Cryopreserved versus non-cryopreserved stem cell autografts in multiple myeloma a restrospective cohort study.

The use of non-cryopreserved hematopoietic stem cells (HSC) can be an alternative to the traditional cryopreserved infusions of HSCs in autologous stem cell transplantation (aHSCT). After high-dose melphalan conditioning (HDM), we sought to compare time to engraftment, overall survival, and safety in multiple myeloma (MM) patients undergoing a first aHSCT after high-dose melphalan conditioning (HDM). We conducted a cohort study from March 2018 to December 2019. Of all autologous transplants performed during this period, 105 were for MM as the first consolidation. Fifty-one patients received a cryopreserved graft; the remaining 54 patients received a fresh infusion. General clinical characteristics were similar between these two groups. Cell viability was higher in non-cryopreserved grafts (95% vs. 86% p < 0.01). Four deaths occurred during hospitalization in the cryopreserved group, one in the non-cryopreserved group. The cumulative incidence of neutrophil and platelet engraftment on D + 25 was higher in the non-cryopreserved compared to the cryopreserved group (98% vs 90% p < 0.01 and 96.2% vs 72.54% p < 0.01 respectively). Additionally, the hospital length of stay was reduced by 4 days for patients for the non-cryopreserved cohort. In summary, the use of non-cryopreserved HSCs after HDM is safe and effective compared to patients who received a cryopreserved graft.

Advances in the Treatment of Cardiac Amyloidosis.

OPINION STATEMENT: Cardiac amyloidosis is associated with a high mortality rate, a long delay between the first signs and the diagnosis but a short interval between diagnosis and death. This scenario has changed recently due to improved disease awareness among doctors and significant progress in diagnosis thanks to multimodal imaging and a multidisciplinary approach. Therefore, during the last few years, we have had access to specific therapies for those patients. Those therapies are quite different depending on the type of amyloidosis, but there has been real progress. Systemic light chain amyloidosis (AL) with cardiac involvement is the most common form of cardiac amyloidosis. The severity of heart disease dictates the prognosis in AL amyloidosis. Advances in chemotherapy and immunotherapy that suppress light chain production have improved the outcomes. These recent improvements in survival rates have enabled therapies such as implanted cardiac defibrillators and heart transplantation that were usually not indicated for patients with advanced light chain amyloid cardiomyopathy to now be applied in selected patients. For transthyretin amyloidosis (ATTR), the second most common form of amyloidosis with cardiac involvement, there is also significant progress in treatment. Until recently, we had no specific therapy for ATTR cardiomyopathy (ATTR-CM), though now disease-modifying therapies are available. Therapies that stabilize transthyretin, such as tafamidis, have been shown to improve outcomes for patients with ATTR-CM. Modern treatments that stop the synthesis of TTR through gene silencing, such as patisiran and inotersen, have shown positive results for patients with TTR amyloidosis. Significant progress has been made in the treatment of amyloid cardiomyopathy, and hopefully, we will see even more progress with the spread of those treatments. We now can be optimistic about patients with this disease.

Regressão do depósito amiloide e normalização da função ventricular em paciente com amiloidose de cadeias leves tipo Kappa / Regression of amyloid deposit and normalization of ventricular function in a patient with Kappa-type light chain amyloidosis

INTRODUÇÃO: A amiloidose por depósito de cadeias leves (AL) é reconhecida como um tipo de amiloidose associado a quadro clínico devastador e prognóstico sombrio se diagnosticada tardiamente. É por este motivo considerada uma urgência médica, pelo fato do tratamento precoce modificar radicalmente a evolução. A presença de sintomas graves de cardiopatia geralmente empobrece o prognóstico e contraindica o transplante de medula óssea (MO). Descrevemos um caso de evolução favorável de amiloidose AL com tratamento medicamentoso. Caso Clínico: paciente de 83 anos, hígido, realizando atividade física intensa, apresentou subitamente piora significativa da capacidade funcional, evoluindo para dispneia aos pequenos esforços e em repouso, em 3 meses de evolução. Foi internado para compensação do quadro em UTI por duas ocasiões, em anasarca, necessitando drogas vasoativas. Realizou ecocardiograma que evidenciou espessura de septo de 19 mm e parede posterior de 17mm, fração de ejeção de 62%, relação E/e'19, e eletrocardiograma com ritmo sinusal e padrão de baixa voltagem no plano frontal. Relação kappa/lambda de 94,6 (valor de referência de 0,26 a 1,65). Foi submetido a biopsia de MO e de gordura abdominal, a imunohistoquímica da MO evidenciou infiltrado intersticialde células plasmocitárias monoclonais para cadeias leves kappae a de gordura abdominal corou com vermelho Congo. Foi submetido aovesquema CyBorD por 1 mês (Ciclofosfamida, Bortezomibe e Dexametasona) Ecocardiograma realizado após 1 ano, evidenciou diminuição significativa da espessura de paredes (septo de 13mm e parede posterior de 12mm). Em 1 ano e 6 meses, o ecocardiograma era normal, com espessura de septo e de parede posterior de 10mm, relação E/e'de 10. COMENTÁRIOS: O diagnóstico de amiloidose de cadeias leves é realizado de forma relativamente simples, com a dosagem da relação kappa/lambda quando esta está muito elevada. Neste caso, a evolução foi muito favorável, com regressão da cardiopatia provavelmente pelo diagnóstico ter sido precoce. O ecocardiograma foi o elemento determinante do diagnóstico e evolução. CONCLUSÃO: A amiloidose de cadeias livres leves tipo Kappa pode ter uma evolução extremamente favorável quando o diagnóstico é feito precocemente, portanto o reconhecimento desta patologia é fundamental para a melhora do prognóstico.

Impact of Treatment Prior to Allogeneic Transplantation of Hematopoietic Stem Cells in Patients with Myelodysplastic Syndrome: Results of the Latin American Bone Marrow Transplant Registry.

It has been suggested that bridging therapy with intensive chemotherapy and/or hypomethylating agents followed by hematopoietic stem cell transplantation (HSCT) can be valuable in the treatment of patients with myelodysplastic syndromes (MDS). However, the influence of this approach on HSCT outcomes remains poorly defined. Therefore, our objective was to investigate the influence of treatment before HSCT in patients with MDS. We retrospectively analyzed data from the Latin American registry of 258 patients from 17 Latin American centers who underwent HSCT from 1988 to 2019. Our data showed that there was pre-HSCT. We detected no significant difference regarding the impact on overall survival of treated and untreated patients before HSCT. Despite these data, the type of previous treatment among treated patients showed a significant difference in overall survival. Treatment with hypomethylating agents together with pre-HSCT chemotherapy seems to result in better survival of the studied population. These data correspond to the first results obtained through cooperative work between various centers in Latin America comparing the different approaches to patients and reflecting their reality and challenges. Therefore, the selection of pretransplant bridge therapy should be analyzed and focus given primarily to those approaches that result in better survival of patients with MDS.

Prevalence of difficult venous access and associated risk factors in highly complex hospitalised patients.

AIMS AND OBJECTIVES: To estimate the prevalence of difficult venous access in complex patients with multimorbidity and to identify associated risk factors. BACKGROUND: In highly complex patients, factors like ageing, the need for frequent use of irritant medication and multiple venous catheterisations to complete treatment could contribute to exhaustion of venous access. DESIGN: A cross-sectional study was conducted. METHODS: 'Highly complex' patients (n = 135) were recruited from March 2013-November 2013. The main study variable was the prevalence of difficult venous access, assessed using one of the following criteria: (1) a history of difficulties obtaining venous access based on more than two attempts to insert an intravenous line and (2) no visible or palpable veins. Other factors potentially associated with the risk of difficult access were also measured (age, gender and chronic illnesses). Univariate analysis was performed for each potential risk factor. Factors with p < 0·2 were then included in multivariable logistic regression analysis. Odds ratios were also calculated. RESULTS: The prevalence of difficult venous access was 59·3%. The univariate logistic regression analysis indicated that gender, a history of vascular access complications and osteoarticular disease were significantly associated with difficult venous access. The multivariable logistic regression showed that only gender was an independent risk factor and the odds ratios was 2·85. CONCLUSIONS: The prevalence of difficult venous access is high in this population. Gender (female) is the only independent risk factor associated with this. Previous history of several attempts at catheter insertion is an important criterion in the assessment of difficult venous access. RELEVANCE TO CLINICAL PRACTICE: The prevalence of difficult venous access in complex patients is 59·3%. Significant risk factors include being female and a history of complications related to vascular access.

Hematological approaches to multiple myeloma: trends from a Brazilian subset of hematologists. A cross-sectional study.

CONTEXT AND OBJECTIVE: For the last nine years, hematologists and oncologists have gathered annually at an educational symposium organized by a Brazilian and an American hospital. During the 2015 Board Review, a survey among the attendees evaluated the differences in management and treatment methods for multiple myeloma (MM). DESIGN AND SETTING: Cross-sectional study during an educational hematology symposium in São Paulo, Brazil. METHODS: Hematologists present at the symposium gave responses to an electronic survey by means of mobile phone. RESULTS: Among the 350 attendees, 217 answered the questionnaire. Most of the participants believed that immunotargeting agents (iTA) might be effective for slowing MM progression in heavily pretreated patients (67%) and that continued exposure to therapy might lead to emergence of resistant clones in patients with MM (76%). Most of the physicians use maintenance therapy after hematopoietic stem cell transplantation (95%) and 45% of them would further restrict it to post-transplantation patients with underlying high-risk disease. The first-line drugs used for transplantation-ineligible patients (TI-MM) were bortezomib-thalidomide-dexamethasone (31%), bortezomib-dexamethasone (28%), lenalidomide-dexamethasone (Rd; 17%) and melphalan-based therapy (10%). Lenalidomide was the drug of choice for post-transplantation maintenance for half of the participants. No significant differences were observed regarding age or length of experience. CONCLUSION: The treatment choices for TI-MM patients were highly heterogenous and the melphalan-based regimen represented only 10% of the first-line options. Use of maintenance therapy after transplantation was a common choice. Some results from the survey were divergent from the evidence in the literature.

Independencia transfusional en un paciente con anemia refractaria con exceso de blastos tipo 2 refractario a 5-azacitidina tratado con deferasirox y agentes estimuladores de colonias / Transfusional independence in a patient with refractory anemia with excess blasts-2 refractory to 5-azacitidine treated with deferasirox and colony stimulating factors

No disponible