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Low-Dose Thalidomide Ameliorates Cytopenias and Splenomegaly in Myelofibrosis With Myeloid Metaplasia: A Phase II Trial

From the Laboratory of Medical Informatics, Unit of Clinical Immunology and Immunohematology, Transfusion Service, Unit of Internal Medicine III, Istituto di Ricovero e Cura a Carattere Scientifico Policlinico San Matteo, Pavia; Unit of Hematology, Azienda Ospedaliera Careggi, Firenze; Unit of Hematology, Policlinico Lescotte, Siena; Unit of Hematology, Azienda Ospedaliera Torrette-Umberto I, Ancona; DH Ematologico, Arcispedale San Maria Nuova, Reggio Emilia; Unit of Hematology, Ospedale G. Moscati, Avellino; Clinica Medica II, Azienda Ospedaliera di Padova; Divisione di Ematologia, Ospedale Casa Sollievo della Sofferenza, San Giovanni Rotondo, Foggia; Divisione di Ematologia CTMO, Ospedale A. Businco, Cagliari; Divisione di Medicina, Ospedale Civile San Biagio, Clusone, Bergamo; Cattedra di Ematologia, Ospedale Sant'Eugenio, Roma, Italy; Département d'Hématologie, Université Catholique de Lille, Lille; Service de Maladies du Sang Centre Hospitalier de Bayonne; Service d' Hématologie Clinique, Limoges Institut National de la Santé et de la Recherche Médicale U268, Institut André Lwoff, Hôpital Paul Brousse, Villejuif; and Service d'Hématologie Clinique, Centre Hospitalier de Lens, France.

Address reprint requests to Giovanni Barosi, MD, Laboratory of Medical Informatics, IRCCS Policlinico San Matteo, viale Golgi 19, 27100 Pavia, Italy; e-mail: barosig{at}smatteo.pv.it

PURPOSE: A phase II dose-escalation trial was conducted to ascertain low-dose thalidomide safety and response in patients with advanced myelofibrosis with myeloid metaplasia (MMM).

PATIENTS AND METHODS: Thalidomide was administered together with current therapy to 63 patients, starting at 50 mg daily and increasing to 400 mg as tolerated.

RESULTS: Half of the patients sustained daily doses more than 100 mg and the drop-out rate was 51% at 6 months: the drop-out rate was lower in patients with high baseline fatigue score. At efficacy analysis, anemia was ameliorated in 22% of the patients and transfusions were eliminated in 39% of transfusion-dependent patients. Platelet count increased by 50 x 10⁹/L or more in 22% of patients with an initial count lower than 100 x 10⁹/L. Splenomegaly decreased by more than 50% of the initial size in 19% of patients. Reduction of an overall disease severity score occurred in 31% of patients and was associated with a significant reduction of fatigue. Disease severity amelioration was independently predicted by a high baseline myeloproliferative index (ie, large splenomegaly, thrombocytosis, or leukocytosis).

CONCLUSION: Low-dose thalidomide displays an acceptable toxicity profile and provides an objective and subjective advantage to a relevant portion of MMM patients.

Presented in abstract form at the 8th Annual Congress of the European Hematology Association, Lyon, France, June 2003.

The Italian authors belong to the Italian Registry of Myelofibrosis with Myeloid Metaplasia and the French authors belong to the French Research Group on Myelofibrosis.

Authors' disclosures of potential conflicts of interest are found at the end of this article.

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