FDA grants fast track designation to dilanubicel

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Cord blood donation
The US Food and Drug Administration (FDA) has granted fast track designation to dilanubicel (NLA101) for use in patients with high-risk hematologic malignancies receiving an allogeneic cord blood transplant. Dilanubicel is a universal-donor, ex-vivo-expanded hematopoietic stem and progenitor cell product. It is intended to induce short-term hematopoiesis, which lasts until a patient’s... [Read Article]
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A new way to expand HSCs for UCB transplant

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Cord blood donation
Photo courtesy of NHS
Researchers say they have discovered a new approach to expand hematopoietic stem cells (HSCs) from umbilical cord blood (UCB). The team identified a protein, YTHDF2, that affects multiple targets and pathways involved in HSC self-renewal. Experiments showed that reducing the function of YTHDF2 allowed UCB HSCs to expand.... [Read Article]
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FDA advises against azithromycin use in allo-HSCT recipients

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HSCT preparation
Photo by Chad McNeeley
The US Food and Drug Administration (FDA) is warning against long-term use of azithromycin (Zithromax, Zmax) in patients who undergo allogeneic hematopoietic stem cell transplant (allo-HSCT). Azithromycin has been used off-label as prophylaxis for bronchiolitis obliterans syndrome in these patients. However, a trial published in JAMA last year suggested... [Read Article]
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Kids may have higher risk of death long after allo-HSCT

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Smita Bhatia
Photo from UAB
Children may have an increased risk of premature death decades after allogeneic hematopoietic stem cell transplant (allo-HSCT), according to a study published in JAMA Oncology. The leading causes of death in the patients studied were infection and chronic graft-vs-host disease (GVHD), patients’ primary disease, and subsequent cancers. “This study shows... [Read Article]
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Orphan designation recommended for OMS721

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HSCT preparation
Photo by Chad McNeeley
The European Medicines Agency’s (EMA’s) Committee for Orphan Medicinal Products (COMP) has issued a positive opinion recommending orphan drug designation for OMS721 as a treatment for high-risk hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA). OMS721 is a monoclonal antibody targeting MASP-2, the effector enzyme of the lectin pathway of... [Read Article]
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Study could change treatment of MLSM7

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Study authors Tamara
Lamprecht, Jason Schwartz,
Jing Ma, and Jeffrey Klco
Photo from St. Jude
Children’s Research
Hospital/Justin Veneman
New findings could help improve treatment of an inherited bone marrow disorder known as myelodysplasia and leukemia syndrome with monosomy 7 (MLSM7), according to researchers. While studying families affected by MLSM7, researchers identified germline mutations... [Read Article]
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Treatments, disease affect spermatogonia in boys

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Male germinal epithelium
showing spermatogonia,
spermatocytes, spermatids,
and spermatozoa
Image from Dreamstime
Alkylating agents, hydroxyurea (HU), and certain non-malignant diseases can significantly deplete spermatogonial cell counts in young boys, according to research published in Human Reproduction. Boys who received alkylating agents to treat cancer had significantly lower spermatogonial cell counts than control subjects or boys... [Read Article]
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FDA approves biosimilar filgrastim

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Vials of drug
Photo by Bill Branson
The US Food and Drug Administration (FDA) has approved the leukocyte growth factor Nivestym™ (filgrastim-aafi), a biosimilar to Neupogen (filgrastim). Nivestym is approved to treat patients with nonmyeloid malignancies who are receiving myelosuppressive chemotherapy or undergoing bone marrow transplant, acute myeloid leukemia patients receiving induction or consolidation chemotherapy,... [Read Article]
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FDA grants UCB product orphan designation

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Cord blood donation
Photo courtesy of NHS
The US Food and Drug Administration (FDA) has granted orphan drug designation to NiCord for hematopoietic stem cell transplant. NiCord is created by expanding and enriching a unit of umbilical cord blood (UCB). The product consists of a CD133-positive fraction—which is cultured for 21 days with nicotinamide, thrombopoietin,... [Read Article]
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Product receives orphan designation for use in HSCT

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Hematopoietic stem cells
in the bone marrow
The US Food and Drug Administration (FDA) has granted orphan drug designation to dilanubicel (NLA101) for the reduction of morbidity and mortality associated with hematopoietic stem cell transplant (HSCT). Dilanubicel is a universal-donor, ex-vivo-expanded hematopoietic stem and progenitor cell product. Dilanubicel is intended to induce short-term hematopoiesis, which... [Read Article]
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New approach to AE reporting needed, group says

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Doctor and patient
Photo courtesy of NIH
A group of experts has called for improvements in reporting adverse events (AEs) that occur in patients with hematologic malignancies. The group highlighted deficiencies in capturing chronic, cumulative, and late AEs; collecting patient-reported outcomes (PROs); reporting AEs associated with hematopoietic stem cell transplant (HSCT); assessing long-term toxicity in... [Read Article]
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Transplant strategy not viable for aggressive B-NHL

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HSCT preparation
Photo by Chad McNeeley
Transplant with radioimmunotherapy (RIT)-based conditioning is a viable treatment option for patients with indolent—but not aggressive—B-cell non-Hodgkin lymphomas (NHLs), according to researchers. Long-term follow-up data showed “excellent” outcomes in patients with indolent B-NHL who received conditioning with 90Y-ibritumomab tiuxetan plus fludarabine and low-dose total body irradiation (TBI) prior... [Read Article]
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A ‘highly effective’ strategy for haplo-HSCT

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Daria Pagliara, MD, PhD
Researchers have identified a “highly effective” transplant strategy for pediatric patients with primary immunodeficiencies who lack a suitable HLA-compatible donor, according to a speaker at the 23rd Congress of the European Hematology Association (EHA). The strategy involves α/β T-cell- and B-cell-depleted haploidentical hematopoietic stem cell transplant (haplo-HSCT) followed by infusion of... [Read Article]
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