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ABSTRACT Background: There has been a revolution in the treatment of Chronic Myeloid Leukemia since imatinib's introduction. However, patient adherence has a great impact on the response obtained with medical treatment. This study's objective was to analyze the drug adherence and the factors that influenced it in patients with Chronic Myeloid Leukemia in a referral hospital in the Brazilian Amazon. Method: This was a retrospective study including 120 patients with Chronic Myeloid Leukemia from January 2002 to December 2014. The adherence was estimated by the Proportion of Days Covered and the persistence by Kaplan-Meier analysis. The data was analyzed in Epi Info 7® software and the relationship between the variables was analyzed by Fisher's exact test. A p-value lower than 0.05 was considered significant. Results: Twenty-seven patients (22.5%) were considered non-adherent. There has been irregular medication use and disinterest in the treatment in 20.83% (n = 25), of which 13 were considered non-adherent (p < 0.001). A total of 26.67% (n = 32) abandoned the treatment for a period. Of those, 56.25% (n = 18) were non-adherent (p < 0.001). Distance to the hospital, lack of medication and side-effects were all non-significant to low adherence. At the end of a 360-day follow-up, 44.16% (n = 53) of patients presented a break in persistence, whose average was 255 days. Conclusion: The adherence found in this study was similar to that found in others of its kind. The only factors that negatively influenced the adherence were disinterest and abandonment of treatment, which can reflect the need to individually educate Chronic Myeloid Leukemia patients.

Resumo em Inglês:

ABSTRACT Introduction and objective: In this study, we evaluated the influence of the transcript type on hematological and clinical parameters, as well as the event-free survival of 50 patients in the Chronic myeloid leukemia chronic phase. Methods: We reviewed the medical records of 55 patients with Chronic myeloid leukemia. The eligibility criteria were based on the availability of hematological and clinical baseline data in the medical records. Data on BCR-ABL transcripts were obtained from medical records. Results: Eighteen patients (36%) had the b2a2 transcript, 24 (48%) had b3a2 and 8 (16%) had b2a2/b3a2. The median platelet count for transcripts b2a2, b3a2 and b2a2/b3a2 was 320.65 × 103/L, 396 × 103/L, and 327.05 × 103/L, respectively (p = 0.896). We could not find any differences in relation to the other hematological parameters, when compared to the transcript type. Comparison between spleen and liver size and type of transcript did not differ inside the groups (p = 0.395 and p = 0.647, respectively) and the association between risk scores and transcript type did not show statistical significance (p > 0.05). The 21-month probability for event-free survival was 21%, 48% and 66% for the transcripts b2a2, b3a2 and b2a2/b3a2 respectively (p = 0.226) Conclusion: We conclude that the expression BCR-ABL transcripts have no influence on hematological, clinical and event-free survival parameters of patients in the Chronic myeloid leukemia chronic phase.

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ABSTRACT Background: Thrombotic thrombocytopenic purpura (TTP) is a potentially fatal disease that requires early diagnosis and treatment that can be made possible by applying the PLASMIC score. This study aims to evaluate this score applicability for patients with suspected TTP in a developing country. Methods: This was a retrospective study performed at a tertiary hospital in the northeastern region of Brazil. Patients were analyzed in two groups: ADAMTS13 activity <10% and activity >10%. Patients were stratified according to the PLASMIC score, and the level of agreement between the PLASMIC score and the ADAMTS13 activity was evaluated. Results: Eight patients with thrombotic microangiopathy were included. Four patients had ADAMTS13 activity <10%, all with a PLASMIC score =6. The other four had ADAMTS13 activity >10%, all with a score <6. Based on a score =6 for presumptive diagnosis of TTP, we attained a 100% diagnostic accuracy in our sample. The PLASMIC score was also able to accurately predict response to plasma exchange and the risk of long-term unfavorable outcomes. Conclusions: The reproducibility of the PLASMIC score was quite satisfactory in our sample. It accurately discriminates between patients who had ADAMTS13 deficiency and those with normal enzyme activity, precluding the need for specific laboratory evaluation, which is not always available. This score can be useful for an early diagnosis and indicates which patients will benefit from the treatment in developing countries.

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ABSTRACT We analyzed the management and outcomes of pregnancies of patients with chronic myeloid leukemia at a single center over fifteen years. Among the 203 CML female patients, there were ten pregnancies in seven women, all of them not planned. In three cases, the chronic myeloid leukemia diagnosis was made during pregnancy. Five patients received tyrosine kinase inhibitors in the first weeks of pregnancy and the drug was interrupted until delivery. One patient lost complete cytogenetic response, and two patients lost the hematological response. A patient with a stable major molecular response had two successful pregnancies without loss of response. There were four premature births. There were no maternal adverse events, fetal malformation or death. All patients received Interferon-alpha during gestation, and two received hydroxyurea for a short period. Leukapheresis was performed in two patients for hyperleukocytosis control. One patient with sickle cell disease died from disease progression six months after delivery. Conclusions: The tyrosine kinase inhibitors ministration should be interrupted during pregnancy. Patients should be advised to achieve a stable and deep molecular response if they plan to conceive, to avoid the risk of disease progression.

Resumo em Inglês:

ABSTRACT Background: Preeclampsia is one of the major health problems causing maternal morbidity and mortality, complicating 3-8% of pregnancies. It has been suggested that the alterations in the coagulation and fibrinolysis play a role in the pathogenesis of preeclampsia. The markers of platelet activation include platelet count, platelet distribution width, mean platelet volume and plateletcrit. Study design: It was a case-controlled study which included a total of 60 patients (30 cases and 30 controls). Blood samples were collected and the platelet indices - platelet count, plateletcrit, mean platelet volume and platelet distribution width - were evaluated using the Sysmex XN1000 and compared between the two groups. Results: The MPV and PDW also showed a significant difference (p > 0.05) between the two groups, with a significant positive correlation with increasing blood pressure (MPV - r = +0.6126, p < 0.05 and PDW - r = +0.6441, p < 0.05). The PC and PCT had lower values in the preeclampsia patients, however the difference between the two groups was not statistically significant. Conclusion: The MPV and PDW showed a significant difference between the two groups and increasing values with increasing BP. However, the PC and PCT in our study did not show a significant correlation with preeclampsia. Thus, the platelet indices, mainly the MPV and PDW, which are economical and easily available, can be reliable in the prediction and early diagnosis of preeclampsia, as well as a marker for the severity of preeclampsia.

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ABSTRACT Introduction and objective: Hairy cell leukemia is an uncommon, indolent B-cell lymphoproliferative disorder. Therapy with cladribine (2-chlorodeoxyadenosine) is able to induce complete remission (CR) in the majority of patients after a single course of treatment. We report the outcomes of patients treated at Aga Khan University Hospital, Karachi, Pakistan. Methods: This was a retrospective review. Medical records of patients were used to collect data. Results: A total of 21 patients with hairy cell leukemia were treated with cladribine. All patients achieved an initial CR. Four patients (19%) required hospitalization and therapy for neutropenic fever. Six patients (29%) relapsed at a median of 48 months. All 6 patients were treated for relapse, out of which 4 achieved CR, 1 had partial response and 1 had refractory disease. The overall survival rate was 90.5%, with a median follow-up of 35 months. Conclusion: A single course of cladribine is able to induce CR in a vast majority of patients. Unfortunately, relapse is not uncommon. Patients who relapse can be successfully retreated with cladribine. Cladribine has impressive efficacy and a favorable acute and long-term toxicity profile when administered to patients with HCL.

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ABSTRACT Objective: This is a quantitative transversal study that aimed to analyze the sociodemographic and clinical characteristics of thalassemia major patients with and without diabetes mellitus. Method: The cohort consisted of 31 thalassemia major patients from a reference center of treatment in Brazil in 2016. The data were obtained from an interview using a questionnaire containing demographic and clinical variables. The results show that 16.1% of the participants with thalassemia major had diabetes mellitus. The participants' ages ranged from 20 to 48 years, with an average of 35 years, mostly students and starting in the formal job market. The most commonly used treatment was the oral desferasirox and the transfusion treatment interval was 15-22 days. Results: Patients with thalassemia major and diabetes mellitus presented altered values of fasting glycemia, serum alanine transaminase, magnetic resonance imaging and bone densitometry. Conclusion: It was concluded that knowledge of the characteristics of this population contributes in the proposal of effective educational strategies in light of the complexity of care and the progression of the disease.

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ABSTRACT Objective: To develop and validate DFConhecimento, an instrument to assess Brazilian healthcare professional providers' knowledge on sickle cell disease. Method: Study carried out in four stages: (1) instrument development; (2) content validation by an Expert Committee; (3) cultural adequacy check at pre-test; (4) instrument reliability analysis by healthcare professional providers supported by Intraclass Correlation Coefficient calculation. The data for content validation and reliability analyses were collected through the web tool eSurv and analyzed within the statistical software and environment R. Results: The instrument, consisting of 13 multiple-choice questions, showed acceptability, with an average Content Validity Index of 0.88. The reliability analysis showed moderate agreement (0.67) indicating that test-retest reproducibility is acceptable. Conclusion: The instrument DFConhecimento showed reliability and internal consistency, proving suitable for measuring Brazilian healthcare professional providers' acquisition of knowledge on sickle cell disease.

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ABSTRACT Background: This study aimed to define the sex-divided reference intervals for platelet indices (mean platelet volume (MPV), platelet distribution width (PDW), platelet large cell ratio (P-LCR) and plateletcrit (PCT)) on the Sysmex XN-10. Methods: A total of 2376 samples were assayed for full blood count on the Sysmex XN-10 haematology analyzer. After removing the outliers, reference intervals were calculated using the mean ± 2SD. The P value 0.05 was adopted to denote statistical significance. Results: There was a statistical significance (α = 0.05) between sex-divided reference intervals for MPV (p = 0.007), P-LCR (p = 0.015) and PCT (p < 0.001), thus separate reference intervals were calculated for these indices, with orientation to the sex-divisions as follows: MPV, 9.1-13.0 fL [males] and 9.2-12.8 fL [females]; P-LCR, 17.6-47.0% [males] and 17.8-47.8% [females]; and PCT, 0.16-0.35% [males] and 0.18-0.37% [females]. No significance was found between sex-divided reference intervals for the PDW (p = 0.838), therefore a reference interval for total individuals was calculated for this platelet measurement as 9.3-17.3 fL. Conclusion: This study showed comparable reference intervals, using the Sysmex XN-10, with the previous literature. It determined the need to define sex-specific reference intervals for the MPV, P-LCR and PCT, but not for the PDW. These reference intervals will allow for low and high values to be facilitated in order to do further research and guide platelet disorder management.

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ABSTRACT Objectives and methods: We evaluated possible relationships between echocardiographic findings and clinical and laboratory parameters, in a cohort of Brazilian patients diagnosed with sickle cell/β-thalassemia, to better understand the cardiac involvement in this disease. Results: Left atrial (LA) and left ventricular (LV) dilation were found in 19.5 and 11% of patients, respectively; systolic left ventricular dysfunction was present in a single patient. There were no differences in masses and volumes of cardiac chambers comparing Sβ0 with Sβ+ patients, and no relationship between these parameters and specific complications of the disease. However, parameters of altered ventricular geometry were significantly correlated with serum creatinine, hepatic transaminases and bilirubin levels. Moreover, 3 patients presented stroke; they were significantly older [53 (41-56) × 37.5 (18-70), p = 0.048], had higher values of LV posterior wall diastolic thickness [10 (10-11) × 8 (6-14), p = 0.03], LV mass [226 (194-260) × 147 (69-537), p = 0.039] and LA/aortic ratio [1.545 (1.48-1.61) × 1.26 (0.9-1.48), p = 0.032]. Conclusions: Cardiac involvement in this disease does not appear to depend on the thalassemia phenotype. The presence of signs of myocardial remodeling in this group of patients was related to multi-organ impairment and rendered a higher propensity for stroke in older patients, suggesting the need for greater vigilance and control of associated factors.

Resumo em Inglês:

ABSTRACT Reevaluation of the deferral from voluntary blood donation by men who have sex with men (MSM) is being discussed in several countries, motivated by the need to ensure a blood supply free from transfusion-transmissible infections (e.g., HIV, syphilis). Policies being considered include: permanent exclusion for any male-male sexual encounter, temporary deferral (3 months, 12 months, 5 years) from the last encounter, or specifying behaviors that differentiate MSM at high risk from those at low risk. Current Brazilian regulations defer MSM from blood donation for 12-months after the last male-male sexual encounter. Broad epidemiological evidence indicates that many MSM are at increased risk for HIV in the present era, and few data exist to distinguish which men are likely to be in the immunological window for detection of these infections. A multicenter study developed in Brazil demonstrated that the history of male-male sex was the most strongly associated with being an HIV-positive blood donor. Meanwhile, the blanket deferral of MSM from blood donation has generated considerable controversy. Rejection of the deferral policies stems in part from perspectives defending human rights, promoting equality and citizenship, and alleging bias and discrimination. The objective of this report is to discuss the current situation of blood donation among MSM in Brazil. We highlight the lack of evidence for a true risk profile for male-male sex in the context of blood donation upon which to base sound policy. We recommend research to establish effective and acceptable criteria for blood donation by MSM and other blood donors.

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ABSTRACT Background: Older patients with acute myeloid leukemia are particularly difficult to treat, as they have a high risk of comorbidities, poor performance status and less tolerability to chemotherapy, as well as a more aggressive disease biology, responsible for the resistance to treatment. There is a need to explore novel therapeutic agents that are more effective and tolerable. Venetoclax, a BCL-2 inhibitor is a promising agent, as BCL-2 overexpression is present in 84% of acute myeloid leukemia patients at diagnosis and 95% of patients at relapse and has been associated with leukemia cell survival, chemotherapy resistance and poor prognosis. Objective: To review the available data about venetoclax in acute myeloid leukemia and how it can influence the treatment in older patients. Methods: Using the Pubmed database, we selected 29 articles published within the last 15 years, considering preclinical and clinical trials and review studies that combined venetoclax with acute myeloid leukemia. Results: Venetoclax has demonstrated promising results in preclinical and clinical trials, especially in patients with poor prognosis and the IDH mutation, with an excellent side-effect profile. However, resistance seems to develop rapidly with venetoclax monotherapy, because of antiapoptotic escape mechanisms. Conclusions: While the results with the use of venetoclax seem encouraging, it is not likely that targeting a single pathway will result in long-term disease control. The solution includes the use of combined therapy to block resistance mechanisms and enhance apoptosis, by reducing MCL-1, increasing BIM or inhibiting the complex IV in the mitochondria.