Thalidomide acts on the microenvironment of myelodysplastic syndromes (MDS) by influencing cytokine networks, and growing evidence supports thalidomide’s usefulness in the management of haematological malignancies, such as MDS. The European Collaboration Group on Myelofibrosis with Myeloid Metaplasia reviewed patients who received at least four weeks’ thalidomide treatment, in doses ranging from 50 mg/day to 400 mg/day. The results showed that 30% of patients had increases in haemoglobin, and, of these, almost 40% became transfusion independent. Platelets were increased in a significant proportion of patients, and approximately 40% of patients had a reduction in their spleen size. Data on thalidomide and acute myeloblastic leukaemia (AML) are conflicting: a recently published study indicated that thalidomide does not have a role in the management of acute myeloblastic leukaemia (AML), while other studies suggest some patients may respond because of thalidomide’s ability to activate natural killer cells and cytotoxic T-lymphocytes. Partial responses to thalidomide treatment have been recorded in patients with lymphoma. In a phase II study to assess the activity of thalidomide in patients with Waldenström’s macroglobulinaemia, a partial response was seen in 25% of patients who received a starting dose of 200 mg, which was escalated in 200 mg increments every 14 days as tolerated to a maximum of 600 mg. Although further study is required, thalidomide shows promise in the treatment of a number of haematological malignancies, many of which currently have limited treatment options and poor prognosis.

Raza A, Meyer P, Dutt D, et al: Thalidomide produces transfusion independence in long-standing refractory anemias of patients with myelodysplastic syndromes. Blood 2001;98:958–965.
Zorat F, Shetty V, Dutt D, et al: The clinical and biological effects of thalidomide in patients with myelodysplastic syndromes. Br J Haematol 2001;4:881–894.
Mufti G, List A, Gore S, Ho A: Myelodysplastic syndrome. Hematology (Am Soc Hematol Educ Program) 2003;176–199.
Saunthararajah Y, Nakamura R, Nam J, et al: HLA-DR15 (DR2) is overrepresented in myelodysplastic syndrome and aplastic anemia and predicts a response to immunosuppression in myelodysplastic syndrome. Blood 2002;100:1570–1574.
Strupp C, Germing U, Scherer A, et al: Thalidomide for the treatment of idiopathic myelofibrosis. Eur J Haematol 2004;72:52–57.
Piccaluga P, Visani G, Pileri S, et al: Clinical efficacy and antiangiogenic activity of thalidomide in myelofibrosis with myeloid mateplasia. A pilot study. Leukemia 2002;16:1609–1614.
Mesa R, Steensma D, Pardanani A, et al: A phase 2 trial of combination low-dose thalidomide and prednisone for the treatment of myelofibrosis with myeloid metaplasia. Blood 2003;101:2534–2541.
Barosi G, Elliot M, Canepa L, et al: Thalidomide in myelofibrosis with myeloid metaplasia: a pooled analysis of individual patient data from five studies. Leuk Lymphoma 2002;43:2301–2307.
Spivak J, Barosi G, Tognoni G, et al: Chronic myeloproliferative disorders. Hematology (Am Soc Hematol Educ Program) 2003; 200–224.
Merup M, Kutti J, Birgergard G, et al: Negligible clinical effects of thalidomide in patients with myelofibrosis with myeloid metaplasia. Med Oncol 2002;19:79–86.
Thomas D, Estey E, Giles F, et al: Single agent thalidomide in patients with relapsed or refractory acute myeloid leukaemia. Br J Haematol 2003;123:436–441.
Steins M, Bieker R, Padro T, et al: Thalidomide for the treatment of acute myeloid leukemia. Leuk Lymphoma 2003;44:1489–1493.
Lowdell M, Craston R, Samuel D, et al: Evidence that continued remission in patients treated for acute leukaemia is dependent upon autologous natural kille cells. Br J Haematol 2002;117:821–827.
Davies F, Raje N, Hideshima T, et al: Thalidomide and immunomodulatory derivative augment natural killer cell cytotoxicity in multiple myeloma. Blood 2001;98:3495–3496.
Kay N, Geyer S, Yaqoob I, et al: Thalidomide (Td) treatment in chronic lymphocytic leukemia (CLL): A North Central Cancer Treatment Group (NCCTG) Study. Blood 2003;102:5162 (Abstract).
Chanan-Khan A, Fallon A, Miller K, et al: Thalidomide (T) and Fludarabine (F) as front-line therapy for patients (pts) with previously untreated chronic lymphocytic leukemia (CLL): results of a phase I trial. Blood 2003;102:2502 (Abstract).
Dispenzieri A, Lacy M, Rajkumar S, et al: Poor tolerance to high doses of thalidomide in patients with primary systemic amyloidosis. Amyloid 2003;10:257–261.
Seldin D, Choufani E, Dember L, et al: Tolerability and efficacy of thalidomide for the treatment of patients with light chain-associated (AL) amyloidosis. Clin Lymphoma 2003;3:241–246.
Myers B, Lachmann H, Russell NH: Novel combination chemotherapy for primary (AL) amyloidosis; clinical, laboratory and serum amyloid protein scan improvement. Br J Haematol 2003;121:803–805.
Wilson EA, Jobanputra S, Jackson R, Parker AN, McQuaker IG: Response to thalidomide in chemotherapy-resistant mantle cell lymphoma: a case report. Br. J Haematol 2002;119:128–130.
Damaj G, Lefrère F, Delarue R, et al: Thalidomide therapy response in relapsed mantle cell lymphoma. Leukemia 2003;17:1914–1915.
Kaufmann H, Raderer M, Woehrer S, et al: Antitumor activity of rituximab plus thalidomide in patients with relapsed/refractory mantle cell lymphoma. Blood 2004;104:2269–2271.
Pro B, Younes A, Albitar M, et al: Thalidomide for patients with recurrent lymphoma. Cancer 2004;100:1186–1189.
Rule SAJ, Copplestone JA, Hamon MD, et al: Single agent thalidomide has significant activity in mantle cell lymphoma. Br J Haematol 2003;121:144.
Rule SAJ: Private communication. Department of Haematology and Oncology, Derriford Hospital, Plymouth, UK.
Dimopoulos M, Zomas A, Viniou N, et al: Treatment of Waldenstrom’s macroglobulinemia with thalidomide. J Clin Oncol 2001;19:3593–3595.
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