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
BackgroundThere are sparse data on the long-term and late effects of hematopoietic cell transplantation (HCT) for sickle cell disease (SCD). ObjectiveThis study aims to establish an international registry of long-term outcomes post-HCT for SCD and demonstrate the...
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JMIR Publications
2022-07-01
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| Series: | JMIR Research Protocols |
| Online Access: | https://www.researchprotocols.org/2022/7/e36780 |
| _version_ | 1797734912985399296 |
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| author | Lakshmanan Krishnamurti Staci D Arnold Ann Haight Allistair Abraham Gregory MT Guilcher Tami John Nitya Bakshi Shalini Shenoy Karen Syrjala Paul L Martin Sonali Chaudhury Gretchen Eames Olusola Festus Olowoselu Matthew Hsieh Josu De La Fuente Kimberly A Kasow Elizabeth Stenger Anne Mertens Fuad El-Rassi Peter Lane Bronwen E Shaw Lillian Meacham David Archer |
| author_facet | Lakshmanan Krishnamurti Staci D Arnold Ann Haight Allistair Abraham Gregory MT Guilcher Tami John Nitya Bakshi Shalini Shenoy Karen Syrjala Paul L Martin Sonali Chaudhury Gretchen Eames Olusola Festus Olowoselu Matthew Hsieh Josu De La Fuente Kimberly A Kasow Elizabeth Stenger Anne Mertens Fuad El-Rassi Peter Lane Bronwen E Shaw Lillian Meacham David Archer |
| author_sort | Lakshmanan Krishnamurti |
| collection | DOAJ |
| description |
BackgroundThere are sparse data on the long-term and late effects of hematopoietic cell transplantation (HCT) for sickle cell disease (SCD).
ObjectiveThis 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.
MethodsThe 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.
ResultsOf 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.
ConclusionsIt 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 |
| first_indexed | 2024-03-12T12:51:12Z |
| format | Article |
| id | doaj.art-30e5d87919a54832b17b351f26b0d417 |
| institution | Directory Open Access Journal |
| issn | 1929-0748 |
| language | English |
| last_indexed | 2024-03-12T12:51:12Z |
| publishDate | 2022-07-01 |
| publisher | JMIR Publications |
| record_format | Article |
| series | JMIR Research Protocols |
| spelling | doaj.art-30e5d87919a54832b17b351f26b0d4172023-08-28T22:42:17ZengJMIR PublicationsJMIR Research Protocols1929-07482022-07-01117e3678010.2196/36780Sickle 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 StudyLakshmanan Krishnamurtihttps://orcid.org/0000-0003-3242-0037Staci D Arnoldhttps://orcid.org/0000-0001-6053-8521Ann Haighthttps://orcid.org/0000-0001-8158-0091Allistair Abrahamhttps://orcid.org/0000-0003-2019-0887Gregory MT Guilcherhttps://orcid.org/0000-0001-9380-4347Tami Johnhttps://orcid.org/0000-0003-0632-3559Nitya Bakshihttps://orcid.org/0000-0002-0876-9506Shalini Shenoyhttps://orcid.org/0000-0002-9283-607XKaren Syrjalahttps://orcid.org/0000-0001-8378-2797Paul L Martinhttps://orcid.org/0000-0001-8141-5678Sonali Chaudhuryhttps://orcid.org/0000-0001-9696-3159Gretchen Eameshttps://orcid.org/0000-0003-3274-3207Olusola Festus Olowoseluhttps://orcid.org/0000-0002-3125-6311Matthew Hsiehhttps://orcid.org/0000-0002-3706-6615Josu De La Fuentehttps://orcid.org/0000-0002-0974-1249Kimberly A Kasowhttps://orcid.org/0000-0002-0651-8666Elizabeth Stengerhttps://orcid.org/0000-0002-1386-6685Anne Mertenshttps://orcid.org/0000-0003-0858-2482Fuad El-Rassihttps://orcid.org/0000-0002-8583-0526Peter Lanehttps://orcid.org/0000-0001-5887-6222Bronwen E Shawhttps://orcid.org/0000-0003-3317-1896Lillian Meachamhttps://orcid.org/0000-0003-4394-0070David Archerhttps://orcid.org/0000-0002-9185-0865 BackgroundThere are sparse data on the long-term and late effects of hematopoietic cell transplantation (HCT) for sickle cell disease (SCD). ObjectiveThis 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. MethodsThe 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. ResultsOf 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. ConclusionsIt 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)DERR1-10.2196/36780https://www.researchprotocols.org/2022/7/e36780 |
| spellingShingle | Lakshmanan Krishnamurti Staci D Arnold Ann Haight Allistair Abraham Gregory MT Guilcher Tami John Nitya Bakshi Shalini Shenoy Karen Syrjala Paul L Martin Sonali Chaudhury Gretchen Eames Olusola Festus Olowoselu Matthew Hsieh Josu De La Fuente Kimberly A Kasow Elizabeth Stenger Anne Mertens Fuad El-Rassi Peter Lane Bronwen E Shaw Lillian Meacham David Archer 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 Research Protocols |
| title | 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 |
| title_full | 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 |
| title_fullStr | 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 |
| title_full_unstemmed | 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 |
| title_short | 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 |
| title_sort | 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 |
| url | https://www.researchprotocols.org/2022/7/e36780 |
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