L‐leucine improves anemia and growth in patients with transfusion‐dependent Diamond‐Blackfan anemia: Results from a multicenter pilot phase I/II study from the Diamond‐Blackfan Anemia Registry-Reference-Cited by-同舟云学术

L‐leucine improves anemia and growth in patients with transfusion‐dependent Diamond‐Blackfan anemia: Results from a multicenter pilot phase I/II study from the Diamond‐Blackfan Anemia Registry

Published:2020-10-06 Issue:12 Volume:67 Page:
ISSN:1545-5009
Container-title:Pediatric Blood & Cancer
language:en
Short-container-title:Pediatr Blood Cancer

Author:

Vlachos Adrianna¹²^ORCID,Atsidaftos Evangelia¹²,Lababidi Mohammad Lutfi¹,Muir Ellen¹²,Rogers Zora R.³,Alhushki Waseem⁴,Bernstein Jonathan⁵,Glader Bertil⁶,Gruner Barbara⁷,Hartung Helge⁸,Knoll Christine⁹,Loew Thomas¹⁰,Nalepa Grzegorz¹¹^ORCID,Narla Anupama⁶,Panigrahi Arun R.¹²,Sieff Colin A.¹³,Walkovich Kelly¹⁴,Farrar Jason E.¹⁵,Lipton Jeffrey M.¹²

Affiliation:

1. Feinstein Institutes for Medical Research Northwell Health Manhasset New York

2. Division of Hematology/Oncology and Cellular Therapy, Cohen Children's Medical Center Northwell Health New Hyde Park New York

3. University of Texas Southwestern Medical Center at Dallas Dallas Texas

4. Pediatric Hematology Oncology Cure4 The Kids Foundation Las Vegas Nevada

5. Pediatric Hematology Oncology Cure 4 The Kids Foundation, Las Vegas, Nevada; presently at Penn State Health Milton S. Hershey Medical Center Hershey Pennsylvania

6. Division of Pediatric Hematology/Oncology Stanford University School of Medicine Stanford California

7. Division of Pediatric Hematology/Oncology University of Missouri Columbia Missouri

8. Division of Hematology The Children's Hospital of Philadelphia Philadelphia Pennsylvania

9. The Center for Cancer and Blood Disorders Phoenix Children's Hospital Phoenix Arizona

10. Division of Pediatric Hematology/Oncology University of Missouri, Columbia, Missouri; presently at University of Kansas Medical Center Kansas City Kansas

11. Department of Pediatric Hematology‐Oncology Indiana University School of Medicine Indianapolis Indiana

12. University of Louisville, Louisville, Kentucky; presently at University of California Davis Sacramento California

13. Harvard Medical School, Dana‐Farber and Boston Children's Cancer and Blood Disorders Center Boston Massachusetts

14. Division of Hematology/Oncology C.S. Mott Children's Hospital Ann Arbor Michigan

15. Pediatric Hematology/Oncology Arkansas Children's Research Institute and University of Arkansas for Medical Sciences Little Rock Arkansas

Publisher

Wiley

Subject

Oncology,Hematology,Pediatrics, Perinatology, and Child Health

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/pbc.28748

Reference14 articles.

1. Diamond Blackfan anemia: a model for the translational approach to understanding human disease

2. The Genetic Landscape of Diamond-Blackfan Anemia

3. Diamond-Blackfan anemia with mandibulofacial dystostosis is heterogeneous, including the novel DBA genesTSR2andRPS28

4. Exome sequencing identifies GATA1 mutations resulting in Diamond-Blackfan anemia

5. Aplasticanemia (chronicerythroblastophthisis) and cortisone;Gasser C;Schweiz Med Wochenschr,1951

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