Thalidomide and its derivatives have been used in the treatment of myelodysplastic syndrome (MDS) because of their anti-angiogenic and immunomodulatory effects. In recent years, some studies have found that thalidomide and its derivatives not only showed significant efficacy in lower-risk MDS patients with del (5q), but also showed advantages in non-del (5q) MDS patients. In addition, the discovery of its molecular targets and new substrates makes it possible to develop a new generation of immunomodulatory drugs (IMiDs) and to design IMiDs-based proteolysis-targeting chimeras. In this review, the new progress in mechanism and clinical application of thalidomide and its derivatives were summarized briefly, so as to provide a more scientific, reasonable and effective scheme to the treatment of MDS.
Humans ; Immunomodulating Agents ; Myelodysplastic Syndromes/drug therapy* ; Thalidomide/therapeutic use*

Henoch-Sch nlein purpura is an acute, self-limited vasculitis syndrome which shows characteristic skin, joint, renal and gastrointestinal manifestations. It is common in childhood and may also occur in adults with fatal complications such as nephritis and gastrointestinal bleeding. We experienced a case of a 20-year-old woman who presented with palpable purpura and severe arthritis. The histopathologic examination of the skin revealed leukocytoclastic vasculitis with perivascular deposition of IgA and she was diagnosed with Henoch-Sch nlein purpura. Despite treatment with prednisolone for one month, she had more aggravated purpura and fatal gastrointestinal bleeding. The symptoms were improved shortly by cyclophosphamide pulse therapy with plasmapheresis but symptoms were aggravated and symmetric mononeuropathy of the ulnar nerve developed. She was treated with 400 mg/day of thalidomide and symptoms were improved. We herein report a case of Henoch-Sch nlein purpura successfully treated with thalidomide which was refractory to prednisolone, immunosuppressive drugs and plasmapheresis.
Adult ; Dermatologic Agents/*therapeutic use ; Female ; Human ; Purpura, Schoenlein-Henoch/*drug therapy ; Thalidomide/*therapeutic use

Behcet's disease is characterized with multifactorial etiopathogenesis and multiclinical pictures. The treatment of patients with Behcet's disease is based on the severity of illness, and the most appropriate management of Behcet's disease requires a multidisciplinary approach. Although various therapeutic modalities have been employed for Behcet's disease, treatment is far from satisfactory. Treatment of Behcet's disease includes local, systemic, or surgical therapies. Limited success has been found with colchicine, azathioprine, indomethacin, cyclophosphamide, chlorambucil, levamisole, transfer factor, fibrinolytic therapy, and systemic corticosteroid. New therapeutic approaches have been introduced for Behcet's disease using cyclosporine, thalidomide, interferon, acyclovir, high-dose corticosteroids or cyclophosphamide pulse therapy, and FK 506. We suggest that therapeutic agents should be selected after thorough evaluation of the immune state of each patient by using various tests and by determining any aggravating or provoking factors involved. In general, a combination-agent regimen is more effective than a single-agent regimen. Early diagnosis and proper treatment can inhibit or at lease slow the progress of the disease remarkably.
Adrenal Cortex Hormones/therapeutic use ; Behcet's Syndrome/therapy* ; Cyclophosphamide/therapeutic use ; Human ; Immunosuppressive Agents/therapeutic use ; Tetracycline/therapeutic use ; Thalidomide/therapeutic use ; Zinc Sulfate/therapeutic use

China ; Humans ; Lenalidomide/therapeutic use* ; Multiple Myeloma/drug therapy* ; Thalidomide ; Therapeutic Equivalency

BACKGROUNDThalidomide is an immunomodulatory and anti-angiogenic drug that has shown promise in patients with myeloma. Trials comparing efficacy of standard melphalan and prednisone (MP) therapy with MP plus thalidomide (MPT) in transplant-ineligible or elderly patients with multiple myeloma (MM) have provided conflicting evidence. This meta-analysis aimed to determine the efficacy and toxicity of thalidomide in previously untreated elderly patients with myeloma.

METHODSMedline, the Cochrane Controlled Trials register, conference proceedings of the American Society of Hematology (1995-2014), the American Society of Clinical Oncology (1995-2014), and CBM, VIP, and CNKI databases were searched for randomized control trials with the use of the medical subject headings "MM " and "thalidomide ". Trials were assessed by two reviewers for eligibility. Meta-analysis was conducted using a fixed effects model. Sensitivity analysis was performed to test the robustness of the findings.

RESULTSOverall, seven trials were identified, covering a total of 1821 subjects. The summary hazard ratio (thalidomide vs. control) was 0.82 (95% confidence interval [CI]: 0.72-0.94) for overall survival (OS), and 0.65 (95% CI: 0.58-0.73) for progression-free survival, in favor of thalidomide treated group. The risk ratio of complete response with induction thalidomide was 3.48 (95% CI: 2.24-5.41). A higher rate of III/IV adverse events were observed in MPT arm compared with the MP arm. However, analysis of sub-groups administering anticoagulation as venous thromboembolism prophylaxis suggested no difference in relative risk of thrombotic events between two arms (RR = 1.47, 95% CI: 0.43-5.07, P = 0.54). Further analysis of trials on the treatment effects of MPT versus MP on adverse events-related mortality showed no statistical difference between two arms (RR = 1.24, 95% CI: [0.95-1.63], P = 0.120).

CONCLUSIONThalidomide appears to improve the OS of elderly and/or transplant-ineligible patients with MM when it is added to standard MP therapy.

Disease-Free Survival ; Humans ; Immunosuppressive Agents ; therapeutic use ; Melphalan ; therapeutic use ; Multiple Myeloma ; drug therapy ; mortality ; Prednisone ; therapeutic use ; Thalidomide ; therapeutic use

Antineoplastic Combined Chemotherapy Protocols ; therapeutic use ; Humans ; Interferons ; Lymphoma, Mantle-Cell ; drug therapy ; Recurrence ; Thalidomide

Humans ; Mouth Diseases ; drug therapy ; Mouth Mucosa ; pathology ; Thalidomide ; therapeutic use

Myelofibrosis, a clonal stem-cell disorder which is difficult to cure, The treatment for most of patients is conservative treatment, but the treatment effect is not ideal. Lenalidomide as a novel immunomodulator in the treatment of blood diseases showed good results in recent years, Its studies have shown that lenalidomide in myelofibrosis also showed a certain effect. As companed with single drug lenalidomide, the thalidomide has a higher response rate, clinical symptoms improved significantly. Ruxolitinib, JAK2 inhibitor, combined with lenalidomide not only can improve the quality of life of patients, but also extend the survival of patients. In addition, lenalidomide combined with prednisone for the treatment of bone marrow fibrosis is more effective and more safe, lenalidomide can significantly improve the clinical symptoms of patients, especially anemia, and prednisone can reduce the hematologic toxicity of lenalidomids. The purpose of this review is to evaluate the efficacy and safety of lenalidomide in the treatment of myelofibrosis, and focuses on the newest clinical research and application progress of lenalidomide for myelofibrosis.
Humans ; Lenalidomide ; therapeutic use ; Prednisone ; Primary Myelofibrosis ; drug therapy ; Quality of Life ; Thalidomide

OBJECTIVETo evaluate the efficacy of thalidomide in the treatment of juvenile idiopathic arthritis (JIA).

METHODSTwelve children with JIA who did not respond to conventional treatment were administered with thalidomide (2 mg/kg daily). The symptoms, signs, and laboratory test results were compared before and after treatment. The thalidomide-related side effects were observed.

RESULTSThe average dosage of prednisone was reduced from 1.92 ± 0.16 mg/kg•d to 0.49 ± 0.42 mg/kg•d in the 12 patients 6 months after thalidomide treatment (P<0.01). Four patients did not need prednisone treatment any more. White blood cell count, erythrocyte sedimentation rate (ESR), C reactive protein (CRP) and serum ferritin (SF) significantly decreased after treatment in all of 12 patients (P<0.01). Hemoglobin level increased to normal in 8 patients after treatment (P<0.01). The number of affected joints decreased from 5 before treatment to zero to 2 after treatment in patients with polyarticular JIA (P<0.01). Signs of hip involvement and Schober's sign turned negative in enthesitis-related cases. No thalidomide-related side effects were observed.

CONCLUSIONSThalidomide is effective in the treatment of JIA in children who do not respond to conventional treatment.

Adolescent ; Arthritis, Juvenile ; blood ; drug therapy ; Child ; Female ; Humans ; Male ; Prednisone ; therapeutic use ; Retrospective Studies ; Thalidomide ; therapeutic use

OBJECTIVETo explore the efficiency and side-effects of the combination of cyclosporine A (CsA) and thalidomide in patients with myelodysplastic syndromes (MDS).

METHODSA total of thirty-seven patients with MDS-RCMD or-RAEB-I were treated with CsA in combination with thalidomide. The initial CsA dose of 3 mg×kg(-1)×d(-1) was administered, all patients had their CsA blood concentration concurrently monitored until it reached and maintained between 100 and 200 µg/L. The initial dose of thalidomide was 50 mg/d, with increasing dose of 50 mg every week until the maximum of 200 mg/d. The hematological response was assessed according to the modified criteria of the International Working Group, and adverse events were graded with the Common Toxicity Criteria (v3.0) of the National Cancer Institute. The response duration and overall survival of the patients were also observed.

RESULTS19/37 cases (51.4%) achieved hematologic improvement (HI)-erythroid response (HI-E), 9/29 cases (31.0%) HI-platelet response (HI-P) and 7/33 cases (21.2%) HI-neutrophil response (HI-N). 15 of 32 transfusion-dependent patients (46.9%) achieved transfusion independence. The median response duration of HI-E, HI-P and HI-N were 88 (4 - 88) weeks, 78 (8 - 84(+)) weeks and 78 (10 - 84(+)) weeks respectively. The median overall survival was 52 months on a 29 (4 - 103) months median follow-up. Some patients developed grades I-II hepatic or nephritic impairment, constipation, lethargy, dizziness, edema, rashes or numbness, and all were tolerable and reversible. No grade III or severer adverse events were observed.

CONCLUSIONCsA in combination with thalidomide appears to be effective mainly in inducing HI-E and relatively well-tolerated for the treatment of patients with MDS.

Anemia, Refractory, with Excess of Blasts ; drug therapy ; Cyclosporine ; therapeutic use ; Humans ; Myelodysplastic Syndromes ; drug therapy ; Thalidomide ; therapeutic use ; Treatment Outcome