Related Experiment Video
Updated: Aug 27, 2026
Comprehensive Protocol to Sample and Process Bone Marrow for Measuring Measurable Residual Disease and Leukemic Stem Cells in Acute Myeloid Leukemia
Published on: March 5, 2018
[Seriousness of severe acquired aplastic anemia in children. Apropos of 32 cases]
Insights
Severe aplastic anemia in children has a poor prognosis, with only 18.7% survival. Bone marrow transplantation offered limited success, highlighting the need for improved pediatric severe aplastic anemia treatments.
Area of Science:
- Pediatric Hematology
- Hematopoietic Stem Cell Transplantation
- Aplastic Anemia Research
Context:
- Retrospective study of 32 pediatric severe aplastic anemia cases (1967-1985) in the Rhône-Alpes region.
- Identified potential toxic or hepatitis-related causes in a subset of cases.
- Defined diagnostic criteria including hemoglobin, reticulocyte, neutrophil, and platelet counts.
Purpose:
- To analyze the outcomes and prognosis of severe aplastic anemia in children.
- To evaluate the effectiveness of different treatment modalities, including bone marrow transplantation.
- To identify factors influencing survival rates in pediatric severe aplastic anemia.
Summary:
- Severe aplastic anemia in children under 16 demonstrated a poor prognosis, with an overall survival rate of 18.7%.
- Non-transplant recipients had very low survival rates (2/19), with most deaths occurring early.
- Bone marrow transplantation from sibling donors yielded a 30.8% survival rate (4/13), with only 2 long-term survivors.
Impact:
- This study underscores the critical need for advancements in treating pediatric severe aplastic anemia.
- Highlights the challenges and limited success of historical treatment approaches.
- Provides valuable data for future research and clinical trial design in pediatric hematology.
Abstract:
Thirty-two cases of severe aplastic anemia in children less than 16 years of age, observed between 1967 and 1985 in the Rhône-Alpes area were studied retrospectively. A toxic cause was suspected in 7 cases, an hepatitis in 5 cases. Patients presented with 2 of the 3 following cytopenias during the 3 weeks following diagnosis: hemoglobin level below 100 g/l, reticulocyte count lower than 20 G/l, neutrophil polymorphonuclear leukocyte count below 0.5 G/l, platelet count lower than 20 G/l. Whatever the treatment, prognosis was poor: 6 surviving patients (18.7%). Nineteen patients did not undergo bone marrow transplantation: only 2 are surviving after 4 years, one after low dose corticosteroid therapy, the other after antilymphocyte globulins and androgen therapy; 10 of 17 deaths occurred in the first 4 months. Thirteen bone marrow transplantations from sibling donors, of which 10 HLA A, B DR identical and 3 haploidentical allowed for 8 prolonged takes but only 4 survivors of whom 2 lasted for more than 2 years.
