January 2021

Each month the members of the Scientific and Education Committee of the International Society of Hematology (ISH) write this newsletter for our subscribers featuring relevant news in Global Hematology.

In this issue we highlight interesting research on Classical Hematology presented at the latest American Society of Hematology Meeting and Exposition.


COVID-19 message

Our hearts go out to all patients and families that have been affected by COVID19, a disease which knows no borders. Our most sincere gratitude to all healthcare personnel involved in any way in the management and care of the COVID19 pandemic for their tireless efforts. The recent success of lightning-fast vaccine development is heartening, and we hope widespread access to effective vaccination is available worldwide very soon.

Follow the latest updates of the World Health Organization here. Visit our website for special written communications on COVID19 and hematology developed by expert clinicians from around the world.

Classical Hematology Highlights from ASH 20

Tranexamic acid fails hematology patients in randomized trial

The A-TREAT trial by Terry B Gernsheimer et al. was designed to assess the efficacy of tranexamic acid (TXA) in patients with hematologic malignancy or who undergoing HSCT with anticipated thrombocytopenia. Intravenous or oral TXA was administered every 8 hours and continued with a platelet count lower than 30,000/µL occurred. Investigators enrolled 330 patients. The primary endpoint, the proportion of patients with WHO grade 2 bleeding or higher during the 30 days after study drug activation, was similar between groups (adjusted odds ratio 0.86 [95% CI 0.52-1-38]).

Mycophenolate add on beats standard steroid treatment in newly diagnosed ITP

Charlotte Bradury, et al. presented the results of the Flight trial, a randomized controlled trial assigning 129 oatints with newly diagnosed ITP in need of treatment to corticosteroids versus steroids plus mycophenolate for 6 months. The primary outcome was time to treatment failure defined as thrombocytopenia <30,000/uL and clinical need of second line treatment, Fewer treatment failures occurred in pateints randomized to MMF (22% vs. 44%; p<0.01) with similar rates of adverse events. Importantly, patient reported outcomes were worse for those treated in the MMF arm, specifically in the physical role, physical function and fatigue, emphasizing the importance of these measures in clinical trials.

Hope for patients with hemophilia B

Results of gene therapy with etranacogene dezaparvovec, an adeno-associated viral vector containing a variant huma factor IX gene was studied in the phase 3 HOPE-B study by Steven Pipe et al. Fifty four of the 75 patients screened were dosed, regardless of the presence of viral neutralizing antibodies. Most patients had severe hemophilia and outcomes after 26 weeks were reported. Following treatment FIX increased to a mean of 37.2% and was maintained after follow-up, 96.3% of patients discontinued prophylaxis and 72.2% reported 0 bleeds. No deaths occurred or treatment related severe adverse events were documented.

Ruxolitinib also successful in chronic GVHD

Ruxolitinib 10 mg BID for 6 cycles was randomly compared to best available therapy in patients with moderate or severe chronic graft versus host disease in an industry-sponsored phase 3 trial by Robert Zeiser, et al. A total of 329 adults were included, with the study meeting its primary endpoint; the overall response rate was 50% vs 26% OR 2.99; p<0.01, while the CR rate was low albeit higher in the Rux arm (7 vs 3%) with similar rates of adverse events.

Functional cure of thalassemia and sickle cell disease with CRISPR-Cas-9?

CLIMB-THAL-111 and CLIMB SCD-121 are first-in-human studies using CRISPR Cas 9 technology to edit the BCL11A transcription factor into avoiding suppression of fetal hemoglobin (HbF) production. The study presented by Haydar Frangoul, et al. reported the results of 5 patients with transfusion-dependent beta-thalassemia and 2 patients with sickle cell disease (SCD). Patients underwent peripheral blood CD34+ cell harvest and BCL11A editing named “CTX001” and were infused after busulfan myeloablative conditioning. All patients had an increase in total Hb and HbF over time with a reduction in transfusion and vaso-occlusive crises after engraftment. Remarkably 4 cases of similar to haemophagocytic lymphohistiocytosis were observed with no fatalities.

Words from the ISH Chair of Council

Dear friends and members of ISH, in this newsletter we show provocative and important studies in the field of so called “benign hematology” now renamed by some as “classical hematology”. For the last couple of malignant hematology has had a louder voice dominating the field. Remarkably, the American Society of Clinical Oncology has more than 40,000 members while ASH has around 18,000… this turns to mind something a heard a colleague during an ISH meeting: “Money is where cancer is”.  Benign blood diseases such as iron deficiency, thrombosis, thalassemia and hemoglobinopathies are highly prevalent throughout the world and affect the lives of millions. Quoting Professor Alice Ma from the University of North Carolina in the USA “we see clotting disorders, bleeding disorders, anemia and thrombocytopenia. We figure things out and patients mostly don’t die”. Last year we learned that patients with COVID-19 can also suffer sometimes not so “benign” hematologic complications, emphasizing the importance of keeping up to date for those of us who treat not only blood cancer but are also “classy” hematologists.

Keep safe,
David Gómez-Almaguer MD
ISH Chair of Council

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Hematology is the official journal of the ISH published online by Taylor Francis. 

It is an international open access journal publishing original research and reviews in the field of general hematology, including oncology, pathology, biology, clinical research and epidemiology.

Hematology aims to bridge the interests and practice of both those carrying out laboratory work and those w hose main inclination is patient care.

ISH members have a reduced author publishing fee to Hematology.


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