The MDS Beacon™

Chinese researchers have found in a pilot study that cyclosporine and thalidomide combination treatment led to improved blood counts and was well-tolerated in myelodysplastic syndromes patients.

“This is the first report that cyclosporine combined with thalidomide could improve the response in low or intermediate-risk myelodysplastic syndromes patients who are not candidates for intensive approaches,” wrote Dr. Zhijian Xiao, one of the study researchers, in an email to The MDS Beacon.

Cyclosporine (Sandimmune) is a drug that suppresses the immune system. It is widely used after patients receive organs from a donor to prevent rejection of the organ. Cyclosporine has been shown to help with blood count recovery in aplastic anemia and myelodysplastic syndromes (MDS) patients.

The researchers hypothesized that combining cyclosporine with thalidomide (Thalomid), an agent that is highly effective in certain blood cancers, could further improve the response in MDS patients .

For their study, the researchers recruited 37 MDS patients who had not received any treatment except for supportive care with transfusions during two months before entering the study.

The patients received 3 to 5 mg/kg oral cyclosporine and thalidomide daily. The initial thalidomide dosage was 50 mg and was increased to 200 mg over several weeks.

The patients were treated for a minimum of 12 weeks. They had the option to continue if the treatment was effective without excessive side effects.

The patients showed an initial response after a median of 7 weeks. The average time of treatment was 24 weeks.  The response continued for a median of 88 weeks.

The researchers found that half of the patients (51.4 percent) with low red blood cell counts showed improved red blood cell counts. Of the patients who required red blood cell transfusions, 48.3 percent achieved transfusion independence.

Of the patients who had low platelet counts, 31 percent had improved platelet counts. One-third (33.3 percent) of platelet-dependent patients achieved transfusion independence.

White blood cell counts improved in 21.2% of the patients who had low neutrophils, a type of white blood cell.

The patients’ median overall survival was 52 months. Intermediate-1 risk patients had a significantly higher rate of overall survival than intermediate-2 risk patients.

The researchers did not find any significant difference in the effectiveness between a lower and higher dose of thalidomide. They therefore suggested using a lower dose as a starting dose for MDS patients.

Side effects were generally mild to moderate and included increased liver enzymes (21.6 percent of patients), fatigue (21.6 percent), constipation (18.9 percent), swelling (18.9 percent), dizziness (13.5 percent), kidney impairment (8.1 percent), numbness (8.1 percent), and skin rash (5.4 percent). None of the patients developed blood clots in the deep veins, a common side effect of thalidomide therapy.

“A larger scale national study including multiple centers is planned to confirm these results,” said Dr. Xiao.

For more information, please see the study in the journal Leukemia Research (abstract).