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

被引：4

作者：

Shimano, Kristin A. ^{[1
,19
]}

Rothman, Jennifer A. ^{[2
]}

Allen, Steven W. ^{[3
]}

Castillo, Paul ^{[4
]}

de Jong, Jill L. O. ^{[5
]}

Dror, Yigal ^{[6
]}

Geddis, Amy E. ^{[7
]}

Lau, Bonnie W. ^{[8
]}

McGuinn, Catherine ^{[9
]}

Narla, Anupama ^{[10
]}

Overholt, Kathleen ^{[11
]}

Pereda, Maria A. ^{[12
]}

Sharathkumar, Anjali ^{[13
]}

Sasa, Ghadir ^{[14
]}

Nakano, Taizo A. ^{[12
]}

Myers, Kasiani ^{[15
]}

Gloude, Nicholas J. ^{[16
]}

Broglie, Larisa ^{[17
]}

Boklan, Jessica ^{[18
]}

机构：

[1] Univ Calif San Francisco, Benioff Childrens Hosp, Dept Pediat, Div Allergy Immunol & Bone Marrow Transplant, San Francisco, CA USA

[2] Duke Univ, Med Ctr, Dept Pediat, Div Pediat Hematol Oncol, Durham, NC USA

[3] Univ Pittsburgh, UPMC Childrens Hosp Pittsburgh, Sch Med, Dept Pediat,Pediat Hematol Oncol, Pittsburgh, PA USA

[4] UF Hlth Shands Childrens Hosp, Dept Pediat, Div Pediat Hematol Oncol, Gainesville, FL USA

[5] Univ Chicago, Dept Pediat, Sect Hematol Oncol Stem Cell Transplantat, Chicago, IL USA

[6] Hosp Sick Children, Dept Pediat, Marrow Failure & Myelodysplasia Program, Div Hematol Oncol, Toronto, ON, Canada

[7] Seattle Childrens Hosp, Canc & Blood Disorders Ctr, Dept Pediat, Seattle, WA USA

[8] Dartmouth Hitchcock, Pediat Hematol Oncol, Lebanon, NH USA

[9] Dept Pediat, Div Pediat Hematol Oncol, Weill Cornell Med, New York, NY USA

[10] Stanford Univ, Sch Med, Dept Pediat, Stanford, CA USA

[11] Indiana Univ, Riley Hosp Children, Dept Pediat, Pediat Hematol Oncol, Indianapolis, IN USA

[12] Childrens Hosp Colorado, Ctr Canc & Blood Disorders, Dept Pediat, Aurora, CO USA

[13] Univ Iowa, Carver Coll Med, Stead Family Dept Pediat, Iowa City, IA USA

[14] Sarah Cannon Transplant & Cellular Therapy Network, San Antonio, TX USA

[15] Univ Cincinnati, Cincinnati Childrens Hosp Med Ctr, Dept Pediat, Div Bone Marrow Transplantat & Immune Deficiency,C, Cincinnati, OH USA

[16] Univ Calif San Diego, Rady Childrens Hosp, Dept Pediat, San Diego, CA USA

[17] Med Coll Wisconsin, Dept Pediat Hematol Oncol Blood & Marrow Transplan, Milwaukee, WI USA

[18] Phoenix Childrens Hosp, Ctr Canc & Blood Disorders, Dept Pediat, Phoenix, AZ USA

[19] 550 16th St Box 0434, San Francisco, CA 94143 USA

来源：

PEDIATRIC BLOOD & CANCER | 2024年 / 71卷 / 08期

关键词：

aplastic anemia; bone marrow failure; pediatric hematology/oncology; ANTI-THYMOCYTE GLOBULIN; RABBIT ANTITHYMOCYTE-GLOBULIN; PAROXYSMAL-NOCTURNAL HEMOGLOBINURIA; COMPLEMENT INHIBITOR ECULIZUMAB; BONE-MARROW-TRANSPLANTATION; SIBLING DONOR TRANSPLANTS; STEM-CELL TRANSPLANTATION; VERSUS-HOST-DISEASE; IMMUNOSUPPRESSIVE THERAPY; ANTILYMPHOCYTE GLOBULIN;

D O I：

10.1002/pbc.31070

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

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.

引用

页数：13

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