TY - JOUR
T1 - Sickle Cell Clinical Research and Intervention Program (SCCRIP)
T2 - A lifespan cohort study for sickle cell disease progression from the pediatric stage into adulthood
AU - Hankins, Jane S.
AU - Estepp, Jeremie H.
AU - Hodges, Jason R.
AU - Villavicencio, Martha A.
AU - Robison, Leslie L.
AU - Weiss, Mitchell J.
AU - Kang, Guolian
AU - Schreiber, Jane E.
AU - Porter, Jerlym S.
AU - Kaste, Sue C.
AU - Saving, Kay L.
AU - Bryant, Paulette C.
AU - Deyo, Jeffrey E.
AU - Nottage, Kerri A.
AU - King, Allison A.
AU - Brandow, Amanda M.
AU - Lebensburger, Jeffrey D.
AU - Adesina, Oyebimpe
AU - Chou, Stella T.
AU - Zemel, Babette S.
AU - Smeltzer, Matthew P.
AU - Wang, Winfred C.
AU - Gurney, James G.
N1 - Funding Information:
We thank the following individuals from St. Jude Children's Research Hospital: Chris Vukadinovich, Jennifer Lanctot, Pei-Lin Chen, and Shannon Wright for coding and programming of databases and systems; Tiana Thomas, Madelene Wilson, Gail Fortner, Ashley Coley, and Ivanka Rankovic for data collection; Kathleen Helton, Scott Hwang, Nicole Alberts, Lisa Jacola, Latika Puri, Doralina Anghelescue, Daniel Garrison, Wassim Chemaitilly, Yan Zheng, Kevin Krull, Sean Phipps, John Brooke, Yutaka Yatsui, Evadnie Rampersaud, Gang Wu, and Kenneth Ataga, for intellectual input on planning future initiatives; and Courtney Mays and Teresa Carr for support with study infrastructure and regulatory matters.
Funding Information:
J.H.E. receives research support from Pfizer and Eli Lilly and Co. and serves as a consultant for Daiichi Sankyo and Global Blood Therapeutics. W.C.W. receives research support from Global Blood Therapeutics. J.S.H. receives research support from Novartis and Global Blood Therapeutics and consultant fees from bluebird bio. K.L.S. receives financial support from Molina Healthcare Clinical Quality Improvement Committee to conduct quality improvement projects, and owns Pfizer stocks. P.C.B. receives research support and speaker fees from Novo Nordisk. M.J.W. is a consultant for Glaxo SmithKline and Novartis and an advisory board member for Rubius, and receives research funding from Biogen. The other authors have no financial relationships relevant to this article to disclose.
Publisher Copyright:
© 2018 Wiley Periodicals, Inc.
PY - 2018/9
Y1 - 2018/9
N2 - Background: Previous natural history studies have advanced the understanding of sickle cell disease (SCD), but generally have not included sufficient lifespan data or investigation of the role of genetics in clinical outcomes, and have often occurred before the widespread use of disease-modifying therapies, such as hydroxyurea and chronic erythrocyte transfusions. To further advance knowledge of SCD, St. Jude Children's Research Hospital established the Sickle Cell Clinical Research and Intervention Program (SCCRIP), to conduct research in a clinically evaluated cohort of individuals with SCD across their lifetime. Procedures: Initiated in 2014, the SCCRIP study prospectively recruits patients diagnosed with SCD and includes retrospective and longitudinal collection of clinical, neurocognitive, geospatial, psychosocial, and health outcomes data. Biological samples are banked for future genomics and proteomics studies. The organizational structure of SCCRIP is based upon organ/system-specific working groups and is opened to the research community for partnerships. Results: As of August 2017, 1,044 (92.3% of eligible) patients with SCD have enrolled in the study (860 children and 184 adults), with 11,915 person-years of observation. Population demographics included mean age at last visit of 11.3 years (range 0.7–30.1), 49.8% females, 57.7% treated with hydroxyurea, 8.5% treated with monthly transfusions, and 62.9% hemoglobin (Hb) SS or HbSB 0 -thalassemia, 25.7% HbSC, 8.4% HbsB + -Thalassemia, 1.7% HbS/HPFH, and 1.2% other. Conclusions: The SCCRIP cohort will provide a rich resource for the conduct of high impact multidisciplinary research in SCD.
AB - Background: Previous natural history studies have advanced the understanding of sickle cell disease (SCD), but generally have not included sufficient lifespan data or investigation of the role of genetics in clinical outcomes, and have often occurred before the widespread use of disease-modifying therapies, such as hydroxyurea and chronic erythrocyte transfusions. To further advance knowledge of SCD, St. Jude Children's Research Hospital established the Sickle Cell Clinical Research and Intervention Program (SCCRIP), to conduct research in a clinically evaluated cohort of individuals with SCD across their lifetime. Procedures: Initiated in 2014, the SCCRIP study prospectively recruits patients diagnosed with SCD and includes retrospective and longitudinal collection of clinical, neurocognitive, geospatial, psychosocial, and health outcomes data. Biological samples are banked for future genomics and proteomics studies. The organizational structure of SCCRIP is based upon organ/system-specific working groups and is opened to the research community for partnerships. Results: As of August 2017, 1,044 (92.3% of eligible) patients with SCD have enrolled in the study (860 children and 184 adults), with 11,915 person-years of observation. Population demographics included mean age at last visit of 11.3 years (range 0.7–30.1), 49.8% females, 57.7% treated with hydroxyurea, 8.5% treated with monthly transfusions, and 62.9% hemoglobin (Hb) SS or HbSB 0 -thalassemia, 25.7% HbSC, 8.4% HbsB + -Thalassemia, 1.7% HbS/HPFH, and 1.2% other. Conclusions: The SCCRIP cohort will provide a rich resource for the conduct of high impact multidisciplinary research in SCD.
KW - disease-modifying therapy
KW - natural history
KW - sickle cell anemia
UR - http://www.scopus.com/inward/record.url?scp=85050383963&partnerID=8YFLogxK
U2 - 10.1002/pbc.27228
DO - 10.1002/pbc.27228
M3 - Article
C2 - 29797644
AN - SCOPUS:85050383963
SN - 1545-5009
VL - 65
JO - Pediatric Blood and Cancer
JF - Pediatric Blood and Cancer
IS - 9
M1 - e27228
ER -