Lege Artis Medicinae

[MYELODYSPLASTIC SYNDROMES - NEW THERAPEUTIC OPTIONS]

GADÓ Klára

DECEMBER 20, 2007

Lege Artis Medicinae - 2007;17(12)

[Myelodysplastic syndrome is a heterogeneous group of acquired clonal disorders of the haematopoietic stem cell characterized by ineffective haematopoiesis, peripheral cytopenia, and a high risk of progression to acute leukaemia. It is a common malignant disease with an increased incidence in the elderly population. Classification is based on a 1999 WHO recommendation, in which morphological features as well as clinical and cytogenetic characteristics are taken into account. Combined with the International Prognostic Scoring System (1997), it is suitable to predict prognosis and response to therapy. Clinical features include symptoms caused by anaemia, infections, and bleeding. Diagnosis is based on peripheral cytopenia and dysplastic morphology, as well as normal or increased cellularity in the bone marrow, with more than 10% of dysplastic cells. The verification of cytogenetic abnormalities is important both for confirming the diagnosis and predicting the prognosis. When designing the treatment strategy, it is essential to take the risk of leukaemia into account. On the other hand, the general state of the patient and the presence of accompanying diseases should also be considered. The goal of the treatment is to increase cell count and to decrease transfusion requirement, eventually to improve quality of life. Supportive therapy is an essential part of the management. In addition, growth factors, immunosuppressive and immunomodulatory agents, low-dose chemotherapy may be applied. Today, cure can only be achieved by allogenic stem cell transplantation. Recent findings in the epigenetic intracellular regulation allowed the definition of new therapeutic targets to develop drugs such as inhibitors of DNA methyltransferase and histone deacetylase.]

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Lege Artis Medicinae

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MATOS Lajos

Lege Artis Medicinae

[AUTOIMMUNE DISEASES TREATED BY AUTOLOGOUS HAEMOPOIETIC STEM CELLS]

ZEHER Margit, SZEGEDI Gyula

[Autoimmune diseases are of multifactorial origin. Due to the disturbed immune tolerance, autoreactive T and B cells target self antigens, which lead to permanent organ damages. Despite of the recently introduced therapeutic protocols, the disease has a chronic course, in many cases with lethal outcome. The efficacy of stem cell therapy has been observed in animal models of autoimmune diseases and in autoimmune diseases associated with haematological disorders. Although this approach has been applied for more than 30 years, its widespread use has been delayed by the serious side effects caused by the conditioning treatments based on oncological protocols. The evaluation of the data of patients who had undergone autologous stem cell therapy revealed that the use of protocols for conditioning treatments that mostly cause lymphoablation, and also, if the procedures are carried out in specialized centres significantly reduce mortality, while the therapeutic efficacy remains optimal. Indications for autologous CD34+ stem cell therapy in patients with autoimmune diseases are internationally established and accepted. New, multicentric investigations have been launched in order to compare the efficacy of various protocols.]

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NEMESÁNSZKY Elemér

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