November 2020
Volume 6, Issue 13
This issue dives into the FDA’s expedited review pathways, the progress and setbacks in gene therapy for hemophilia, and more.
Table of Contents
November 2020 Issue
Reader Responses: Is hypogammaglobulinemia alone an indication for treatment for CLL?
Here’s how readers responded to a You Make the Call question about whether hypogammaglobulinemia alone is an indication for treatment of CLL.
Disclaimer: ASH does...
In Oral Maintenance Therapy in AML, Do All Roads Lead to the Epigenome?
Maintenance therapy to reduce relapse of acute myeloid leukemia (AML) and prolong overall survival (OS) has been explored since the 1960s.1 Chemotherapeutic and immunologic...
Editor’s Corner: Two Ears, One Mouth
I consider myself blessed to come from a medical family. My parents were physicians who were sweethearts in medical school in Egypt. They had...
November 2020 Issue
Is Gene Editing the Answer to Eradicating Malaria in Africa?
In 2018, there were 228 million cases of malaria worldwide. The overwhelming majority of those cases – more than 90% – were in sub-Saharan...
Who’s Watching the Watchdog?
With most drugs now approved through one of the FDA’s expedited-review pathways, is the agency approving drugs too quickly?
In recent years, the FDA has...
Gene Therapy for Hemophilia: Progress and Setbacks
As the FDA delayed the approval of the world’s first gene therapy for hemophilia A, companies forged ahead with phase III trials to gather...
Pulling Back the Curtain: Tait Shanafelt, MD
In this edition, Tait Shanafelt, MD, talks about his journey to becoming an expert in physician wellness, with a stop as a house-painting business...
November 2020 Issue
You Make the Call: Is hypogammaglobulinemia alone an indication for treatment for CLL?
This month, Ariela Noy, MD, discusses whether hypogammaglobulinemia alone is an indication for treatment of CLL.
And don't forget to check out next month's clinical...
Meet the Munich Leukemia Laboratory: Using Modern Technology to Find Clues to Hematologic Mysteries
At its core, the Munich Leukemia Laboratory (MLL) is like any other diagnostic lab.
“We receive samples and send reports,” one of its three cofounders,...
November 2020 Issue
ASH Announces Executive Committee Election Results, New Clinical Practice Guidelines, and more
The Votes Are In! ASH Announces Executive Committee Election Results
The American Society of Hematology (ASH) has announced the election of four new members to...
How Common Is Venous Thromboembolism in Patients Hospitalized With COVID-19?
Reports have suggested that venous thromboembolism (VTE) is a frequent complication of COVID-19 infection, and patients are at an increased thrombotic risk even with...
Addition of Elotuzumab Prolongs Survival in Relapsed or Refractory Myeloma
Treatment with elotuzumab plus lenalidomide and dexamethasone (ERd) was associated with significant and clinically meaningful reductions in mortality risk, compared with lenalidomide and dexamethasone...
Fresh Frozen Plasma May Help Maintain Successful Pregnancy in Women With Congenital TTP
Prophylactic fresh frozen plasma (FFP) infusions may be necessary to replenish ADAMTS13 activity and maintain a successful pregnancy in expectant mothers with congenital thrombotic...
Smokeless Tobacco Linked to Subtherapeutic INR in Warfarin-Treated Patient
Failure to achieve a therapeutic international normalized ratio (INR) with warfarin was associated with chronic exposure to vitamin K-rich smokeless chewing tobacco in a...
Evaluating the TP53 State and Its Implications for Myelodysplastic Syndromes Outcomes
In a new study published in Nature Medicine, researchers evaluated the effect of the TP53 allelic state on the clinical presentation and outcomes of...
Large-Scale NIH-Sponsored COVID-19 Vaccine Trials Underway
Four large-scale, phase III clinical trials to evaluate vaccines against COVID-19 codeveloped by the National Institutes of Health (NIH) and pharmaceutical companies have launched...
Sequential Pembrolizumab and Chemotherapy Results in Excellent Responses in Newly Diagnosed Classical Hodgkin Lymphoma
A brief course of pembrolizumab monotherapy followed by AVD chemotherapy (doxorubicin, vinblastine, and dacarbazine) was associated with reductions in metabolic tumor volume and prolonged...
Crizotinib Receives Priority Review for Pediatric ALCL
The FDA has granted priority review to a supplemental new drug application for crizotinib to treat pediatric patients with relapsed/refractory systemic anaplastic large cell...
FDA Approves Mepolizumab for Hypereosinophilic Syndrome
The FDA has approved mepolizumab to treat adults and children aged 12 and older who have had hypereosinophilic syndrome (HES) for 6 months or...
FDA Grants Priority Review to Melflufen for Myeloma
The FDA granted priority review to a new drug application for INN melphalan flufenamide, or melflufen, in combination with dexamethasone for the treatment of...
PF4-Dependent P-Selectin Assay Highly Accurate in Diagnosing Heparin-Induced Thrombocytopenia
A study published in Blood established the diagnostic accuracy of a PF4-dependent P-selectin expression assay (PEA) for heparin-induced thrombocytopenia (HIT), with a sensitivity similar...
Remembering Patti Massicotte, Nobel Prizes Awarded for Discovery of Hepatitis C and CRISPR, and...
Remembering Patti Massicotte (1955 – 2020)
Mary Patricia “Patti” Massicotte, MD, FRCP, died on October 1, 2020 from cancer.Dr. Massicotte was Professor and Director of Clinical...
Azacitidine Tablets Approved for AML Following Chemotherapy
The FDA has approved azacitidine tablets, formerly known as CC-486, for the continued treatment of adults with acute myeloid leukemia (AML) who achieved first...
Idecabtagene Vicleucel Granted Priority Review for Myeloma
The FDA has granted priority review to the biologics license application of idecabtagene vicleucel (ide-cel), for the treatment of adult patients with MM who...
NASEM Releases Sickle Cell Disease Report
The National Academies of Sciences, Engineering, and Medicine (NASEM) recently released a report titled “Addressing Sickle Cell Disease: A Strategic Plan and Blueprint for...
