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Sickle Cell Transplantation Evaluation of Long-term and Late Effects Registry (STELLAR) to Compare Long-term Outcomes After Hematopoietic Cell Transplantation to Those in Siblings Without Sickle Cell Disease and in Nontransplanted Individuals With Sickle Cell Disease: Design and Feasibility Study.

Publication ,  Journal Article
Krishnamurti, L; Arnold, SD; Haight, A; Abraham, A; Guilcher, GM; John, T; Bakshi, N; Shenoy, S; Syrjala, K; Martin, PL; Chaudhury, S; Lane, P ...
Published in: JMIR Res Protoc
July 6, 2022

BACKGROUND: There are sparse data on the long-term and late effects of hematopoietic cell transplantation (HCT) for sickle cell disease (SCD). OBJECTIVE: This study aims to establish an international registry of long-term outcomes post-HCT for SCD and demonstrate the feasibility of recruitment at a single site in the United States. METHODS: The Sickle Cell Transplantation Evaluation of Long-Term and Late Effects Registry (STELLAR) was designed to enroll patients with SCD ≥1 year post-HCT, their siblings without SCD, and nontransplanted controls with SCD to collect web-based participant self-reports of health status and practices by using the Bone Marrow Transplant Survivor Study (BMTSS) surveys, health-related quality of life (HRQOL) using the Patient-Reported Outcomes Measurement Information System (PROMIS) Pediatric Profile-25 or Pediatric Profile-29 survey, chronic graft-versus-host disease (cGVHD) using the symptom scale survey, daily pain using an electronic pain diary, the economic impact of HCT using the financial hardship survey, sexual function using the PROMIS Sexual Function SexFSv2.0 survey, and economic productivity using the American Time Use Survey (ATUS). We also piloted retrieval of clinical data previously submitted to the Center for International Blood and Marrow Transplant Research (CIBMTR); recorded demographics, height, weight, blood pressure, waist and hip circumferences, timed up and go (TUG) test, and handgrip test; and obtained blood for metabolic screening, gonadal function, fertility potential, and biorepository of plasma, serum, RNA, and DNA. RESULTS: Of 100 eligible post-HCT patients, we enrolled 72 (72%) participants aged 9-38 (median 17) years. We also enrolled 19 siblings aged 5-32 (median 10) years and 28 nontransplanted controls with SCD aged 4-46 (median 22) years. Of the total 119 participants, 73 (61%) completed 85 sets of surveys and 41 (35%) contributed samples to the biorepository. We completed ATUS interviews of 28 (24%) participants. We successfully piloted retrieval of data submitted to the CIBMTR and expanded recruitment to multiple sites in the United States, Canada, the United Kingdom, and Nigeria. CONCLUSIONS: It is feasible to recruit subjects and conduct study procedures for STELLAR in order to determine the long-term and late effects of HCT for SCD. INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID): DERR1-10.2196/36780.

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Published In

JMIR Res Protoc

DOI

ISSN

1929-0748

Publication Date

July 6, 2022

Volume

11

Issue

7

Start / End Page

e36780

Location

Canada

Related Subject Headings

  • 4206 Public health
  • 4203 Health services and systems
  • 1117 Public Health and Health Services
  • 1103 Clinical Sciences
 

Citation

APA
Chicago
ICMJE
MLA
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Krishnamurti, Lakshmanan, Staci D. Arnold, Ann Haight, Allistair Abraham, Gregory Mt Guilcher, Tami John, Nitya Bakshi, et al. “Sickle Cell Transplantation Evaluation of Long-term and Late Effects Registry (STELLAR) to Compare Long-term Outcomes After Hematopoietic Cell Transplantation to Those in Siblings Without Sickle Cell Disease and in Nontransplanted Individuals With Sickle Cell Disease: Design and Feasibility Study.JMIR Res Protoc 11, no. 7 (July 6, 2022): e36780. https://doi.org/10.2196/36780.
Krishnamurti L, Arnold SD, Haight A, Abraham A, Guilcher GM, John T, Bakshi N, Shenoy S, Syrjala K, Martin PL, Chaudhury S, Eames G, Olowoselu OF, Hsieh M, De La Fuente J, Kasow KA, Stenger E, Mertens A, El-Rassi F, Lane P, Shaw BE, Meacham L, Archer D. Sickle Cell Transplantation Evaluation of Long-term and Late Effects Registry (STELLAR) to Compare Long-term Outcomes After Hematopoietic Cell Transplantation to Those in Siblings Without Sickle Cell Disease and in Nontransplanted Individuals With Sickle Cell Disease: Design and Feasibility Study. JMIR Res Protoc. 2022 Jul 6;11(7):e36780.

Published In

JMIR Res Protoc

DOI

ISSN

1929-0748

Publication Date

July 6, 2022

Volume

11

Issue

7

Start / End Page

e36780

Location

Canada

Related Subject Headings

  • 4206 Public health
  • 4203 Health services and systems
  • 1117 Public Health and Health Services
  • 1103 Clinical Sciences