Treatment of relapsed/refractory severe aplastic anemia in children: Evidence-based recommendations

Kristin A Shimano¹, Ghadir Sasa², Larisa Broglie³, Nicholas J Gloude⁴, Kasiani Myers⁵, Taizo A Nakano⁶, Anjali Sharathkumar⁷, Jennifer A Rothman⁸, Maria A Pereda⁶, Kathleen Overholt⁹, Anupama Narla¹⁰, Catherine McGuinn¹¹, Bonnie W Lau¹², Amy E Geddis¹³, Yigal Dror¹⁴, Jill L O de Jong¹⁵, Paul Castillo¹⁶, Steven W Allen¹⁷, 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 Sarah Cannon Transplant and Cellular Therapy Network, San Antonio, Texas, USA.
³ Department of Pediatric Hematology/Oncology/Blood and Marrow Transplantation, Medical College of Wisconsin, Milwaukee, Wisconsin, USA.
⁴ Department of Pediatrics, University of California San Diego, Rady Children's Hospital, San Diego, California, USA.
⁵ Department of Pediatrics, Division of Bone Marrow Transplantation and Immune Deficiency, University of Cincinnati College of Medicine, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.
⁶ Department of Pediatrics, Center for Cancer and Blood Disorders, Children's Hospital Colorado, Aurora, Colorado, USA.
⁷ Stead Family Department of Pediatrics, University of Iowa Carver College of Medicine, Iowa City, Iowa, USA.
⁸ Division of Pediatric Hematology/Oncology, Duke University Medical Center, Durham, North Carolina, USA.
⁹ Department of Pediatrics, Pediatric Hematology/Oncology, Riley Hospital for Children at Indiana University, Indianapolis, Indiana, USA.
¹⁰ Department of Pediatrics, Stanford University School of Medicine, Stanford, California, USA.
¹¹ Department of Pediatrics, Division of Pediatric Hematology Oncology, Weill Cornell Medicine, New York, New York, USA.
¹² Department of Pediatrics, Pediatric Hematology-Oncology, Dartmouth-Hitchcock, Lebanon, Pennsylvania, USA.
¹³ Department of Paediatrics, Division of Hematology/Oncology, Cancer and Blood Disorders Center, Seattle Children's Hospital, Seattle, Washington, USA.
¹⁴ Department of Pediatrics, Marrow Failure and Myelodysplasia Program, The Hospital for Sick Children, Toronto, Canada.
¹⁵ Department of Pediatrics, Section of Hematology/Oncology/Stem Cell Transplantation, University of Chicago, Chicago, Illinois, USA.
¹⁶ Department of Pediatrics, Division of Pediatric Hematology Oncology, UF Health Shands Children's Hospital, Gainesville, Florida, USA.
¹⁷ Department of Pediatrics, Pediatric Hematology/Oncology, University of Pittsburgh School of Medicine, UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, USA.
¹⁸ Department of Pediatrics, Center for Cancer and Blood Disorders, Phoenix Children's Hospital, Phoenix, Arizona, USA.

PMID: 38764170
DOI: 10.1002/pbc.31075

Review

Treatment of relapsed/refractory severe aplastic anemia in children: Evidence-based recommendations

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

. 2024 Aug;71(8):e31075.

doi: 10.1002/pbc.31075. Epub 2024 May 19.

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

PMID: 38764170
DOI: 10.1002/pbc.31075

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 relapsed or refractory SAA.

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

PubMed Disclaimer

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. Barone A, Lucarelli A, Onofrillo D, et al. Diagnosis and management of acquired aplastic anemia in childhood. Guidelines from the Marrow Failure Study Group of the Pediatric Haemato‐Oncology Italian Association (AIEOP). Blood Cells Mol Dis. 2015;55(1):40‐47.
1. Samarasinghe S, Veys P, Vora A, Wynn R. Paediatric amendment to adult BSH Guidelines for aplastic anaemia. Br J Haematol. 2018;180(2):201‐205.
1. Killick SB, Bown N, Cavenagh J, et al. Guidelines for the diagnosis and management of adult aplastic anaemia. Br J Haematol. 2016;172(2):187‐207.
1. Pierri F, Dufour C. Management of aplastic anemia after failure of frontline immunosuppression. Expert Rev Hematol. 2019;12(10):809‐819.

Publication types

Actions

MeSH terms

Actions
Actions
Actions
Actions
Actions
Actions

LinkOut - more resources

Full Text Sources
- Ovid Technologies, Inc.
- Wiley
Medical
- MedlinePlus Health Information

Save citation to file

Email citation

Add to Collections

Add to My Bibliography

Your saved search

Create a file for external citation management software

Your RSS Feed

Treatment of relapsed/refractory severe aplastic anemia in children: Evidence-based recommendations

Affiliations

Treatment of relapsed/refractory severe aplastic anemia in children: Evidence-based recommendations

Authors

Affiliations

Abstract

References

REFERENCES

Publication types

MeSH terms

LinkOut - more resources

Full Text Sources

Medical