Hematology

Michael Thompson, MD, PhD, FASCO, Aurora Advanced Healthcare, outlines novel therapies being used in the treatment of hematologic malignancies and identified biomarkers for deciding on a treatment approach.

A new approach using gene editing technology could allow chimeric antigen receptor (CAR) T cells to target CD33 in patients with acute myeloid leukemia (AML) but prevent the cells from attacking healthy stem cells, too.

A stem cell mutation that leads to bacterial translocation may help identify patients at risk of developing leukemia.

The interim analysis of the phase 3 A.R.R.O.W. trial found that once-weekly carfilzomib at 70 mg/m2 had superior progression-free survival and overall response rates compared with twice-weekly carfilzomib at 27 mg/m2.

An experimental drug for patients with acute myeloid leukemia (AML) with mutations in the gene encoding isocitrate dehydrogenase 1 (IDH1) was found to be safe with durable remissions.

Two abstracts presented at the International Society of Pharmacoeconomic and Outcomes Research Annual International Meeting highlighted patient experiences in blood cancers. One highlighted the worries of patients with acute myeloid leukemia, and the other analyzed economic burdens and resource utilization for patients with non-Hodgkin lymphoma.

Two study abstracts presented at the International Society of Pharmacoeconomics and Outcomes Research Annual International Meeting look at the cost burden of diffuse large B-cell lymphoma (DLBCL). One analyzed the lifetime cost of relapsed/refractory DLBCL, including third-line and subsequent treatments, and the other examined how being diagnosed with DBLCL can increase the cost burden of chronic conditions among Medicare beneficiaries.

A new study analyzed the burden of multiple myeloma around the world, as well as access to effective treatments. From 1990 to 2016, incident cases and deaths from the disease have grown substantially.

Analysis of patient-reported outcomes found patients with relapsed/refractory B-cell acute lymphoblastic leukemia who were treated with inotuzumab ozogamicin reported better quality of life, functioning, and symptom scores compared with patients receiving standard-of-care chemotherapies.

A phase III randomized trial, with results being presented in June at the American Society of Clinical Oncology Annual Meeting, found the addition of nelarabine to standard chemotherapy regimen in patients with T-cell malignancies can improve disease-free survival rates.

The FDA has approved Pfizer’s epoetin alfa-epbx (Retacrit), a biosimilar to epoetin alfa (Epogen), marking the first biosimilar approval of 2018 and the first biosimilar erythropoiesis-stimulating agent approved in the United States.

A new case study found that an acute myeloid leukemia patient has remained cancer free for 9 months following treatment with the chimeric antigen receptor (CAR) T-cell treatment, CYAD-01, and a bone marrow transplant.

The FDA has approved daratumumab in combination with bortezomib, melphalan, and prednisone to treat patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant.

This week, the top managed care stories included a report that insurance coverage gains are reversing; a deal to lower the cost of Praluent for Express Scripts members; findings that synthetic opioids are playing a bigger role in overdose deaths.

Tisagenlecleucel, sold as Kymriah, has gained its second indication following the FDA's approval of the chimeric antigen receptor T-cell therapy for the treatment of adult patients with relapsed or refractory large B-cell lymphoma, the most common form of non-Hodgkin lymphoma.

The chimeric antigen receptor T-cell therapy tisagenlecleucel has been approved for a second type of blood cancer; the National Institutes of Health has started recruiting individuals for a database that will include data on more than 1 million people; Kansas’ request to impose a 3-year lifetime limit on Medicaid benefits is testing just how open the Trump administration is to allowing states flexibility.

Chimeric antigen receptor (CAR) T-cell treatments are still new enough that there are still unknown regarding long-term side effects, which is something patients need to understand before they undergo treatment, said Shannon L. Maude, MD, PhD, of The Children's Hospital of Philadelphia.

Researchers have identified a new gene that may predispose individuals to develop acute lymphoblastic leukemia (ALL). In addition, variants in the gene can influence patient response to treatment.

At the Academy of Managed Care Pharmacy’s Managed Care & Specialty Pharmacy Annual Meeting, experts addressed the cost of treating patients with hemophilia and the importance of care coordination to ensure a more holistic approach toward caring for these patients.

A new blood test could negate the need for bone biopsies to diagnose a variety of cancers, including multiple myeloma. The test uses a small, low-cost plastic chip that delivers the same diagnostic information of a biopsy through a simple blood draw.

A novel drug that targets MDMX and MDM2, which inhibit a protein that suppresses tumors when they are overexpressed, has tripled the median survival rate in an animal model of human acute myeloid leukemia, according to new research.

Thomas LeBlanc, MD, of the Duke Cancer Institute, addresses the importance of adding a palliative care specialist to the cancer care team.

Since patients who receive CAR T-cell therapy experience unique adverse events, there will need to be education for providers who care for these patients, explained Stephen Schuster, MD, of the Perelman School of Medicine.

The identification of 40 genes involved with the development of multiple myeloma could lead to the development of more personalized treatments.

While increased risk of neurocognitive issues, such as long-term problems with attention, is common in survivors of childhood acute lymphoblastic leukemia (ALL), the risk may actually begin before treatment, according to a study published in JAMA Oncology.

Researchers have identified genetic subtypes of diffuse large B-cell lymphoma that revise the molecular classification of the disease and could provide insight into why some patients respond to treatment and others don’t.

Sancilio Pharmaceuticals announced that it has received the European Medicines Agency’s Orphan Designation for its SC411 (which it plans to market as Altemia), a proposed treatment for sickle cell disease in pediatric patients, in the European Union. The FDA granted the proposed drug a similar designation—the Rare Pediatric Disease designation—in 2017.

While there has been progress with using chimeric antigen receptor (CAR) T cells to treat multiple myeloma, these treatments aren’t ready for prime time, said Nina Shah, MD, associate professor, University of California, San Francisco, School of Medicine.

Researchers have been trying to find a way to screen populations at high risk of developing a precursor condition to multiple myeloma in order to intervene early and reduce disease prevalence and mortality.

Despite price tags well over $350,000 for treatment, tisagenlecleucel, approved for children with B-cell acute lymphoblastic leukemia, and axicabtagene ciloleucel, approved for adults with certain B-cell subtypes of non-Hodgkin lymphoma, are considered cost effective, according to a report from the Institute for Clinical and Economic Review.