Hematology

There is not a lot of evidence on how well nonpharmacologic treatments work to treat sickle cell disease–related pain, and it can be difficult to get people access to these treatments, said C. Patrick Carroll, MD, director of psychiatric services, Sickle Cell Center for Adults, associate professor of psychiatry, Johns Hopkins Medicine.

Toxicities like cytokine release syndrome, along with other factors, limit widespread use of CAR T-cell therapies, said Reona Sakemura, MD, PhD, postdoctoral researcher at the Mayo Clinic.

Three proteasome inhibitors (PIs) are approved for patients with multiple myeloma (MM): Bortezomib, a first-in class PI, fights both newly diagnosed and relapsed/refractory MM (RRMM); carfilzomib, a next-generation PI, treats RRMM as both a monotherapy and in combination; and ixazomib, the first oral PI, treats RRMM in combination. PIs work by preventing the proteasomes in cancerous plasma cells from “recycling” what is essentially garbage protein.

Patients had never been particularly enthusiastic about using opioids to treat their pain related to sickle cell disease, but they are more cautious now, especially as they are often meet with suspicion of addiction, said C. Patrick Carroll, MD, director of psychiatric services, Sickle Cell Center for Adults, associate professor of psychiatry, Johns Hopkins Medicine.

Managing people with sickle cell disease and complex chronic pain is difficult and requires some trial and error, said C. Patrick Carroll, MD, director of psychiatric services, Sickle Cell Center for Adults, associate professor of psychiatry, Johns Hopkins Medicine.

The Institute for Clinical and Economic Review (ICER) published a draft evidence report on crizanlizumab (Adakveo), voxelotor (Oxbryta), and L-glutamine (Endari) for sickle cell disease, as well as a draft scoping document on valoctocogene roxaparvovec, an investigational gene therapy, and emicizumab (Helimbra) for hemophilia.

https://www.pharmacytimes.org/on-demand/managing-vasoocclusive-crisis-in-sickle-cell-disease-a-look-at-the-clinical-and-economic-burden

Results for Truxima, recently launched in the United States, and complement inhibitors were presented at the 61st American Society of Hematology Annual Meeting and Exposition.

Symptom burden, functional ability, and quality of life are necessary considerations when treating hematologic malignancies in older patients.

Reviews of apixaban in active cancer, Medicare costs after CAR T-cell therapy, and the need for financial assistance for novel therapies.

A pair of interviews on investigational therapies whose sponsors reported updates at the 61st American Society of Hematology Annual Meeting and Exposition: UCART19 from Servier and a revamped anti-BCMA therapy ide-cel from bluebird bio.

Coverage from the 61st American Society of Hematology Annual Meeting and Exposition in Orlando, Florida, featured results for allogeneic or "off the shelf" CAR T-cell treatments and bispecific antibodies.

A study identifying the different factors influencing peripheral blood hematopoietic stem cell (PBSC) mobilization and collection in multiple myeloma (MM) and lymphoma cases determined that PBSC mobilization yielded sufficient CD34+ cell counts in both patients with MM and lymphoma, but the failure rates were high.

Sickle cell disease is the most common red blood cell disorder in the United States, having been diagnosed in approximately 100,000 African Americans alone. The lack of oxygen from sickle cell buildup can lead to acute pain crises, joint and organ damage, stroke, and reduced life expectancy.

In the evolving treatment landscape for sickle cell disease, current standards of treatment involve 4 drugs that have been FDA approved, with further innovations and limitations adding to the paradigm of care.

Research has shown that fedratinib favorably impacts health-related quality of life for patients with myelofibrosis across the board, said Ruben Mesa, MD, director of UT Health San Antonio MD Anderson Cancer Center.

Immunotherapy continues to be increasingly used for treating multiple myeloma (MM) with a recent report providing an overview of monoclonal antibodies (mAbs) currently approved for treating MM, as well as, compounds that are under investigation.

As a result of the opioid epidemic, there have been instances where it has been difficult to treat patients with pain related to their sickle cell disease because of new policies in place intended to curb addiction and overdoses, said C. Patrick Carroll, MD, director of psychiatric services, Sickle Cell Center for Adults, associate professor of psychiatry, Johns Hopkins Medicine.

In research presented at the 61st American Society of Hematology Annual Meeting and Exposition, Abby Statler, PhD, MPH, MA, research associate, Cleveland Clinic, and her colleagues found that African Americans with acute myeloid leukemia (AML) may be excluded from clinical trials due to renal dysfunction, despite it having no impact on AML outcomes.

An executive with Jazz Pharmaceuticals said the ability to extend survival time in patients with secondary AML, and potentially offer them improved odds for transplant is an advance over traditional chemotherapy.

The terminology for smoldering myeloma has been around for decades, but more discussion over what it means and who it really applies to is needed, said C. Ola Landgren, MD, PhD, professor of medicine and chief of the Myeloma Service at Memorial Sloan Kettering Cancer.

The company presented updated phase 1 results for a revamped version of bb2121 that point to sustained responses for patients with relapsed/refractory multiple myeloma.

As more patients with cancer survive, the risk of cardiac complications due to the effects of therapies has become a concern to oncologists and cardiologists alike.