National U.S. Patient and Transplant Data for Krabbe Disease
Krabbe disease (KD) is a leukodystrophy caused by mutations in the galactosylceramidase gene. Presymptomatic hematopoietic stem cell transplantation (HSCT) is associated with improved outcomes, but most data are from single-center studies. Our objective was to characterize national patterns of HSCT...
Guardado en:
| Autores principales: | , , |
|---|---|
| Formato: | article |
| Lenguaje: | EN |
| Publicado: |
Frontiers Media S.A.
2021
|
| Materias: | |
| Acceso en línea: | https://doaj.org/article/b015126da5714d1aad426af2846253cf |
| Etiquetas: |
Agregar Etiqueta
Sin Etiquetas, Sea el primero en etiquetar este registro!
|
| id |
oai:doaj.org-article:b015126da5714d1aad426af2846253cf |
|---|---|
| record_format |
dspace |
| spelling |
oai:doaj.org-article:b015126da5714d1aad426af2846253cf2021-11-11T08:03:57ZNational U.S. Patient and Transplant Data for Krabbe Disease2296-236010.3389/fped.2021.764626https://doaj.org/article/b015126da5714d1aad426af2846253cf2021-11-01T00:00:00Zhttps://www.frontiersin.org/articles/10.3389/fped.2021.764626/fullhttps://doaj.org/toc/2296-2360Krabbe disease (KD) is a leukodystrophy caused by mutations in the galactosylceramidase gene. Presymptomatic hematopoietic stem cell transplantation (HSCT) is associated with improved outcomes, but most data are from single-center studies. Our objective was to characterize national patterns of HSCT for KD including whether there were disparities in HSCT utilization and outcomes. We conducted a retrospective study of KD patients ≤ age 18 years from November 1, 2015, through December 31, 2019, using the U.S. Children's Hospital Association's Pediatric Health Information System database. We evaluated outcomes for HSCT, intensive care unit days, and mortality, comparing age, sex, race/ethnicity, rural/urban location, and median household income. We identified 91 KD patients. HSCT, performed in 32% of patients, was associated with reduced mortality, 31 vs. 68% without HSCT (p < 0.003). Trends included the fact that more males than females had HSCT (39 vs. 23%); more Asian and White patients had HSCT compared to Black or Hispanic patients (75, 33, 25, and 17%, respectively); and patients from households with the lowest-income quartile (< $25,000) had more HSCT compared to higher-income quartiles (44 vs. 33, 30, and 0%). Overall, receiving HSCT was associated with reduced mortality. We noted trends in patient groups who received HSCT. Our findings suggest that disparities in receiving HSCT could affect outcomes for KD patients.Gabrielle GhabashJacob WilkesJoshua L. BonkowskyJoshua L. BonkowskyFrontiers Media S.A.articleleukodystrophyKrabbe disease (globoid cell leukodystrophy)pediatric neurologyhematopoietic stem cell transplantationdisparities (health racial)PediatricsRJ1-570ENFrontiers in Pediatrics, Vol 9 (2021) |
| institution |
DOAJ |
| collection |
DOAJ |
| language |
EN |
| topic |
leukodystrophy Krabbe disease (globoid cell leukodystrophy) pediatric neurology hematopoietic stem cell transplantation disparities (health racial) Pediatrics RJ1-570 |
| spellingShingle |
leukodystrophy Krabbe disease (globoid cell leukodystrophy) pediatric neurology hematopoietic stem cell transplantation disparities (health racial) Pediatrics RJ1-570 Gabrielle Ghabash Jacob Wilkes Joshua L. Bonkowsky Joshua L. Bonkowsky National U.S. Patient and Transplant Data for Krabbe Disease |
| description |
Krabbe disease (KD) is a leukodystrophy caused by mutations in the galactosylceramidase gene. Presymptomatic hematopoietic stem cell transplantation (HSCT) is associated with improved outcomes, but most data are from single-center studies. Our objective was to characterize national patterns of HSCT for KD including whether there were disparities in HSCT utilization and outcomes. We conducted a retrospective study of KD patients ≤ age 18 years from November 1, 2015, through December 31, 2019, using the U.S. Children's Hospital Association's Pediatric Health Information System database. We evaluated outcomes for HSCT, intensive care unit days, and mortality, comparing age, sex, race/ethnicity, rural/urban location, and median household income. We identified 91 KD patients. HSCT, performed in 32% of patients, was associated with reduced mortality, 31 vs. 68% without HSCT (p < 0.003). Trends included the fact that more males than females had HSCT (39 vs. 23%); more Asian and White patients had HSCT compared to Black or Hispanic patients (75, 33, 25, and 17%, respectively); and patients from households with the lowest-income quartile (< $25,000) had more HSCT compared to higher-income quartiles (44 vs. 33, 30, and 0%). Overall, receiving HSCT was associated with reduced mortality. We noted trends in patient groups who received HSCT. Our findings suggest that disparities in receiving HSCT could affect outcomes for KD patients. |
| format |
article |
| author |
Gabrielle Ghabash Jacob Wilkes Joshua L. Bonkowsky Joshua L. Bonkowsky |
| author_facet |
Gabrielle Ghabash Jacob Wilkes Joshua L. Bonkowsky Joshua L. Bonkowsky |
| author_sort |
Gabrielle Ghabash |
| title |
National U.S. Patient and Transplant Data for Krabbe Disease |
| title_short |
National U.S. Patient and Transplant Data for Krabbe Disease |
| title_full |
National U.S. Patient and Transplant Data for Krabbe Disease |
| title_fullStr |
National U.S. Patient and Transplant Data for Krabbe Disease |
| title_full_unstemmed |
National U.S. Patient and Transplant Data for Krabbe Disease |
| title_sort |
national u.s. patient and transplant data for krabbe disease |
| publisher |
Frontiers Media S.A. |
| publishDate |
2021 |
| url |
https://doaj.org/article/b015126da5714d1aad426af2846253cf |
| work_keys_str_mv |
AT gabrielleghabash nationaluspatientandtransplantdataforkrabbedisease AT jacobwilkes nationaluspatientandtransplantdataforkrabbedisease AT joshualbonkowsky nationaluspatientandtransplantdataforkrabbedisease AT joshualbonkowsky nationaluspatientandtransplantdataforkrabbedisease |
| _version_ |
1718439342250131456 |