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Updated: Apr 15, 2026

Use of Hematopoietic Stem Cell Transplantation to Assess the Origin of Myelodysplastic Syndrome
Published on: October 3, 2018
The role of hematopoietic stem cell transplantation in chronic myeloid leukemia
Alois Gratwohl1, Helen Baldomero, Jakob Passweg
1Hematology, University Hospital, Dittingerstrasse 4, 4053, Basel, Switzerland, alois.gratwohl@unibas.ch.
Insights
Allogeneic hematopoietic stem cell transplantation (HSCT) for chronic myeloid leukemia (CML) needs updated guidelines. Integrating disease and transplant risk offers a balanced approach for optimal patient outcomes.
Area of Science:
- Hematology
- Oncology
- Transplantation Medicine
Background:
- Allogeneic hematopoietic stem cell transplantation (HSCT) is a recommended therapy for chronic myeloid leukemia (CML), typically as second or third-line treatment.
- The advent of tyrosine kinase inhibitors (TKIs) has led to a significant decrease in HSCT for CML in the chronic phase.
- Current recommendations primarily focus on disease risk, such as TKI failure, potentially overlooking other critical factors.
Purpose of the Study:
- To propose an updated, balanced appraisal of allogeneic HSCT for individual CML patients.
- To advocate for the integration of HSCT into treatment algorithms earlier in the disease course.
- To redefine the role of HSCT based on a comprehensive assessment of disease risk, transplant risk, and macroeconomic factors.
Main Methods:
- Review of current HSCT recommendations for chronic myeloid leukemia.
- Analysis of disease risk stratification in CML.
- Evaluation of transplant-related risks and macroeconomic considerations.
Main Results:
- The introduction of TKIs has significantly reduced HSCT utilization in chronic phase CML.
- Existing recommendations may not adequately address the evolving treatment landscape for CML.
- A more individualized approach considering disease and transplant risks is warranted.
Conclusions:
- HSCT should be considered earlier, particularly at the first TKI failure for high-risk CML patients with low transplant risk.
- For CML patients with very advanced disease and high transplant risk, HSCT may be best reserved for research settings.
- Updated guidelines are necessary to optimize HSCT utilization in chronic myeloid leukemia management.
Abstract:
Allogeneic hematopoietic stem cell transplantation (HSCT) is currently recommended as 2nd or 3rd line therapy for patients with chronic myeloid leukemia (CML) in first chronic phase or as salvage for patients with very advanced disease. As a consequence, numbers of HSCT in chronic phase have dropped significantly since the introduction of tyrosine kinase inhibitors (TKI), numbers of transplants in advanced disease to a lesser extent. These current recommendations consider primarily disease risk, defined as failure of TKI therapy; they might need to be adapted. We propose a more balanced appraisal of HSCT for individual patients which should include disease risk, transplant risk, and macroeconomic aspects. HSCT should be integrated into the treatment algorithms from diagnosis and be considered very early at first TKI failure for patients with high disease but low transplant risk. For patients with very advanced disease and high transplant risk in contrast, HSCT might only be recommended in a restricted research setting.
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