The MDS Beacon™

Researchers from the Instituto Di Ricovero e Cura a Carattere Scientifico in Italy presented a systematic review of 10 thalidomide treatment studies at the 14th Congress of the European Hematology Association (EHA). They found that thalidomide (Thalomid) doses provide effective and tolerable therapy for certain myelodysplastic syndrome (MDS) patients, particularly patients with recent diagnoses and low International Prognostic Score System (IPSS) scores.

The researchers reviewed 10 Phase 1-2 studies of thalidomide treatment, examining the efficacy of the drug in the treatment of 527 MDS patients. The majority of studies recorded higher red blood cell counts and transfusion-independence within two to three months of treatment initiation. The duration of thalidomide responses varied from three months to more than six years. Two studies suggested that thalidomide may offer a possible survival advantage to MDS patients, improving their ability to consistently produce healthy blood cells.

The researchers identified responses associated with all thalidomide dose sizes, ranging between 50 mg and 1000 mg per day. No correlation was found between the response characteristics and concentrations of red blood cell hormones. The patients’ previous, alternative treatments did not interfere with the effectiveness of the thalidomide treatments. However, patients with lower IPSS scores (the system by which doctors classify patients according to symptoms and prognoses) and recent diagnoses (less than a year) most frequently displayed improved health after the thalidomide treatments.

Due to the application of thalidomide treatments, the researchers recognized occasional changes in marrow cell structure, resulting in new marrow morphology and improved red blood cell production.

Data documented side effects, including sedation, constipation, skin rash, and peripheral nerve damage. These side effects induced 15 percent to 67 percent of patients to quit the studies. Overall, 44 percent of patients dropped from the thalidomide treatments, particularly elderly patients receiving more than 200 mg per day.

Blood clots were a rare side effect, but the frequency of clot formation increased with higher doses of thalidomide. Patients in the studies did not utilize anti-clotting medications, and the studies did not examine the use of anti-clotting medication in preventing thalidomide-related clots.

Based on the results of the 10 studies, the use of thalidomide treatments was recommended for low-risk patients with transfusion-dependent anemia, characterized by an inability to maintain normal red blood cell counts. The researchers supported the use of thalidomide treatments for patients, who are not candidates for other approaches, including epoetins, Revlimid (lenalidomide), high dose chemotherapy, and hypomethylating agents like Vidaza (azacitidine) and Dacogen (decitabine).

In the absence of serious side effects, early doses of thalidomide, increasing from 100 mg per day to no more than 200 mg per day over 12 weeks, may produce favorable responses in young MDS patients. Smaller doses may also produce favorable responses in elderly MDS patients.

For more information, see abstract 0264 from the “Myelodysplastic Syndromes I” session of the 14th Congress of the EHA.