Treatment of newly diagnosed severe aplastic anemia in children: Evidence-based recommendations

Kristin A Shimano¹, Jennifer A Rothman², Steven W Allen³, Paul Castillo⁴, Jill L O de Jong⁵, Yigal Dror⁶, Amy E Geddis⁷, Bonnie W Lau⁸, Catherine McGuinn⁹, Anupama Narla¹⁰, Kathleen Overholt¹¹, Maria A Pereda¹², Anjali Sharathkumar¹³, Ghadir Sasa¹⁴, Taizo A Nakano¹², Kasiani Myers¹⁵, Nicholas J Gloude¹⁶, Larisa Broglie¹⁷, Jessica Boklan¹⁸

Affiliations

¹ Department of Pediatrics, Division of Allergy, Immunology, and Bone Marrow Transplant, University of California San Francisco Benioff Children's Hospital, San Francisco, California, USA.
² Department of Pediatrics, Division of Pediatric Hematology/Oncology, Duke University Medical Center, Durham, North Carolina, USA.
³ Department of Pediatrics, Pediatric Hematology/Oncology, University of Pittsburgh School of Medicine, UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, USA.
⁴ Department of Pediatrics, Division of Pediatric Hematology Oncology, UF Health Shands Children's Hospital, Gainesville, Florida, USA.
⁵ Department of Pediatrics, Section of Hematology/Oncology/Stem Cell Transplantation, University of Chicago, Chicago, Illinois, USA.
⁶ Department of Pediatrics, Marrow Failure and Myelodysplasia Program, Division of Hematology/Oncology, The Hospital for Sick Children, Toronto, Canada.
⁷ Department of Pediatrics, Cancer and Blood Disorders Center, Seattle Children's Hospital, Seattle, Washington, USA.
⁸ Department of Pediatrics, Pediatric Hematology-Oncology, Dartmouth-Hitchcock, Lebanon, New Hampshire, USA.
⁹ Department of Pediatrics, Division of Pediatric Hematology Oncology, Weill Cornell Medicine, New York, New York, USA.
¹⁰ Department of Pediatrics, Stanford University School of Medicine, Stanford, California, USA.
¹¹ Department of Pediatrics, Pediatric Hematology/Oncology, Riley Hospital for Children at Indiana University, Indianapolis, Indiana, USA.
¹² Department of Pediatrics, Center for Cancer and Blood Disorders, Children's Hospital Colorado, Aurora, Colorado, USA.
¹³ Stead Family Department of Pediatrics, University of Iowa Carver College of Medicine, Iowa City, Iowa, USA.
¹⁴ Sarah Cannon Transplant and Cellular Therapy Network, San Antonio, Texas, USA.
¹⁵ Department of Pediatrics, University of Cincinnati College of Medicine, Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.
¹⁶ Department of Pediatrics, University of California San Diego, Rady Children's Hospital, San Diego, California, USA.
¹⁷ Department of Pediatric Hematology/Oncology/Blood and Marrow Transplantation, Medical College of Wisconsin, Milwaukee, Wisconsin, USA.
¹⁸ Department of Pediatrics, Center for Cancer and Blood Disorders, Phoenix Children's Hospital, Phoenix, Arizona, USA.

PMID: 38757488
DOI: 10.1002/pbc.31070

Review

Treatment of newly diagnosed severe aplastic anemia in children: Evidence-based recommendations

Kristin A Shimano et al. Pediatr Blood Cancer. 2024 Aug.

. 2024 Aug;71(8):e31070.

doi: 10.1002/pbc.31070. Epub 2024 May 16.

Authors

Affiliations

¹ Department of Pediatrics, Division of Allergy, Immunology, and Bone Marrow Transplant, University of California San Francisco Benioff Children's Hospital, San Francisco, California, USA.
² Department of Pediatrics, Division of Pediatric Hematology/Oncology, Duke University Medical Center, Durham, North Carolina, USA.
³ Department of Pediatrics, Pediatric Hematology/Oncology, University of Pittsburgh School of Medicine, UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, USA.
⁴ Department of Pediatrics, Division of Pediatric Hematology Oncology, UF Health Shands Children's Hospital, Gainesville, Florida, USA.
⁵ Department of Pediatrics, Section of Hematology/Oncology/Stem Cell Transplantation, University of Chicago, Chicago, Illinois, USA.
⁶ Department of Pediatrics, Marrow Failure and Myelodysplasia Program, Division of Hematology/Oncology, The Hospital for Sick Children, Toronto, Canada.
⁷ Department of Pediatrics, Cancer and Blood Disorders Center, Seattle Children's Hospital, Seattle, Washington, USA.
⁸ Department of Pediatrics, Pediatric Hematology-Oncology, Dartmouth-Hitchcock, Lebanon, New Hampshire, USA.
⁹ Department of Pediatrics, Division of Pediatric Hematology Oncology, Weill Cornell Medicine, New York, New York, USA.
¹⁰ Department of Pediatrics, Stanford University School of Medicine, Stanford, California, USA.
¹¹ Department of Pediatrics, Pediatric Hematology/Oncology, Riley Hospital for Children at Indiana University, Indianapolis, Indiana, USA.
¹² Department of Pediatrics, Center for Cancer and Blood Disorders, Children's Hospital Colorado, Aurora, Colorado, USA.
¹³ Stead Family Department of Pediatrics, University of Iowa Carver College of Medicine, Iowa City, Iowa, USA.
¹⁴ Sarah Cannon Transplant and Cellular Therapy Network, San Antonio, Texas, USA.
¹⁵ Department of Pediatrics, University of Cincinnati College of Medicine, Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.
¹⁶ Department of Pediatrics, University of California San Diego, Rady Children's Hospital, San Diego, California, USA.
¹⁷ Department of Pediatric Hematology/Oncology/Blood and Marrow Transplantation, Medical College of Wisconsin, Milwaukee, Wisconsin, USA.
¹⁸ Department of Pediatrics, Center for Cancer and Blood Disorders, Phoenix Children's Hospital, Phoenix, Arizona, USA.

PMID: 38757488
DOI: 10.1002/pbc.31070

Abstract

Severe aplastic anemia (SAA) is a rare potentially fatal hematologic disorder. Although overall outcomes with treatment are excellent, there are variations in management approach, including differences in treatment between adult and pediatric patients. Certain aspects of treatment are under active investigation in clinical trials. Because of the rarity of the disease, some pediatric hematologists may have relatively limited experience with the complex management of SAA. The following recommendations reflect an up-to-date evidence-based approach to the treatment of children with newly diagnosed SAA.

Keywords: aplastic anemia; bone marrow failure; pediatric hematology/oncology.

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References

REFERENCES

1. Camitta BM, Thomas ED, Nathan DG, et al. Severe aplastic anemia: a prospective study of the effect of early marrow transplantation on acute mortality. Blood. 1976;48(1):63‐70.
1. Shimano KA, Narla A, Rose MJ, et al. Diagnostic work‐up for severe aplastic anemia in children: Consensus of the North American Pediatric Aplastic Anemia Consortium. Am J Hematol. 2021;96(11):1491‐1504.
1. Rovo A, Tichelli A, Dufour C, Saa‐Wp E. Diagnosis of acquired aplastic anemia. Bone Marrow Transplant. 2013;48(2):162‐167.
1. Camitta BM, Storb R, Thomas ED. Aplastic anemia (first of two parts): pathogenesis, diagnosis, treatment, and prognosis. N Engl J Med. 1982;306(11):645‐652.
1. Li FP, Alter BP, Nathan DG. The mortality of acquired aplastic anemia in children. Blood. 1972;40(2):153‐162.

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Treatment of newly diagnosed severe aplastic anemia in children: Evidence-based recommendations

Affiliations

Treatment of newly diagnosed severe aplastic anemia in children: Evidence-based recommendations

Authors

Affiliations

Abstract

References

REFERENCES

Publication types

MeSH terms

LinkOut - more resources

Full Text Sources