Belantamab mafodotin, pomalidomide and dexamethasone in refractory multiple myeloma: a phase 1/2 trial.

Due to evolving treatment standards for newly diagnosed multiple myeloma, many patients will be triple-class exposed after initial relapses and have poor survival. Novel therapies and combinations are therefore required to improve outcomes. B cell maturation antigen (BCMA)-targeted biologics have emerged as an important new area of therapeutics for relapsed multiple myeloma. The two-part ALGONQUIN trial evaluated various doses and schedules of the anti-BCMA antibody-drug conjugate belantamab mafodotin plus pomalidomide and dexamethasone for patients who are lenalidomide refractory and proteosome inhibitor exposed. The primary endpoints, including evaluating dose-limiting toxicities, establishing the recommended Part 2 dose (RP2D) and overall response rate for patients treated at the RP2D, were met. Secondary efficacy endpoints included progression-free survival and overall survival. Patients treated on study (N = 87) had a median of three previous regimens and 55.2% were triple-class refractory. At the RP2D the most common adverse events were decrease in best-corrected visual acuity (71.1%), keratopathy (65.8%), fatigue (57.9%), infection (47.4%; 7.9% grade ≥3), neutropenia (39.5%) and thrombocytopenia (39.5%). For RP2D patients (n = 38), the overall response rate was 85.3%, ≥very good partial response 75.7% and estimated two-year progression-free survival 52.8% (95% confidence interval, 33.9% to 82.4%), at a median follow-up of 13.9 months. The RP2D schedule was associated with manageable antibody-drug conjugate-associated corneal adverse events and improved tolerability without compromising efficacy. Belantamab mafodotin plus pomalidomide and dexamethasone induced durable responses with promising overall survival in relapsed multiple myeloma, the results of which are yet to be confirmed in the phase 3 DREAMM-8 study. ClinicalTrials.gov Identifier: NCT03715478 .

First Line Treatment of Newly Diagnosed Transplant Ineligible Multiple Myeloma: Recommendations from the Canadian Myeloma Research Group Consensus Guideline Consortium.

Although the availability of effective novel treatments has positively impacted the quality of life and survival of newly diagnosed multiple myeloma (MM) patients, benefits in the transplant ineligible MM population may be limited by functional/frailty status. The Canadian Myeloma Research Group Consensus Guideline Consortium proposes consensus recommendations for the first-line treatment of transplant ineligible MM. To address the needs of physicians and people diagnosed with MM, this document further focuses on eligibility for transplant, frailty assessment, management of adverse events, assessment of treatment response, and monitoring for disease relapse. The Canadian Myeloma Research Group Consensus Guideline Consortium will periodically review the recommendations herein and update as necessary.

Twelve tips for using ePosters as an active learning strategy.

ePosters (electronic Posters), a modification of traditional paper-based posters have gained popularity in medical education conferences since 2011. ePoster in educational settings differs from the traditional poster in that it allows the ePoster creator to focus on the learning process rather than reporting scientific outcomes. However, there is limited literature comparing ePosters to traditional paper-based posters and their impact on the student learning experience. ePosters as an assessment tool are well suited for online learning. This article presents twelve tips for using ePosters as an active learning strategy in classrooms and describes how to incorporate ePosters as a formative and summative assessment tool in health professions education, at all levels.

Hereditary hemochromatosis and JAK2-positive polycythemia vera.

A 59-year-old man was diagnosed with JAK2-positive polycythemia vera. Subsequently, further laboratory testing revealed elevated ferritin and iron saturation. Genetic testing for HFE gene mutation screen revealed that the patient was positive for heterozygous C282Y mutation. The patient was ultimately diagnosed with both polycythemia vera and hereditary hemochromatosis.

Refractory acquired thrombotic thrombocytopenic purpura in a patient with sickle cell trait successfully treated with caplacizumab.

Methods: We report a case of a 20-year-old Nigerian male who presented with acquired thrombotic thrombocytopenic purpura (aTTP) and sickle cell trait. The coexistence of published cases of TTP and sickle cell hemoglobinopathies is rare.Results: Despite the initial treatment with plasma exchange and glucocorticoids, our patient relapsed and also required caplacizumab which resulted in successful remission.Discussion: We conclude by reviewing the cases of TTP in patients with sickle cell hemoglobinopathies and review how vaso-occlusive crises with multiorgan injury can mimic TTP.Conclusion: Ours is the first published case of aTTP with confirmed ADAMTS13 autoantibodies in a patient with a sickle cell hemoglobinopathy and contributes to the literature on the successful use of caplacizumab in clinical practice.

Correction to: The International/Canadian Hereditary Angioedema Guideline.

[This corrects the article DOI: 10.1186/s13223-019-0376-8.].

The International/Canadian Hereditary Angioedema Guideline.

This is an update to the 2014 Canadian Hereditary Angioedema Guideline with an expanded scope to include the management of hereditary angioedema (HAE) patients worldwide. It is a collaboration of Canadian and international HAE experts and patient groups led by the Canadian Hereditary Angioedema Network. The objective of this guideline is to provide evidence-based recommendations, using the GRADE system, for the management of patients with HAE. This includes the treatment of attacks, short-term prophylaxis, long-term prophylaxis, and recommendations for self-administration, individualized therapy, quality of life, and comprehensive care. New to the 2019 version of this guideline are sections covering the diagnosis and recommended therapies for acute treatment in HAE patients with normal C1-INH, as well as sections on pregnant and paediatric patients, patient associations and an HAE registry. Hereditary angioedema results in random and often unpredictable attacks of painful swelling typically affecting the extremities, bowel mucosa, genitals, face and upper airway. Attacks are associated with significant functional impairment, decreased health-related quality of life, and mortality in the case of laryngeal attacks. Caring for patients with HAE can be challenging due to the complexity of this disease. The care of patients with HAE in Canada, as in many countries, continues to be neither optimal nor uniform. It lags behind some other countries where there are more organized models for HAE management, and greater availability of additional licensed therapeutic options. It is anticipated that providing this guideline to caregivers, policy makers, patients, and advocates will not only optimize the management of HAE, but also promote the importance of individualized care. The primary target users of this guideline are healthcare providers who are managing patients with HAE. Other healthcare providers who may use this guideline are emergency and intensive care physicians, primary care physicians, gastroenterologists, dentists, otolaryngologists, paediatricians, and gynaecologists who will encounter patients with HAE and need to be aware of this condition. Hospital administrators, insurers and policy makers may also find this guideline helpful.

Flow cytometry and immune thrombocytopenic purpura.

Although Immune thrombocytopenic purpura is a common disorder that family physicians, internists and hematologists face in their everyday practice, its diagnosis rests only on "exclusion" and its therapy is based on algorithms where "trial and error" is the rule. Flow cytometry, if simplified and standardized, could provide a quicker and better diagnostic accuracy. Studies of the lymphocyte subset using flow cytometry and more elaborate immune studies are paving the way for a better understanding of the disease and in identification of prognostic markers. Such studies may even help stratify the first-line therapy responder and assist in the use of the arsenal of immune suppressive therapy with better precision.