Vidaza followed by stem cell transplant for children with MDS

  • Myelodysplastic syndrome (MDS) is a condition where the bone marrow stops functioning properly.1,2
  • Adults with MDS may be treated with medications such as Vidaza (azacitidine), but there are no approved treatments for children with MDS.1,2
  • In a small patient-series, children treated with Vidaza before stem cell transplant showed improvements with potential signs of prolonged survival.2

Pop quiz: Name me an organ. Kidney, heart, and…bone marrow?

Right you are! Bone marrow is an organ and its job is to make blood. Can you imagine a broken bone marrow that doesn’t make red blood cells, which we need to carry oxygen; or white blood cells, which we need to fight off infections; or platelets, which we need to stop ourselves from bleeding?

MDS is precisely one condition where the bone marrow stops making blood cells properly.2

MDS is mostly found in people who are more than 70 years old. But sometimes, children can get MDS as well.1,2


About 1 in 33 children with cancer has MDS.2


MDS is a difficult condition to treat. For adults, one strategy is to treat with Vidaza to suppress the MDS and prepare for stem cell transplantation to replace the bone marrow.1,2

Seeing as there are no approved treatments for children with MDS, would this strategy work for them as well?

It might, suggests Dr. Waespe and colleagues in a study published in the journal Haematologica.2

The patient-series looked at the records of 22 children who were treated with or without Vidaza before receiving stem cell transplantation at the Hospital for Sick Children in Toronto, Canada.2

For the 8 children who were treated with Vidaza followed by stem cell transplant, their symptoms of MDS improved by 3-fold vs. children who did not receive Vidaza.2

As for survival, all the children treated with Vidaza before receiving stem cell transplantation survived beyond 4 years without disease progression or relapse, vs. 45.4% of children not treated with Vidaza before transplantation.2

But overall survival was not significantly different between children who were treated with or without Vidaza, possibly due to the small study size.2

Also of note, benefit with Vidaza was not without its risks. Major side effects reported in the study were moderate-to-severe nausea and vomiting, and severe appendicitis.2

Bottom line

  • Vidaza followed by stem cell transplant may be a viable treatment for some children with MDS.
  • For physicians attempting this treatment approach, note the moderate-to-severe (grade 3-4) side effects including nausea/vomiting and infections.
  • Larger studies are needed to confirm the survival benefit of this treatment strategy.

Source study: Waespe N, et al. Haematologica 2016. Published ahead of print: August 18, 2016. A paid subscription may be required.

References: 1. National Comprehensive Cancer Network. NCCN guidelines: Myelodysplastic syndromes version 1.2017. Available at: https://www.nccn.org. Accessed on October 10, 2016. 2. Waespe N, et al. Haematologica 2016. Published ahead of print: August 18, 2016.

Photo adapted from original by abbamouse, used under license.

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