Patient-Reported Outcomes (PRO) Data Collection
What are PRO data?
PRO are defined by the FDA1 as “any report of the status of a patient's health condition that comes directly from the patient, without interpretation of the patient's response by a clinician or anyone else.” Participant- (including patients, donors, caregivers, and others) reported outcomes (collectively referred to as PRO) are collected from subjects and include quality of life (QOL), biologic and physiologic variables, symptom status, functional and financial status, and general health perceptions. PRO have gained acceptance as outcomes that supplement traditional survival endpoints. PRO have been shown to improve engagement and facilitate patient-physician discussion among cancer patients.2 In addition, studies have demonstrated that PRO add value in determining HCT outcomes, including survival and QOL, and that QOL post-HCT or post-cellular therapy can be accurately assessed by PRO, and groups at risk for poor QOL can be identified.3
How CIBMTR collects PRO data
The CIBMTR Survey Research Group centrally collects PRO from participants electronically, on paper, and by phone. Survey Research Group staff members contact participants directly to administer PRO surveys and follow-up with non-responders. Staff members may also consent and enroll patients in minimal-risk, PRO-only studies. The Survey Research Group also manages PRO data within the CIBMTR's electronic PRO (ePRO) system, building and maintaining PRO survey forms, reviewing data for completeness, managing data transfers, and preparing extracts for analysis.
Patient-Reported Outcomes Measurement Information System (PROMIS®) is a set of measures that evaluate physical, mental, and social health in adults and children. PROMIS measures form the backbone of the CIBMTR ePRO system and most PRO surveys. Electronically and by phone, these measures are administered via computer adaptive testing through an application programming interface (API). Paper PROMIS measures are administered as static short forms.
The Survey Research Group manages the CIBMTR ePRO system, which integrates multiple applications: A user-friendly ePRO interface in Qualtrics®; a widely used proprietary software; automated tracking and alerting functionality in Salesforce®, the client-relationship management system used by CIBMTR that communicates directly with FormsNet; and an API. The system is scalable, allowing additional measures (including financial toxicity, occupational functioning, and socio-demographics) to be collected to study specific study aims or long-term survivorship issues.
CIBMTR PRO protocol
Since 2020, CIBMTR has managed the Protocol for Collection of Patient Reported Outcomes (PRO) Data. Under this protocol, CIBMTR collects a routine set of PRO data, which is then added to and governed by the CIBMTR Observational Database Protocol.
Data from the CIBMTR PRO protocol is available for any Working Committee or other CIBMTR observational study. PRO data from select clinical trials may also be available.
Clinical trials and other studies
CIBMTR supports many clinical trials with PRO data collection. During the protocol development phase, the Survey Research Group manager will work with CIBMTR scientific leadership to support the protocol team in the selection of PRO measures to meet the aims of the study and draft PRO-related sections of the protocol, consent form, and any study plans. In BMT CTN trials, a PRO Working Group will ensure the protocol team includes a PRO expert to help write, review, and approve sections relevant to PROs in all draft protocols. The BMT CTN PRO Working Group will also lend expertise in PRO instrument selection, assessment, monitoring, and analysis.
A Survey Research Group survey data analyst will develop the survey in the ePRO database along with paper versions and communication templates, and support translation into required languages. Over the course of a clinical trial, the Survey Research Group centrally collects PRO data from participants, and staff members track and communicate PRO compliance with the protocol team.
Clinical trials that include PRO collection
| Study name | Sponsor | Population | PRO time points | PRO content |
|---|---|---|---|---|
| 0201 - PBSC vs Marrow - QoL | BMT CTN | Adult HCT recipients | Pre-HCT, 6 month, 1 year, 2 year, 5 year | Global QOL, FACT-BMT, Mental Health Inventory (MHI), occupational functioning, chronic GVHD module |
| 09-SQOL | CIBMTR | Adult and pediatric HCT recipients | Pre-HCT, Day 100, Day 180, Day 365 | |
| 1102 - MDS | BMT CTN | Adult MDS patients, age 50-75, seeking HCT | At enrollment, 6 months, 12 months, 18 months, 24 months, 36 months post-enrollment | EQ-5D, FACT-G, SF-36 |
| 13-SCP (Survivorship Care Plan) | NMDP | Adult HCT recipients, 2-5 years post-HCT | Pre-enrollment, 6 months post-enrollment | Confidence in survivorship, cancer treatment distress, knowledge of transplant exposure, healthcare utilization, health behaviors, SF-12, health self-efficacy, care plan use |
| Palliative Care Study | Massachusetts General Hospital | Adult HCT recipients | Single time-point, 3-12 months post-HCT | PROMIS, perceptions of supportive / palliative care, palliative care needs assessment, palliative care knowledge scale |
| 1702 – CTRL-ALT-D / Donor Source | BMT CTN | Adult and pediatric HCT recipients | Pre-HCT, 1 year, 2 year | PROMIS, Lee chronic GVHD symptom scale |
| 1703 – PROGRESS III | BMT CTN | Adult HCT recipients | Pre-HCT, Day 100, Day 180, Day 365 | PROMIS, Lee chronic GVHD symptom scale |
| 1704 - CHARM | BMT CTN | Adult HCT recipients | Pre-HCT, Day 100, Day 180, Day 365 | PROMIS, KPS, IADL, frailty, physical activities |
| ACCESS | NMDP | Adult and pediatric HCT recipients | Pre-HCT, Day 100, Day 180, Day 365 | PROMIS, Lee cGVHD symptom scale, Comprehensive Score of financial Toxicity (COST) |
| 1904 – BMFD | BMT CTN | Adult and pediatric HCT recipients | Pre-HCT, Day 180, Day 365 | PROMIS |
| 2001 – GT in SCD | BMT CTN | Adult and pediatric GT recipients | Pre-GT, Day 730 | PROMIS, Household Material Hardship |
To propose a study utilizing PRO surveys, visit the Propose a Working Committee Study page.
Contacts and references
- Deborah Mattila, Survey Research Group Manager, dmattila@nmdp.org
- Rachel Cusatis, PhD, Senior Scientific Director of Patient-Centered Research and Survivorship, rcusatis@mcw.edu
- Visit the Propose a Working Committee Study page to propose a study using existing PRO data. Contact Deborah Mattila and Rachel Cusatis to propose new study-specific PRO surveys.
- Visit the PRO Data Collection Protocol page to see which data have been collected and are available for research
Citations
1Food, U. S., Administration, D. & Others. Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims. Fed. Regist. 74, 65132–65133 (2009).
2Howell, D. et al. Patient-reported outcomes in routine cancer clinical practice: A scoping review of use, impact on health outcomes, and implementation factors. Annals of Oncology vol. 26 1846–1858 (2015).
3.1Mussetti, A. et al. Prospective Pilot Study of Telehealth as Domiciliary Follow-up after Hematopoietic Cell Transplantation during the COVID19 Pandemic (Preprint). JMIR Form. Res. (2020) doi:10.2196/26121.
3.2Detmar, S. B., Muller, M. J., Schornagel, J. H., Wever, L. D. V. & Aaronson, N. K. Health-Related Quality-of-Life Assessments and Patient-Physician Communication: A Randomized Controlled Trial. JAMA 288, 3027–3034 (2002).
3.3Pidala, J., Anasetti, C. & Jim, H. Health-related quality of life following haematopoietic cell transplantation: patient education, evaluation and intervention. Br. J. Haematol. 148, 373–385 (2010).