Grandchildren of GRNDaD: Shifts in disease-modifying therapy at the adolescent transition in sickle cell disease

Matthew Chang, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
Daniel Semakula, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
Jane A. Little, University of North Carolina, Chapel Hill, North Carolina, USA.
Julie Kanter, University of Alabama at Birmingham, Birmingham, Alabama, USA.
Deepa G. Manwani, Children's Hospital at Montefiore, Albert Einstein College of Medicine, Bronx, New York, USA.
Amma Owusu-Ansah, UH Rainbow Babies & Children's Hospital, Cleveland, Ohio, USA.
Alice J. Cohen, Newark Beth Israel Medical Center, Newark, New Jersey, USA.
Robert M. Cronin, The Ohio State University Wexner Medical Center, Grandview Heights, Ohio, USA.
Payal C. Desai, Wake Forest School of Medicine, Atrium Health, Levine Cancer Institute, Charlotte, North Carolina, USA.
John J. Strouse, Duke University School of Medicine, Durham, North Carolina, USA.
Lisa M. Shook, Cincinnati Children's Hospital, Cincinnati, Ohio, USA.
Farzana Sayani, University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania, USA.
Dana LeBlanc, LSU Health Sciences Center - New OrleansFollow
Marsha J. Treadwell, University of California San Francisco School of Medicine, San Francisco, California, USA.
Marisol Betensky, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
Stephanie H. Guarino, ChristianaCare Health System, Wilmington, Delaware, USA.
Molly W. Mandernach, University of Florida College of Medicine, Gainesville, Florida, USA.
Alan R. Anderson, Prisma Health, University of South Carolina, Greenville, South Carolina, USA.
Seethal A. Jacob, Indiana University School of Medicine, Indianapolis, Indiana, USA.
Suzanne Saccente, University of Arkansas for Medical Sciences/Arkansas Children's Hospital, Little Rock, Arkansas, USA.
Ashok B. Raj, Norton Children's Hospital, University of Louisville, Louisville, Kentucky, USA.
Ofelia A. Alvarez, University of Miami Health System, Miami, Florida, USA.
Sana Saif Ur Rehman, Washington University School of Medicine in Saint Louis, St Louis, Missouri, USA.
Sanjay Shah, Phoenix Children's Hospital, Phoenix, Arizona, USA.
Sophie M. Lanzkron, Thomas Jefferson University, Philadelphia, Pennsylvania, USA.

Document Type

Article

Publication Date

7-31-2025

Publication Title

British journal of haematology

Abstract

Characterizing the modern person living with sickle cell disease (SCD) in the United States has been limited without a well-curated longitudinal registry. To address this, the Globin Research Network for Data and Discovery (GRNDaD) registry strives to collect clinical outcomes and quality of life metrics from Institutional Review Board-approved centres across the United States. Here, we examined the use of different disease-modifying therapies in (actively consented) adults and children with HgbSS and HgbS-β0 thalassaemia (SCA) from 38 sites. Of the 3169 active patients in GRNDaD, about 65% of subjects were on hydroxyurea (hydroxycarbamide; HU), and 2130 had SCA. As predicted, the absolute neutrophil counts were lower and mean corpuscular volumes were higher for patients on HU. However, there was a lower proportion of patients on HU in older age groups. In contrast, chronic RBC transfusion utilization was nearly twice as high in the 18- to 29-year-old age group than in the 11- to 17-year-old age group. For novel therapeutics, we examined use prior to voxelotor's removal from the market and prior to publication of the negative phase III trial of crizanlizumab. Voxelotor utilization in this cohort was three times that reported by claims data while crizanlizumab usage was nearly double, suggesting high-quality comprehensive sickle cell care could increase utilization of novel therapies.

PubMed ID

40741658

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