1. SUB-TYPE OF MDS: All patients with MDS (but see basic exclusion criteria below)
2. SEVERITY OF MDS: Revised International Prognostic Scoring System (IPSS-R) score > 3 (intermediate, high or very high).
3. NAME OF DRUG: Venetoclax. Venetoclax is an inhibitor of BCL-2, thus promoting programmed death of tumour cells.
4. Aims and benefits: This is a randomised double-blind, Phase 3 study aimed at determining the safety and effectiveness of Venetoclax when used in combination with Azacitidine in newly diagnosed patients with Higher risk Myelodysplastic syndrome.
5. Primary outcome: To determine complete response and overall survival.
6. Secondary outcome: To determine the rate of achieving transfusion independence.

• Basic inclusion criteria:
  • Patients with IPSS-R score of > 3
  • ECOG performance status of ≤ 2
• Basic exclusion criteria:
  • Prior therapy with hypomethylating agent or allogeneic stem cell transplant.
  • Therapy-related MDS, MDS evolving from previous MPN, MDS/MPN including CMML, atypical CML and juvenile myelomonocytic leukaemia.
• Trial sites/locations and name of physician in charge of trial (none are currently recruiting):
  • University Hospitals Bristol NHS Foundation Trust, Bristol
  • Oxford University Hospitals NHS Foundation Trust, Oxford
  • Cardiff & Vale University Health Board, Cardiff
  • NHS Grampian, Aberdeen
  • University Hospitals Birmingham NHS Foundation Trust, Birmingham
  • University College London Hospitals NHS Foundation Trust, London
  • The Royal Marsden NHS Foundation Trust, London
  • University Hospital Plymouth NHS Trust, Plymouth
  • University Hospitals Dorset NHS Foundation Trust, Poole

If you think this information is relevant to you, please discuss it with your own physician.