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Center for International Blood and Marrow Transplant Research

WARRIOR-SCD, a Cure Sickle Cell Initiative Long Term Follow-Up Study

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WARRIOR-SCD, Whole-person Assessment of Response and Risk after transformational therapies: Investigating Outcomes and Resilience in Sickle Cell Disease, a Cure Sickle Cell (SCD) Initiative Study is an observational study that follows individuals with SCD who have received gene therapy (GT) or hematopoietic cell transplantation (HCT) as part of a clinical trial or standard-of-care treatment to evaluate the long-term safety and effectiveness of these therapies. 

The study is part of the NHLBI-supported SCD Long-Term Follow-Up (LTFU) Consortium, which includes two cohorts: (1) a non-curative/non-transformative therapy cohort managed by the SCD Natural History Data Resource (SCD NHDR), and (2) a transformative/curative therapy cohort of patients treated with GT or HCT, developed by CIBMTR. 

For the GT/HCT cohort, CIBMTR will collect clinical data from the medical record, patient-reported outcomes (PROs), and biospecimens collected at participating treatment centers. Biospecimens will be collected under the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) 2402 Hematopoietic Cell Transplant and Gene Therapy for Non-Malignant Blood Disorders Biobank Resource (HOPE Resource) Protocol at centers who activate that study; it is recommended, but not required, to activate the HOPE protocol to participate in the LTFU study.  Through the collaboration with the SCD NHDR, outcomes for GT and HCT recipients will be compared with those of similar patients who did not receive these transformative therapies. 

On this page:

Center Enrollment: July 2026 - October 2026

Patient Enrollment: Begins October 26, 2026

Study Objectives

The primary objective of this study is to collect long-term follow-up data from individuals with SCD who have received GT or HCT. The study will focus on the following clinical outcomes of interest, with specific details pertaining to each outcome outlined below. 

  • Acute and chronic pain 
  • End-organ function, including brain, lung, heart, kidney, and endocrine 
  • Other specific complications of SCD (e.g., retinopathy, priapism) 
  • Complications of conditioning and the GT and HCT therapies 
  • Development of malignancy 
  • Reproductive health 
  • Quality of life 
  • Death

Eligibility

Center:

  • Existing CIBMTR Network Center

Patient:

  • Inclusion
    • Individuals who receive gene therapy or HCT for SCD on a NHLBI funded clinical trial in 2017 or later, or
    • Individuals who received or are scheduled to receive gene therapy or HCT for SCD in 2023 or later, either as standard-of-care or on an investigational protocol at participating centers
    • Individuals who signed (or legal guardian signed) the CIBMTR Research Database Protocol consent and assent as age appropriate
  • Exclusion
    • Individual (or legal guardian signed) did not provide consent to the CIBMTR Research Database Protocol 

Protocols and Consents

This study uses the existing CIBMTR Research Database and PRO Data Collection Protocols and Consents to collect study data. Collection of specimens uses the BMT CTN 2402 HOPE Resource Protocol and Consents. Centers do not need to submit a separate WARRIOR-SCD study protocol or consent form to their local IRB for their patients to participate.

Patient-Reported Outcomes (PRO)

CIBMTR will collect patient-reported outcome (PRO) data from patients who consent to participate in the PRO portion of this study, to better understand long-term impacts and improve patient quality of life. This study follows the standard topics and time points for PRO data collection, with some additional SCD-specific questions. The CIBMTR will manage consenting and data collection for this part of the study; there is no additional work requirement for centers.

The PRO surveys include questions about the below topics: 

  • Anxiety

  • Depression

  • Pain Interference

  • Pain Behavior

  • Pain Quality

  • Fatigue

  • Social Roles and Activities (ADULT ONLY)

  • Physical Function (ADULT ONLY)

  • Mobility (PEDS ONLY)

  • Sleep Disturbance

  • Sexual Function (ADULT ONLY)

  • ASCQ-Me Pain Episodes (15 years and older)

  • Family Relationships (PEDS ONLY)

  • Peer Relationships (PEDS ONLY)

  • Financial toxicity (ADULT, PARENT/GUARDIAN ONLY)

  • Sociodemographic information (ADULT, PARENT/GUARDIAN ONLY)

For participants under 18 years of age, a parent or legal guardian will be required to complete a short survey collecting financial and demographic information at most timepoints.

CIBMTR Center Requirements

Eligible centers are expected to complete the following clinical data, patient-reported outcomes (PRO) data, and sample collection activities:

Clinical Data Collection

CRF-level gene therapy or HCT data reporting, as well as submission of study-specific supplemental data for 15 years, including uploading selected reports and test results.

  1. Obtain CIBMTR Research Database Consent
  2. Obtain consent and assent, as age appropriate, from eligible SCD patients to participate in the CIBMTR Research Database Protocol and authorize submission of their data to CIBMTR.
    • Inform patients that participation in the compensated PRO portion of the study requires that they check the box for future contact by CIBMTR on the CIBMTR Research Database Protocol consent form.

Patient-Reported Outcomes (PRO) Data Collection

  • Inform patients about the opportunity for PRO Study Participation at the time of Research Database Protocol consent and provide eligible patients and/or their parent/guardian with the study flyer.
    • If the patient elects to future contact by CIBMTR on the CIBMTR Research Database Protocol consent, explain that CIBMTR will contact them separately to obtain consent for the PRO portion of the study.
    • Note: Study flyers have been approved by the central NMDP IRB.
  • Participants who complete the PRO surveys will receive a $50 gift card for the initial survey and $25 gift card for each subsequent survey completed during the study period.

Sample Collection

Centers and patients participating in this study are encouraged to also elect to participate in BMT CTN Protocol 2402, HOPE Resource, if that protocol is active at their center.  Note: sample collection participation is not required to participate in the Research Database Protocol-governed part of this study.

  1. Obtain BMT CTN 2402 Protocol Consent, if patient elects to participate

Study Incentives and Reimbursement

  • Incentive: $5000 one-time payment upon enrollment of first CRID and supplemental data collection 
  • Incentive: $5300 per CRID enrolled after submission of pre-infusion supplemental data forms
  • Incentive: $5000 per CRID enrolled in the BMT CTN 2402 Protocol
  • Standard form reimbursement rates will apply
  • Study-specific supplemental forms: $215 for each pre and post infusion form
  • Clinical Report uploads: $25 per time point

For further information and reporting directions, CIBMTR centers participating in the study should log into the CIBMTR Portal and refer to the Study Resources tile.

Study Contacts

Reference Materials

English Speaking Patient Flyers

Spanish Speaking Patient Flyers