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Updated: Aug 9, 2025
Use of Hematopoietic Stem Cell Transplantation to Assess the Origin of Myelodysplastic Syndrome
Published on: October 3, 2018
Allogeneic stem cell transplantation without preconditioning in a child with therapy-related myelodysplastic
Yi-Ling Tung1, Yi-Lun Wang1, Tsung-Yen Chang2
1Department of Pediatrics, Chang Gung Children's Hospital, Chang Gung University, Taoyuan, Taiwan.
Insights
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) without preconditioning offers a promising treatment for therapy-related myelodysplastic syndrome (t-MDS) in infants, particularly those with MLL-rearranged ALL and recurrent infections.
Area of Science:
- Hematologic Malignancies
- Stem Cell Transplantation
- Pediatric Oncology
Background:
- Infants with mixed-lineage leukemia (MLL)-rearranged leukemia often resist standard therapy and are poor candidates for allogeneic hematopoietic stem cell transplantation (allo-HSCT).
- Therapy-related myelodysplastic syndrome (t-MDS) in these patients carries a poorer prognosis than de novo MDS.
Observation:
- A case of a child with t-MDS that evolved from MLL-rearranged acute lymphoblastic leukemia (ALL) is presented.
- The patient experienced recurrent infections, complicating treatment decisions.
Findings:
- Successful treatment of t-MDS was achieved using HSCT without toxic preconditioning.
- This approach proved effective even when a matched sibling donor was unavailable, utilizing an unrelated donor.
Implications:
- Allogeneic HSCT without preconditioning is a viable and promising therapeutic option for t-MDS, especially in cases of recurrent or persistent infections.
- Omitting preconditioning may preserve immune function, crucial for managing post-transplant complications in pediatric leukemia survivors.
Rationale:
Infants with mixed-lineage leukemia (MLL)-rearranged leukemia are usually refractory to standard induction therapy and are not immediate candidates for allogeneic hematopoietic stem cell transplantation (allo-HSCT). Chromosome 11q23 translocations, resulting in MLL rearrangement, have been well characterized in infant acute lymphoblastic leukemia (ALL). While t(4;11) ALL continues to have carry a bleak prognosis, patients with therapy-related myelodysplastic syndrome (t-MDS) have a shorter median overall survival than those compared with de novo MDS.
Patient Concerns:
We describe a child with t-MDS who evolved from MLL-rearranged ALL and was successfully treated with HSCT without toxic preconditioning.
Diagnoses:
MDS diagnosis was based on morphological characteristics of bone marrow dysplasia in patients with clinical manifestations evidence of hematopoiesis impairments by different combinations of anemia, leukopenia, neutropenia, and thrombocytopenia.
Interventions:
Although the best donor for allo-HSCT is generally considered an human leukocyte antigen-matched sibling, only ~ 30% of patients have a suitable sibling. HSCT from an unrelated donor is a suitable option for patients with t-MDS who do not have matched sibling donors.
Outcomes:
Allo-HSCT without recipient preconditioning could be a promising treatment option for t-MDS, especially for patients with recurrent or persistent infections.
Lessons:
Cytogenetics, prognosis, and treatment of t-MDS are briefly discussed. Preconditioning before allo-HSCT seriously damages immune function. This work reviews our experience with a patient with t-MDS following ALL complicated by recurrent infections, and highlights our choice to omit preconditioning from allo-HSCT.
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