NEWS
Zanubrutinib Approved for Waldenström Macroglobulinemia
The U . S . Food and Drug Administration ( FDA ) has approved zanubrutinib for the treatment of adult patients with Waldenström Macroglobulinemia ( WM ).
This approval is based on efficacy data from the ASPEN trial comparing zanubrutinib with ibrutinib in adult patients with MYD88 L265P – mutated WM . In Cohort 1 , 201 patients were randomized to receive either zanubrutinib 160 mg twice daily or ibrutinib 420 mg once daily until disease progression or unacceptable toxicity . Patients in Cohort 2 had MYD88 wildtype ( n = 26 ) or MYD88 mutation unknown WM ( n = 2 ) and were treated with zanubrutinib 160 mg twice daily .
Outcomes used to support approval were partial response or better as assessed by an independent review committee based on standard consensus response criteria from the International Workshop on Waldenström ’ s Macroglobulinemia-6 , as well as duration of response ( DOR ). Across all patients receiving zanubrutinib , the response rate was 77.5 %. At 12 months , the event-free DOR for patients treated with zanubrutinib was 94.4 %. In Cohort 2 , response as assessed by independent review committee was seen in 50 % of response evaluable patients ( 13 out of 26 ).
Adverse events reported in ≥20 % of patients treated with zanubrutinib were neutropenia , upper respiratory tract infection , thrombocytopenia , rash , hemorrhage , musculoskeletal pain , anemia , bruising , diarrhea , pneumonia , and cough .
The recommended oral dosage of zanubrutinib is 160 mg twice daily or 320 mg once daily .
Source : FDA press release , August 31 , 2021 .
Nearly Half of Transgender Patients Report Discrimination
A recent report by the Center for American Progress ( CAP ) found that nearly 50 % of transgender people in the U . S . have experienced mistreatment by a medical provider , including refusal of care and verbal or physical abuse .
In June 2020 , CAP surveyed more than 1,500 LGBTQ + individuals . Among these respondents , nearly one in two transgender individuals reported being mistreated by a health-care provider , and the number was higher among transgender people of color at 68 %.
About 28 % of these respondents reported postponing or avoiding receiving necessary medical care for fear of experiencing discrimination , and 48 % said their health insurance company had denied them genderaffirming care . ( Findings from this study are illustrated in Data Stream , “ The Transgender Gap ,” on page 53 .)
The “ onus should not be on individuals ” to find access to quality and nondiscriminatory medical care , said Sharita Gruberg , vice president of CAP ’ s LGBTQ Research and Communications Project and one of the report ’ s authors . “ It really should be on [ health-care ] institutions to do the right thing , and the resources and guidance is out there .”
See Editor ’ s Corner on page 6 for the patient and provider perspective on achieving health equity for transgender patients .
Source : The Hill , August 18 , 2021 .
New Plan to Cover Uninsured in Non- Medicaid Expansion States
As part of a $ 3.5 trillion package proposal , Congressional Democrats are crafting a new federal program to provide health insurance coverage to more than 2 million people living in the 12 U . S . states that declined to expand Medicaid under the Affordable Care Act ( ACA ).
First , the plan would subsidize private insurance on the ACA marketplace for this population until 2025 or 2026 , when a new federal coverage program would kick in . As the program could represent a step toward a larger , government-run public health insurance option , it may draw criticism from the health care industry .
“ We have significant concerns with any proposal that would establish a new Federal Medicaid lookalike program to fill the coverage gap ,” the Federation of American Hospitals wrote to Congress in August . “ The formation and implementation of a new federal program or a Medicaid public option would be complex and costly , burdened by bureaucracy and rulemaking that would unnecessarily delay access to care for millions .” The group suggested permanently subsidizing private ACA marketplace insurance instead .
Source : The Hill , August 25 , 2021 .
Gavo-Cel Granted Orphan Drug Designation for Cholangiocarcinoma
The FDA has granted orphan drug designation to T-cell therapy gavocabtagene autoleucel ( gavo-cel ) for the treatment of cholangiocarcinoma .
TCR 2 Therapeutics , the manufacturer of gavo-cel , is developing cancer treatments using T-cell receptor ( TCR ) Fusion Construct T cells ( TRuC ® -T cells ) which recognize and kill cancer cells by harnessing signaling from the entire TCR independent of human leukocyte antigens . TC-110 , another therapy developed by TCR 2 Therapeutics , is currently being evaluated for the treatment of adult patients with CD19-positive acute lymphocytic leukemia and aggressive or indolent non-Hodgkin lymphoma in a phase I / II clinical trial .
A phase I / II study of gavo-cel in patients with refractory mesothelin-expressing solid tumors , including malignant mesothelioma , ovarian cancer , and cholangiocarcinoma , is ongoing .
Source : TCR 2 Therapeutics press release , September 2 , 2021 .
New U . S . Department of Health and Human Services Office Will Address Climate Change as a Public Health Issue
The U . S . Department of Health and Human Services ( HHS ) has launched the new Office of Climate Change and Health Equity to address the public health risks brought on by climate change , based on a significant body of research showing that certain populations are disproportionately affected . These include individuals with chronic illnesses or mobility challenges , older people , low-income individuals , Black and indigenous populations and other people of color , people in certain occupational groups , and women and girls .
“ Its mission is to protect the health of people experiencing a disproportionate share of climate impacts and health inequities from wildfires to drought , to hurricanes to floods ,” said Secretary of Health and Human Services Xavier Becerra .
One of the office ’ s first tasks will be taking account of greenhouse gas emissions from various parts of the health care sector , according to U . S . officials . Other initiatives could include making hospital participation in Medicare and Medicaid contingent on meeting certain environmental goals as well as performance targets for physical plant adaptations , waste management , supply chains , and sustainable energy use .
While the American Academy of Pediatrics , the Asthma and Allergy Foundation of America , and the American Psychological Association support the new office ’ s initiatives , other sectors of the medical industry might resist . For example , hospitals are concerned about complying with new environmental regulations while struggling with the demands of the COVID-19 pandemic .
The Occupational Safety and Health Administration adopted temporary standards to improve workplace safety during the pandemic , including running air purification systems at a higher level in hospitals , resulting in more carbon emissions and increased energy use , said Michelle Hood , executive vice president and chief operating officer at the American Hospital Association . “ Financial penalties would not address the root cause of these issues ,” Ms . Hood added .
Source : The Wall Street Journal , August 30 , 2021 .
26 ASH Clinical News October 2021