American Board of Internal Medicine (ABIM) Certification Practice Exam

What is the primary treatment for high-risk myelodysplastic syndrome?

1. Chemotherapy

2. Radiation therapy

3. Hematopoietic stem cell transplantation

4. Targeted therapy

The correct answer is: Hematopoietic stem cell transplantation

Hematopoietic stem cell transplantation (HSCT) is considered the primary treatment for high-risk myelodysplastic syndrome (MDS) due to its potential for curative outcomes. High-risk MDS is characterized by significant cytogenetic abnormalities and poor prognosis, with a higher likelihood of progressing to acute myeloid leukemia (AML). HSCT offers the opportunity to replace the dysfunctional bone marrow with healthy hematopoietic stem cells, potentially reversing the disease process. The rationale behind opting for HSCT in these patients includes the ability to address the underlying issues of ineffective hematopoiesis and to provide a strong enough immune response against any residual malignant cells. Additionally, in high-risk cases, patients often require treatment that can effectively manage both the dysplastic cells and prevent the progression of the disease, which HSCT can accomplish adequately. Conversely, while chemotherapy can induce remission in some patients with MDS, it is generally less effective in high-risk cases and can lead to increased toxicity. Radiation therapy is not a standard treatment for MDS and is more suitable for localized malignancies. Targeted therapy has shown promise in various hematologic malignancies but has limited roles in MDS, especially in high-risk settings, where the primary goal remains achieving