Myelodysplastic syndrome
ICD-10 D46 · ICD-11 2A3Z

Treatment of Higher-Risk Myelodysplastic Syndrome (IPSS-R ≥4.0) in Patients Aged 70 or Younger Without an Available Stem Cell Donor

This protocol addresses a distinct and clinically important scenario: a relatively younger patient with higher-risk MDS who, despite being a potential candidate for intensive approaches, does not currently have an available allogeneic stem cell donor.

Clinical Scenario

Higher-risk myelodysplastic syndrome is defined as an IPSS-R score of 4.0 or above — encompassing high- and very high-risk patients, as well as the remaining intermediate-risk patients in this range. This protocol applies specifically to patients who are 70 years of age or younger, have no unfavourable karyotype, and for whom no allogeneic stem cell donor is currently available.

Treatment Approach (Partial Overview)

In fit patients meeting this profile, treatment may involve an AML-like intensive chemotherapy regimen — considered particularly where marrow blast burden is substantial and ideally used as a bridge toward transplant should a donor become available — or an alternative structured hypomethylating agent schedule, generally pursued over multiple courses. The complete criteria for selecting between these pathways, their sequencing, and the full regimen specifications are available in the protocol below.

The complete regimen — including agent selection, scheduling, and eligibility details — is in the full protocol.
Instant Access to Structured Evidence-Based Regimens

References

DOI: 10.1016/j.annonc.2020.11.002

  1. ‘Higher-risk’ MDS includes patients with IPSS-R 4.0, i.e. high- and very high-risk, and the remaining intermediate-risk IPSS-R patients.
  2. Patients of 70 years and no unfavourable karyotype
  3. Patients of >70 years or younger but without a donor for allo-SCT
  4. It can be envisaged for fit patients (generally <70 years of age) without unfavourable cytogenetics (especially patients with a normal karyotype) and >10% marrow blasts, preferably as a bridge to allo-SCT [I, B].
  5. Suggested regimens with equivalent efficacy are combinations of cytarabine with idarubicin, or fludarabine [IV, B].
  6. As most patients only respond to azacitidine after several courses, at least six courses are recommended as part of the following schedule: azacitidine 75 mg/m2/day subcutaneously for 7 consecutive days every 28 days [II, B].
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