Preliminary data suggest UCART19 is safe, effective

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Reuben Benjamin, MD, PhD
Photo by Jen Smith
Preliminary data on UCART19—the first off-the-shelf, anti-CD19, allogeneic chimeric antigen receptor (CAR) T-cell therapy—suggest it can produce complete responses (CRs) and minimal residual disease (MRD) negativity, and side effects are manageable. Investigators pooled data from the phase 1 pediatric (PALL) and adult (CALM) trials of UCART19... [Read Article]
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Emapalumab found safe, effective in primary HLH

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Outside the San Diego Convention Center, site of the 2018 ASH Annual Meeting
© ASH/Luke Franke 2018
Emapalumab, an interferon gamma-blocking antibody, controls disease activity and has a favorable safety profile in pediatric patients with primary hemophagocytic lymphohistiocytosis (HLH), according to research presented at the 2018 ASH Annual Meeting. Investigators believe emapalumab, which was... [Read Article]
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ALL regimens clear disease in kids with MPAL

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2018 ASH Annual Meeting
Photo by Jen Smith
Pediatric patients with mixed phenotype acute leukemia (MPAL) can achieve minimal residual disease (MRD) negativity with acute lymphoblastic leukemia (ALL)-directed chemotherapy, according to new research. In a retrospective study, most pediatric MPAL patients who received ALL-directed chemotherapy achieved an MRD-negative complete response (CR). Ninety-three percent of patients... [Read Article]
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FDA approves first treatment for primary HLH

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Emapalumab (Gamifant)
Photo from Business Wire
The U.S. Food and Drug Administration (FDA) has approved emapalumab-lzsg (Gamifant®) to treat primary hemophagocytic lymphohistiocytosis (HLH). Emapalumab, an interferon gamma-blocking antibody, is approved to treat to treat patients of all ages (newborn and older) with primary HLH who have refractory, recurrent, or progressive disease or who cannot... [Read Article]
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Lower threshold for platelet transfusions appears safer

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Infant in a neonatal
intensive care unit
Photo by Chris Horry
A lower threshold for platelet transfusions may be safer for preterm infants with severe thrombocytopenia, a new study suggests. Researchers randomized preterm infants with severe thrombocytopenia to receive transfusions at platelet count thresholds of 50,000 per cubic millimeter or 25,000 per cubic millimeter.... [Read Article]
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Palliative care guidelines relevant for hematologists, doc says

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Husband holding hands
with wife in hospital
Photo from Pexels
The latest edition of the national palliative care guidelines provides new clinical strategies relevant to hematology practice in the United States, according to a physician-researcher specializing in hematology. The guidelines represent a “blueprint for what it looks like to provide high-quality, comprehensive palliative care... [Read Article]
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Cost-effectiveness of CAR T-cell therapy

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Tisagenlecleucel (Kymriah)
Photo from Novartis
Tisagenlecleucel has the potential to be cost-effective for pediatric B-cell acute lymphoblastic leukemia (B-ALL) patients in the United States, according to researchers. The group found evidence to suggest the chimeric antigen receptor (CAR) T-cell therapy—which has a list price of $475,000—may prove cost-effective if long-term survival benefits are... [Read Article]
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Transfusion errors more common in kids than adults, study suggests

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Sarah Vossoughi, MD
Photo by Neil Osterweil
Even the most vigilant hospitals experience transfusion errors and problems with blood storage, according to researchers. A review of data from 32 U.S. hospitals showed that pediatric transfusions were associated with a higher rate of safety problems than adult transfusions, with errors differing by age group. The most... [Read Article]
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Bacteremic sepsis in ALL tied to neurocognitive dysfunction

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Child with leukemia
Photo by Bill Branson
Bacteremic sepsis during acute lymphoblastic leukemia (ALL) treatment may contribute to neurocognitive dysfunction later in life, results of a cohort study suggest. Pediatric ALL survivors who had sepsis while on treatment performed worse on measures of intelligence, attention, executive function, and processing speed than survivors with... [Read Article]
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