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Evaluation of bone marrow therapeutic response in patients with high risk myelodysplastic syndrome


Authors: P. Vašeková 1;  L. Plank 1,2;  P. Szépe 1,2;  J. Marcinek 1,2;  T. Balhárek 1,2
Authors‘ workplace: Konzultačné centrum bioptickej diagnostiky ochorení krvotvorby, Ústav patologickej anatómie JLF Univerzity Komenského a UN v Martine 1;  Konzultačné centrum bioptickej diagnostiky ochorení krvotvorby Martinské bioptické centrum, s. r. o., v Martine 2
Published in: Transfuze Hematol. dnes,21, 2015, No. 2, p. 67-73.
Category: Comprehensive Reports, Original Papers, Case Reports

Overview

Myelodysplastic syndrome (MDS) represents a heterogeneous group of clonal disorders of haematopoietic bone marrow (BM) stem cells manifesting as ineffective haematopoiesis and morphologically visible dysplasias, leading to peripheral cytopenias involving one or more blood cell lineages. The clinical course of the disease is variable and individual. It may lead either to the onset of fatal cytopenias due to progressive BM failure or, on the contrary, to the development of secondary acute myeloid leukaemia (AML). Diagnosis of the disease requires close correlation of the clinical picture, laboratory parameters and standardized evaluation of BM biopsy. Despite some progress in the treatment of patients with higher risk MDS (IPSS group int-2 and high), the current therapeutic options are limited and usually allogeneic haematopoietic stem cell transplantation, chemotherapy, supportive care and hypomethylating therapy may be considered.

From 2008–2014, 76 patients were identified with high-risk MDS in our biopsy register who were treated with chemotherapy, the hypomethylating agent azacytidine or stem cell transplantation and whose bone marrow bio-psies were available before and after treatment. The effect of the applied therapy was evaluated with focusing on the histopathological type of MDS before and after therapy and also on any change of the histo-morphological picture, particularly the BM blast count, progression to secondary AML and progression of myelofibrosis. The series consisted of 76 patients (47 men and 29 women); the average age was 62 years (27–82 years). The most common type of high-risk MDS was RAEB 2 (32 patients) and secondary AML (21 patients). 39 patients were treated only with the hypomethylating agent azacytidine; in 12 cases chemotherapy was also administered. 22 patients were treated with chemotherapy alone. In 3 patients, only information about peripheral stem cell transplantation was available, the total number of transplantations was 7. Hypomethylating therapy resulted in: a) blast cell count reduction in 16% of cases, b) disease stabilization without striking changes of the morphological picture in nearly 42% of cases, c) rapid disease progression despite therapy in approx. 35% of cases and d) gradual progression of myelofibrosis during the disease course in 8% of cases. Whether the progression of fibrosis represents an effect of hypomethylating treatment or is part of the natural course of MDS remains unknown, the same is true for the persistence or progression of cytopenia/s.

Key words:
high risk MDS, hypomethylation therapy, bone marrow trephine biopsy


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