Thursday, January 19, 2023

Myelodysplastic syndrome

 Myelodysplastic syndrome

Patient evaluation template:

ECOG:

IPSS-R:IPSS-R

IPSS-M: Molecular IPSS

Prognosis based on IPSS category

Transfusion requirement: e.g 2 units PRBC every 3 weeks etc

Transplant candidate: Yes or no

Goals of treatment: Curative versus palliative

Supportive care plan: GCSF, transfusion, antimicrobials.


Treatment options:

1. Standard of care: Azacitidine is the only hypomethylating agent shown to improve survival.

AZA-001 study: Azacitidine demonstrated increased OS compared with conventional care regimen, 24.4 months vs 15 months, respectively (P = .0001). In addition, there was a 74% OS improvement with azacitidine (hazard ratio [HR], 0.58.

However with repeat studies the OS has been in the 15-17 month range and the OS of the AZA-001 study has not been duplicated.

2. Side effects: Initial worsening of cytopenias in the first 1–4 months of treatment, lack of adequate predictors of response, and prolonged therapy

3. Other agents, including oral combinations: IV decitabine ( no OS benefit), oral decitabine with cedazuridine, similar CR to azacitidine but no OS benefit so far. However, QOL factors are better. Increases compliance and reduces vascular access.


Other options not yet SOC:

1. Venetoclax 400 mg 14 days out of a 28 day cycle + azacitidine 75mg/m2 day 1-5. High CR but 1% mortality

2. IDH 1 and IDH2 mutated MDS relapsed setting with IDH inhibitors with high CR rates > 40%.

3. Sabatilomab targets TIM-3, Magrolimab anti CD 47


AZA: dose adjustment from cancer care ontario

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