Compliance during therapy of patients with chronic myeloid leukemia

Stałe utrzymywanie właściwego dawkowania doustnych inhibitorów kinazy tyrozynowej (TKI) BCR-ABL1 ma zasadnicze znaczenie w uzyskaniu optymalnej odpowiedzi u chorych z przewlekłą białaczką szpikową (CML). Dlatego przestrzeganie zaleceń lekarskich (compliance, adherence) u tych pacjentów jest bardzo ważne. Często problem ten jest przypisywany jedynie choremu, chociaż wpływ na przestrzeganie zaleceń mają także sam lekarz i system opieki zdrowotnej, przebieg choroby i leczenia, a także czynniki ekonomiczne i społeczne. W pracy przedstawiono problem nieprzestrzegania zaleceń lekarskich u chorych z CML leczonych TKI oraz działania, które należy podjąć, aby to zjawisko ograniczyć. Hematologia 2010; 1, 3: 239-243Continuous and adequate BCR-ABL1 tyrosine kinase inhibitors (TKI) dosing is essential to achieve optimal therapeutic outcomes in patients with chronic myeloid leukemia (CML). Hence, patient adherence, defined as the extent to which a person’s behavior corresponds with the agreed recommendations of a healthcare provider, is critical. Though often trivialized as a patient problem, adherence behavior is influenced also by the clinician and the healthcare system, the disease and its treatment, and economic and social factors. In this paper, the issue of non compliance in patients with CML treated with TKI is reviewed. Possible interventions how to improve the adherence is also discussed. Hematologia 2010; 1, 3: 239-24

Unusual clinical course of polycythemia vera – case report

Polycythemia vera is mainly diagnosed in the age of 40–80. In people under 20 years of age, it is very rare. A typical marker for confirmation of the diagnosis of polycythemia vera is a mutation of gene JAK 2 in exon 14, in position 617, which is found in about 95% of patients with this diagnosis.Patients without the mutation require additional examination to state the final diagnosis. A search for the mutations in other exons due to the diversity of mutations and the related complexity of molecular testing is not applicable in routine laboratory diagnostics. Here we present a case of polycythemia vera diagnosed in a patient aged 19 without typical mutation of gene JAK 2 in exon 14. Conducted additional tests revealed presence of the mutation in exon 12 of gene JAK 2. The most common complication of polycythemia vera is the arterial and venous thrombosis, which could be the result of not only an increased hematocrit, but also coexisting congenital disorders leading to thrombophilia, as occurred in the presented case

The EUTOS long-term survival (ELTS) score is superior to the Sokal score for predicting survival in chronic myeloid leukemia

Prognostic scores support clinicians in selecting risk-adjusted treatments and in comparatively assessing different results. For patients with chronic-phase chronic myeloid leukemia (CML), four baseline prognostic scores are commonly used. Our aim was to compare the prognostic performance of the scores and to arrive at an evidence-based score recommendation. In 2949 patients not involved in any score development, higher hazard ratios and concordance indices in any comparison demonstrated the best discrimination of long-term survival with the ELTS score. In a second step, of 5154 patients analyzed to investigate risk group classification differences, 23% (n = 1197) were allocated to high-risk by the Sokal score. Of the 1197 Sokal high-risk patients, 56% were non-high-risk according to the ELTS score and had a significantly more favorable long-term survival prognosis than the 526 high-risk patients according to both scores. The Sokal score identified too many patients as high-risk and relatively few (40%) as low-risk (versus 60% with the ELTS score). Inappropriate risk classification jeopardizes optimal treatment selection. The ELTS score outperformed the Sokal score, the Euro, and the EUTOS score regarding risk group discrimination. The recent recommendation of the European LeukemiaNet for preferred use of the ELTS score was supported with significant statistical evidence

Recommendations for assessment of co-morbidities and tyrosine kinase inhibitor choice in patients suffering from chronic myeloid leukemia

Treatment of chronic myeloid leukemia with tyrosine kinase inhibitors (TKI) is very effective. The vast majority of patients achieve deep and long-lasting therapeutic responses including those on molecular level. Understanding the adverse effects of TKIs is of great importance during life-long therapy. The safety profile of TKI should be considered especially when used in patients suffering from various co-morbidities. In this paper, treatment recommendations for patients suffering from diabetes mellitus, atherosclerosis with circulation disturbances in peripheral arteries, congestive heart failure, pulmonary hypertension and pleural effusions are presented. Drug interactions between TKIs and medicaments used in the treatment of the above-mentioned co-morbidities are discussed

Clinical characteristics of essential thrombocythemia patients depend on the mutation status

The impact of the mutation status on the clinical course and the outcome of essential thrombocythemia (ET) patients has not yet been completely established. A total of 171 patients with diagnosed ET were tested and subsequently grouped, according to their mutation status – Janus Kinase 2 () – 112 patients, calreticulin () – 36 patients, and thrombopoietin receptor () – 5 patients. Moreover, 18 individuals were triple-negative (with non-mutated , , and ). -mutated patients preferentially were male, with higher platelets (PLT) counts (mean PLT = 1 002.3) and lower hemoglobin and hematocrit levels at the diagnosis, compared to the (mean PLT = 933.6), (mean PLT = 940.8) and triple-negative patients (mean PLT = 822.6) ( = 0.0035). The patients with mutated, and the triple-negative ones had a lower risk of arterial and venous thrombosis (3% and 5.6% cases at the time of diagnosis, respectively) than the patients with mutation (7.2%) ( = 0.9210). The overall survival rate did not differ statistically between the groups

Rekomendacje PALG dotyczące diagnostyki i leczenia przewlekłej białaczki szpikowej w 2013 r.

Tyrosine kinase inhibitors (TKIs) have dramatically improved the results of therapy of chronic myeloid leukemia. However the management with more potent second generation TKIs (2GTKIs) is required for imatinib-intolerant and imatinib-resistant patients. The frontline use of 2GTKIs results in higher rates of deeper and faster responses than with imatinib, it can enable many patients to qualify for participation in studies of treatment-free remission. The BCR/ABL transcript level at 3 months of TKI treatment has become a new prognostic factor. In this paper the recent clinical results of chronic myeloid leukemia therapy with imatinib in first line and with 2GTKIs in first and second line settings are reviewed, and updated PALG recommendations regarding the diagnosis, standard monitoring procedures, definitions of responses and treatment are presented

The EUTOS long-term survival (ELTS) score is superior to the Sokal score for predicting survival in chronic myeloid leukemia

Prognostic scores support clinicians in selecting risk-adjusted treatments and in comparatively assessing different results. For patients with chronic-phase chronic myeloid leukemia (CML), four baseline prognostic scores are commonly used. Our aim was to compare the prognostic performance of the scores and to arrive at an evidence-based score recommendation. In 2949 patients not involved in any score development, higher hazard ratios and concordance indices in any comparison demonstrated the best discrimination of long-term survival with the ELTS score. In a second step, of 5154 patients analyzed to investigate risk group classification differences, 23% (n = 1197) were allocated to high-risk by the Sokal score. Of the 1197 Sokal high-risk patients, 56% were non-high-risk according to the ELTS score and had a significantly more favorable long-term survival prognosis than the 526 high-risk patients according to both scores. The Sokal score identified too many patients as high-risk and relatively few (40%) as low-risk (versus 60% with the ELTS score). Inappropriate risk classification jeopardizes optimal treatment selection. The ELTS score outperformed the Sokal score, the Euro, and the EUTOS score regarding risk group discrimination. The recent recommendation of the European LeukemiaNet for preferred use of the ELTS score was supported with significant statistical evidence.Peer reviewe

Retrospective analysis of nilotinib and dasatinib efficacy in second line treatment of chronic myeloid leukemia in Polish hematological centers

Until now, there has been no randomized study directly comparing the activity of second-generation BCR-ABL tyrosine kinase inhibitors (TKI-2G) nilotinib and dasatinib in chronic myeloid leukemia (CML). The aim of our study was to retrospectively analyze efficacy of nilotinib and dasatinib in the real life setting of CML with resistance or intolerance of imatinib. Of 108 included patients treated in polish hematology centers, 75 received dasatinib and 33 patients received nilotinib. Rates of complete cytogenetic response (CCyR) did not differ between the two groups of patients. After six months of therapy, CCyR was achieved in 34.7% of patients treated with dasatinib and 38.7% treated with nilotinib (p=0.86), while after 12 months, the CCyR rates were 60.0% and 77.0% in dasatinib and nilotinib groups, respectively (p=0.11). Moreover, we have not observed any significant difference in the probability of progression-free survival (p=0.89) or overall survival (p=0.99) between patients treated with these two TKI-2G. In conclusion, the results of our analysis indicate that nilotinib and dasatinib have comparable and satisfactory efficacy in the treatment of CML patients refractory or intolerant to imatinib. Our findings support current strategy of choice of IKT-2G according to drug toxicity profile and risk of specific adverse events in an individual patient