Experts at The Johns Hopkins Kimmel Cancer are nationally and internationally recognized as leaders in the research and treatment of myelodysplastic syndromes and other blood disorders. Our experienced and dedicated team provides our patients with innovative treatments and the best quality of care possible.
A Multi-center Phase I Trial of Ipilimumab in Patients with Myelodysplastic Syndromes following Hypomethylating Agent Failure. Clin Cancer Res. 2018 Aug 1;24(15):3519-3527. doi: 10.1158/1078-0432.CCR-17-3763. Epub 2018 May 1. PMID 29716921.
In a phase I clinical trial, Johns Hopkins researchers B. Douglas Smith, Amy DeZern and others tested the safety and efficacy of an immunotherapy drug called ipilimumab in patients with MDS whose disease did not respond to standard therapy called hypomethylating agents. These patients have no other treatment options and poor survival rates. In the study of 29 patients, the researchers found that ipilimumab is safe but had limited effectiveness in stabilizing the disease and prolonging survival when given on its own.
Genomic Biomarkers to Predict Resistance to Hypomethylating Agents in Patients With Myelodysplastic Syndromes Using Artificial Intelligence. JCO Precis Oncol. 2019;3:PO.19.00119. doi: 10.1200/po.19.00119. Epub 2019 Sep 20. PMID 31663066.
Johns Hopkins clinician Amy DeZern and her colleagues used artificial intelligence to look at common genetic mutations in patients with MDS to determine which mutations were associated with resistance to treatment with hypomethylating agents, which are the standard care for MDS. After screening 433 patients for 29 common mutations, the researchers show that these genetic markers can identify with high accuracy about one-third of patients with MDS who will not respond to hypomethylating agents.
Luspatercept in Patients with Lower-Risk Myelodysplastic Syndromes. N Engl J Med. 2020 Jan 9;382(2):140-151. doi: 10.1056/NEJMoa1908892. PMID 31914241.
In this paper, Johns Hopkins clinician Amy DeZern and her colleagues test the drug luspatercept in patients with lower-risk MDS who have anemia (a lack of healthy red blood cells). These patients either did not respond to other drugs to increase red blood cell production or had a poor reaction to the drugs. In a phase 3 clinical trial of 229 patients, the researchers found that luspatercept reduced the risk of severe anemia in patients with lower-risk MDS who had been receiving regular blood transfusions.