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Principii contemporane de tratament al anemiilor aplastice la copii


Author: Valentin Ţurea
Degree:doctor habilitat of medicine
Speciality: 14.00.09 - Pediatrics
Year:2004
Scientific consultant: Alexei Mascian
doctor habilitat, professor
Institution:
Scientific council:

Status

The thesis was presented on the 9 September, 2004
Approved by NCAA on the 14 October, 2004

Abstract

Adobe PDF document0.71 Mb / in romanian

Summary

The carried out research let us evaluate and introduce four programs of therapy of children’s aplasic anaemia (AA). 191 patients wereclassified into groups according to the t criteria Саmitta, based on gravity of hemodepression (the decrease of number of granulocytes, platelet, reticulocytes), so, these were groups with light, grave and severe AA.

The hormone glucocorticoid and splenectomy therapy , used in the treatment of 55 patients (splenectomy was used in 28 cases), ended with the worst results from all the groups of patients - 14 children (25,5 %). The actuarial five-year survival rate of these patients was 23,4±5,9%.

Cyclosporine A (CsA) monotherapy was used in the treatment of 68 patients. The therapeutic effect in this group of patients was recorded in 33 cases (48,5%), the best index was attained at moderate forms of AA-47,1 % at grave form and 81,3 % at light form of AA. The actuarial five-year survival rate of these patients was 49,9±6,7%. The determination of the concentration of medication in blood plasma let us not only maintain the normal level of CsA, but prevent such undesirable effect of the medication as toxicity and “cyclosporine dependence”.

The combination of CsA with antithymocyte/antilymphocyte globulin let us achieve the remission of disease in many patients (58,9%) and guarantee more high-quality and prolonged positive response to the therapy. The use of antilymphocyte globulin as the second line of therapy points at different mechanisms of the effect of these medications, and this fact determines the necessity of their simultaneous use.

The greatest effect was achieved in case of the use of the combined immunosuppressive therapy in conjunction with factors, stimulating the growth of colonies - the effectiveness of this type of therapy was fixed in 93,1 % of cases at children with AA. The actuarial five-year survival rate of t hese patients was 93,1%.

One of the most important aspects of the research is the analysis of infectious therapy of children with AA and its influence on the choice of one or another scheme of immunosuppressive therapy. The obtained results prove the early prescription of СsА and combin ed immunosuppressive therapy in conditions of corresponding antibacterial therapy, and the use of factors, stimulating the growth of colonies, “conditions” the hematopoietic cells predecessors and provides early granulocyte response.

The statistic analysis with the purpose of revelation of the factors, determining the course and prognosis of the disease, is carried out in this work. The data, received in all examined groups of patients, determine the significance of the phenomenon of positive response to the therapy and preservation of the base of hematopoiesis, presented by the preservation of the number of granulo cytes and platelet for the positive course of the disease.

The discriminate analysis of basic factors of AA let us determine with high level of reliability the forecasting significance of these factors for the prognosis of the disease - the probability of survival can be forecast in 83,3% of cases; the lethal outcome - with 100% exactness.