[Multiple Myeloma Treatment Guidelines by the Portuguese Group of Multiple Myeloma]. / Recomendações do Grupo Português do Mieloma Múltiplo para Tratamento do Mieloma Múltiplo.

The treatment of multiple myeloma has profoundly changed with the introduction of several innovative therapies. The optimization of therapeutic sequencing through the combined use of the various drugs developed in recent years and the attention given to the characteristics of patients have allowed the reduction of toxicities and increased survival and quality of life of patients with multiple myeloma. These treatment recommendations from the Portuguese Multiple Myeloma Group offer guidance for first-line treatment and progression/relapse situations. These recommendations are given highlighting the data that justify each choice and referring to the respective levels of evidence that support these options. Whenever possible, the respective national regulatory framework is presented. These recommendations constitute an advance towards the best treatment of multiple myeloma in Portugal.

O tratamento do mieloma múltiplo tem sido amplamente alterado com introdução de várias terapêuticas inovadoras. A otimização da sequenciação terapêutica através do uso combinado dos vários fármacos desenvolvidos nos últimos anos e a atenção dada às características dos doentes têm permitido diminuir toxicidades e aumentar a sobrevivência dos doentes, bem como aumentar a sua qualidade de vida. As presentes recomendações terapêuticas do Grupo Português do Mieloma Múltiplo oferecem orientações para o tratamento de primeira linha e progressão/recaída. As recomendações são fundamentadas evidenciando os dados que justificam cada escolha e referindo os respetivos níveis de evidência que suportam essas opções. Sempre que possível é apresentado o respetivo enquadramento regulamentar nacional. Estas recomendações constituem um avanço para o melhor tratamento do mieloma múltiplo em Portugal.

Use of Brentuximab Vedotin in a Non-HIV Patient with Primary Effusion Lymphoma.

Primary effusion lymphoma is a rare and aggressive large B-cell lymphoma presenting as malignant effusion with poor prognosis. Although it is more prevalent among HIV patients, it has also been described in non-HIV immunocompromised individuals. Given its rarity, there are no large randomized trials regarding the best therapeutic option. The choice of therapy is based on retrospective studies, case reports and preclinical data. We present the case of a non-HIV patient with relapsed disease after treatment with CHOP who was then successfully treated with brentuximab vedotin, achieving complete remission. LEARNING POINTS: Primary effusion lymphoma is a rare entity in non-HIV patients, so there are no clinical trials regarding therapeutic options or management decisions.Targeted therapy with brentuximab vedotin has been used in other CD30 positive malignancies and in HIV-related primary effusion lymphoma with good outcomes.We present the case of a non-HIV patient with primary effusion lymphoma who was successfully treated with brentuximab vedotin, which highlights the potential role of a new therapeutic approach in this subgroup of patients.

Multiple myeloma in elderly patients-a Portuguese multicentric real-life study.

Patients older than 75 years old with multiple myeloma (MM) have shorter survival and are usually treated differently from what features in clinical trials. In this study, the authors characterized the Portuguese population of MM patients above 75 years old, treated between 2009 and 2016. We compared the outcomes obtained with bortezomib-based protocols (BBP), thalidomide-based protocols (TBP), and chemotherapy (CT) using univariate and multivariate controlling for age, performance status, International Staging System score, renal impairment, and number of comorbidities. We retrieved data from 386 patients, treated in 12 hospitals. Three hundred thirty-one cases were analyzed: 119 patients treated with BBP, 65 with TBP, 147 with CT. Median age was 79 years; CT-treated patients were older, had a worse performance status, and have more comorbidities. The median follow-up was 25 months. The 2-year OS was 58% and the median OS was 29.5 months. Patients treated with BBP had more frequently very good partial response (VGPR) or better response, and the subgroup of more fit patients had a significantly longer progression-free survival (PFS) and OS. The most frequently grade 3-4 toxicities were hematologic, infectious, and neurologic and were significantly lower in TBP and CT groups vs BBP. The most common second line was CT, followed by lenalidomide. Patients treated with lenalidomide had a higher probability of VGPR or better and a superior 1-year PFS. Despite the limitations of a retrospective study, our cohort represents the reality of older patients with MM in a western country. The hazard of death or progression was higher for old, fit patients treated, in first line, with CT and with TBP compared with that of BBP.

Endomyocardial Fibrosis With End-Stage Heart Failure as a Consequence of a Myeloproliferative Neoplasm With Hypereosinophilia.

Hypereosinophilic syndrome is characterized by an overproduction of eosinophils that infiltrate and damage multiple organs. Cardiac dysfunction occurs frequently and is a main cause of morbidity and mortality. We describe the case of a middle-aged man diagnosed with a myeloproliferative neoplasm associated with hypereosinophilia and treated with imatinib. He was diagnosed with cardiac involvement by hypereosinophilic syndrome at a late stage, with an established restrictive cardiomyopathy. Because of end-stage heart failure, he successfully received a heart transplant. This disease might not be considered a contraindication for heart transplantation.

BCR-ABL V280G Mutation, Potential Role in Imatinib Resistance: First Case Report.

INTRODUCTION: The identification of BCR-ABL expression as the defining leukemogenic event in chronic myeloid leukemia (CML) and the introduction of BCR-ABL tyrosine kinase inhibitors in 2001 have revolutionized disease management, leading to a reduction in mortality rates and accordingly an increase in the estimated prevalence of CML. CASE REPORT: Based on medical records and clinical follow-up, the authors present the case of a Philadelphia chromosome-positive CML patient who developed resistance to imatinib. Quantitative reverse transcription-polymerase chain reaction testing revealed a V280G BCR-ABL mutation. DISCUSSION AND CONCLUSIONS: This is the first report describing a new BCR-ABL kinase domain mutation-V280G-that might be associated with resistance to imatinib. Approximately 15% to 30% of patients treated with imatinib discontinue treatment due to resistance or intolerance. More than 90 BCR-ABL mutations were detected so far, conferring variable degrees of drug resistance, with consequent clinical, therapeutic, and prognostic impact.