title: Evaluation of efficacy of alemtuzumab in 5 patients with aplastic anemia and/or myelodysplastic neoplasm.
abstract: Patients with aplastic anemia or hypoplastic myelodysplastic syndrome (MDS) may respond to immunosuppressive therapy, including the anti-CD52 antibody alemtuzumab. We analyzed treatment responses to alemtuzumab in 5 patients with MDS or aplastic anemia (AA) evolving to MDS. Two patients with hypoplastic MDS (hMDS) showed a partial response (PR) to alemtuzumab. In both responding patients, a concomitant paroxysmal nocturnal hemoglobinuria (PNH) clone was detected before therapy. One responder relapsed after 15 months and underwent successful allogeneic stem cell transplantation. Both patients are still alive and in remission after 40 and 20 months, respectively. The other patients showed no response to alemtuzumab. One patient died from pneumonia 4 months after treatment. In summary, our data confirm that alemtuzumab is an effective treatment option for a subset of patients with MDS, even in the presence of a PNH clone.
authors.affiliation: Department of Internal Medicine I, Division of Hematology & Hemostaseology, AKH, Medical University of Vienna, Währinger Gürtel 18-20, 1090, Vienna, Austria. wolfgang.fuereder@meduniwien.ac.at.
authors.forename: Wolfgang
authors.initials: W
authors.lastname: Füreder
authors.affiliation: Department of Internal Medicine I, Division of Hematology & Hemostaseology, AKH, Medical University of Vienna, Währinger Gürtel 18-20, 1090, Vienna, Austria.
authors.forename: Sabine
authors.initials: S
authors.lastname: Cerny-Reiterer
authors.affiliation: Department of Internal Medicine I, Division of Hematology & Hemostaseology, AKH, Medical University of Vienna, Währinger Gürtel 18-20, 1090, Vienna, Austria.
authors.forename: Wolfgang R
authors.initials: WR
authors.lastname: Sperr
authors.affiliation: Department of Clinical Pathology, Medical University of Vienna, Vienna, Austria.
authors.forename: Leonhard
authors.initials: L
authors.lastname: Müllauer
authors.affiliation: Department of Laboratory Medicine, Medical University of Vienna, Vienna, Austria.
authors.forename: Eva
authors.initials: E
authors.lastname: Jäger
authors.affiliation: Department of Laboratory Medicine, Medical University of Vienna, Vienna, Austria.
authors.forename: Ilse
authors.initials: I
authors.lastname: Schwarzinger
authors.affiliation: Department of Internal Medicine V, Hospital Hietzing, Vienna, Austria.
authors.forename: Klaus
authors.initials: K
authors.lastname: Geissler
authors.affiliation: Department of Internal Medicine I, Division of Hematology & Hemostaseology, AKH, Medical University of Vienna, Währinger Gürtel 18-20, 1090, Vienna, Austria.
authors.forename: Peter
authors.initials: P
authors.lastname: Valent
mesh_terms: D000328:Adult; D000368:Aged; D000074323:Alemtuzumab / Q000008:administration & dosage*; D000741:Anemia, Aplastic / Q000150:complications / Q000188:drug therapy* / Q000473:pathology*; D000970:Antineoplastic Agents / Q000008:administration & dosage*; D000074322:Antineoplastic Agents, Immunological / Q000008:administration & dosage; D005260:Female; D006801:Humans; D008137:Longitudinal Studies; D008297:Male; D008875:Middle Aged; D054437:Myelodysplastic-Myeloproliferative Diseases / Q000150:complications / Q000188:drug therapy* / Q000473:pathology*; D016896:Treatment Outcome
chemical_list: D000970:Antineoplastic Agents; D000074322:Antineoplastic Agents, Immunological; D000074323:Alemtuzumab
keywords: Myelodysplastic syndrome; Paroxysmal nocturnal hemoglobinuria (PNH); Progenitor cells