The Efficacy of Thalidomide for the Symptomatic Large Granular Lymphocytic Leukemia - a Prospective Phase Ⅱ Multicenter Clinical Trial From China
There is currently no standard first-line treatment for LGLL. The investigators used the TPM regimen (thalidomide + prednison + methotrexate ) to treat LGLL since 2020, enrolling a total of 54 patients and achieving an overall response rate (ORR) of 88.9% and a complete response (CR) rate of 75.9%. To further explore this hypothesis, the investigators designed this study to observe the efficacy of thalidomide monotherapy in patients with symptomatic LGLL. The investigators speculate that thalidomide plays a major role in the significant improvement of the TPM regimen compared to the MTX regimen. Patients with LGLL are treated with thalidomide at 50 to 100 mg. If the desired response is not achieved at specific time points, methotrexate is added. Thalidomide monotherapy is administered for up to 3 courses, and the TM regimen can also be used for up to 3 courses. The overall response rate with thalidomide monotherapy serves as the primary study endpoint.
• The patient fully understands the study, voluntarily participates, and signs the informed consent form (ICF);
• The patient must meet the diagnostic criteria for LGLL;
• The patient can be of any gender, aged 18 years or older;
• The patient is either untreated or not properly treated previously, or has poor efficacy (not reaching PR) or relapsed after being treated with regimens not based on methotrexate/thalidomide;
• The patient has indications for LGLL treatment, meeting at least one of the following criteria:
‣ ANC \< 0.5 × 10\^9/L, or neutropenia with recurrent infections
⁃ HGB \< 100 g/L or requiring red blood cell transfusions for maintenance
⁃ PLT \< 50 × 10\^9/L
⁃ Concurrent autoimmune disease requiring treatment
⁃ Symptomatic splenomegaly
⁃ Severe B symptoms (unexplained fever, temperature over 38°C; night sweats; weight loss of 10% or more within six months)
⁃ Pulmonary hypertension;
• ECOG score of 0-2;
• The patient's expected survival period is 6 months or more.