lymphoma, myeloma) 
• 
Disease status (remission vs progression) 
• 
Line of treatment (first-line vs subsequent lines) 
Laboratory 
• 
Full blood counts at baseline and as per standard of care during hospitalization 

 QOL-ONE PRO-CT                                Versione 1 del 06.10.2025 
 
P a g .  14 | 16 
 
Treatment-related data 
• 
Treatment modality (CAR-T or allo-SCT) 
• 
Conditioning regimen details 
• 
Product specifics (CAR-T product characteristics or stem cell source/donor type) 
Adverse events 
Adverse events will be systematically captured and graded according to Common 
Terminology Criteria for Adverse Events (CTCAE, version 5.0). The following specific 
toxicities of interest will be documented: 
• 
Mucositis 
• 
Infections 
• 
Graft-versus-host disease (GvHD; allo-SCT group only) 
• 
Cytokine release syndrome (CRS; CAR-T group only) 
• 
Immune effector cell-associated neurotoxicity syndrome (ICANS; CAR-T group only) 
10. Schedule of Procedures 
Procedure / 
Assessment 
T1: 
Admission 
T2: 
Infusion  
T3: Day 3 
Post-Infusion 
T4: Day 10 
Post-Infusion 
T5: 
Discharge 
Informed consent 
✔ 
– 
– 
– 
– 
Eligibility check 
✔ 
– 
– 
– 
– 
Demographics 
✔ 
– 
– 
– 
– 
Clinical variables 
(comorbidities, 
disease type, status, 
line of treatment) 
✔ 
– 
– 
– 
– 
Laboratory tests 
(full blood counts) 
✔ 
(baseline) 
As per 
SOC 
As per SOC 
As per SOC 
As per SOC 
Treatment details 
(conditioning 
regimen, product 
specifics) 
– 
✔ 
– 
– 
– 

 QOL-ONE PRO-CT                                Versione 1 del 06.10.2025 
 
P a g .  15 | 16 
 
Procedure / 
Assessment 
T1: 
Admission 
T2: 
Infusion  
T3: Day 3 
Post-Infusion 
T4: Day 10 
Post-Infusion 
T5: 
Discharge 
Adverse events 
(CTCAE vX.X) 
– 
✔ (from 
infusion) 
✔ 
✔ 
✔ 
HM-PRO 
questionnaire 
✔ 
✔ 
✔ 
✔ 
✔ 
Questionnaire 
scan & upload to 
REDCap 
✔ 
✔ 
✔ 
✔ 
✔ 
✔ = performed 
SOC = standard of care 
11. Dissemination of Results 
Study results will be submitted for presentation at scientific conferences and for publication 
in peer-reviewed journals. All publications will maintain patient confidentiality and comply 
with authorship standards. 
12. Funding and Conflicts of Interest 
This study is sponsored by QOL-ONE Research Association. No conflicts of interest are 
declared by the principal investigators or collaborators. 
13. References 
1. D’Souza A, Fretham C. Current Uses and Outcomes of Hematopoietic Cell 
Transplantation (HCT): CIBMTR Summary Slides, 2020. 
2. Neelapu SS, et al. Chimeric antigen receptor T-cell therapy — assessment and 
management of toxicities. Nat Rev Clin Oncol. 2018. 
3. Bachanova V, Perales MA. Modern cellular therapy for hematologic malignancies. 
Blood Rev. 2021. 
4. Wingard JR, et al. Long-term health and quality of life after hematopoietic cell 
transplantation. Biol Blood Marrow Transplant. 2011. 
5. Bevans M, El-Jawahri A. Patient-reported outcomes in hematopoietic cell 
transplantation: past, present, and future. Biol Blood Marrow Transplant. 2019. 
6. Pidala J, et al. Quality of life after allogeneic hematopoietic cell transplantation. Blood. 
2009. 
7. Neelapu SS, et al. Toxicities of CAR T-cell therapy: recognition and management. Nat 
Rev Clin Oncol. 2018. 

 QOL-ONE PRO-CT                                Versione 1 del 06.10.2025 
 
P a g .  16 | 16 
 
8. Lee DW, et al. Current concepts in the diagnosis and management of cytokine release 
syndrome. Blood. 2014. 
9. Gust J, et al. Endothelial activation and blood-brain barrier disruption in neurotoxicity 
after CAR-T therapy. Cancer Discov. 2017. 
10. Wood WA, et al. Patient-reported outcomes in CAR-T therapy: longitudinal 
assessment. J Clin Oncol. 2021. 
11. Kröger N, et al. Quality of life after allo-SCT. Bone Marrow Transplant. 2011. 
12. Barata A, et al. Long-term outcomes and QoL after CAR-T therapy. Haematologica. 
2022. 
13. Sidana S, et al. Longitudinal Patient-Reported Outcomes with CAR-T Cell Therapy 
Versus Autologous and Allogeneic Stem Cell Transplant: PROs with CAR-T Therapy 
Versus Stem Cell Transplant. Transplantation and Cellular Therapy. 2022 Aug; 
28(8):473-482.  
14. Arther N, et al. Inpatient symptom burden in patients receiving CAR-T therapy. JCO 
Oncol Pract. 2022. 
15. Bevans M, Mitchell SA, Barrett JA. Symptom burden and supportive care needs in 
hematopoietic cell transplant patients. Cancer. 2008. 
16. Goswami P, et al. Development of a hematological malignancy-specific PRO measure 
(HM-PRO): content validity. Front Pharmacol. 2020. 
17. Goswami P, et al. Reliability of HM-PRO: internal consistency and test-retest 
validation. Front Pharmacol. 2020. 
18. Goswami P, et al. Construct validity of HM-PRO: correlation with EORTC QLQ-C30 and 
FACT-G. Front Pharmacol. 2020. 
19. Oliva EN, et al. Paper and electronic versions of HM-PRO: equivalence study. J Comp 
Eff Res. 2019. 
20. Andersen CL, et al. Danish translation and validation of HM-PRO. J Patient Rep 
Outcomes. 2025. 
21. FDA. Patient-Reported Outcome Measures: Use in Medical Product Development to 
Support Labeling Claims. Guidance for Industry. 2009. 
22. Berger ML, et al. Prospective observational studies to assess comparative 
effectiveness: ISPOR good research practices task force report. Value Health. 2012.