RESUMO
The aim of this study was to analyze the therapeutic results and survival of patients with myelofibrosis treated with ruxolitinib in comparison with a group on standard therapy. It is a cross-sectional, retrospective, non-interventional, real-life study that was performed between January 2000 and February 2023. Patients treated between 2000 and 2016, before the introduction of ruxolitinib, constituted the control group (n = 45), while those treated after May 2016, after ruxolitinib inclusion, constituted the active group (n = 66). Demographic characteristics, clinical indicators, the severity of the disease, and survival were explored using Kaplan-Meier survival analyses. Spearman's correlation, linear regression, and other statistical analyses were performed. According to the Kaplan-Meier analysis, there was a 75.33% reduction in the fatality risk in the sample. On a general-population level, the fatality risk in the group treated with ruxolitinib varied between 7.9% and 77.18% compared to that of the risk in the control group. There was a decrease in blood parameters (leukocytes, hemoglobin, and platelets) and spleen size. During the first six months, the spleen size of the patients on ruxolitinib decreased by 6%, and during the second six months, it decreased by another 9%. This study shows that patients in a real-life clinical setting treated with ruxolitinib exhibited improved clinical signs of the disease, had a lower symptom severity, and survived longer than patients on standard therapy before ruxolitinib's entrance into the national market. The improvements correlate with those reported in randomized clinical trials.
RESUMO
We aimed to explore symptom severity and adherence to therapy for patients with myelofibrosis treated with ruxolitinib in Bulgaria. It is a prospective, non-interventional study performed at the specialized hospital for active treatment of hematological diseases in Sofia during 2022-2023. Date of diagnosis, demographic characteristics, clinical indicators, ruxolitinib dose, and other data points were collected. Clinical indicators were assessed at baseline, in the middle, and at the end of observation. Severity of symptoms was measured with MPN-SAF TSS and adherence to therapy with the Morisky 4 questionnaire six times during the observation. The mean age of diagnosis was 58.5 years, with the average duration of disease of 3 years. Patients' laboratory results were within physiological ranges, with spleen size experiencing a constant decrease. The average value for the severity of the symptoms per MPN-SAF TSS results decreased significantly, indicating better disease control. The average adherence to therapy did not change and remained high at around 9 points, except for one patient. In conclusion the treatment of myelofibrosis patients with ruxolitinib decreased symptom severity and spleen size. Patients were adherent to the therapy over the observed period, but as treatment duration increases, the risk of adherence decreases.
RESUMO
BACKGROUND Hemophagocytic lymphohistiocytosis (HLH) is a rare syndrome. It is a result of uncontrolled hyperactivation of the reticuloendothelial system with a release of a huge amount of proinflammatory cytokines in the bloodstream, causing a cytokine storm. It may be primary due to genetic defects and secondary, triggered by viruses, bacteria, parasites, lymphoproliferative, and autoimmune diseases. We present a rare case of HLH triggered by leishmaniasis. HLH accounts for only about 1% of all leishmaniasis cases. CASE REPORT The patient was a 40-year-old previously healthy woman, who has been diagnosed with secondary HLH caused by leishmaniasis. A chronic Epstein-Barr virus (EBV) infection was initially mistakenly interpreted as a trigger agent, because Leishmania amastigotes, present on the first bone marrow biopsy, were not recognized. The treatment with cyclosporin A and corticosteroids suppressed the cytokine storm and prevented the development of complications. Two months later, because of a reactivation of the disease, the patient was referred to a hematologist. A second bone marrow aspiration was performed, in which numerous Leishmania parasites were finally identified. CONCLUSIONS The aim of this case report is to provide more information about the rare phenomenon of secondary HLH triggered by leishmaniasis. Early recognition of the syndrome and its triggering agents will improve disease outcomes and prevent unnecessary treatment with immunosuppressive drugs.
