Miscellaneous Hematological Agents

Tibsovo Approved for Relapsed/Refractory AML With IDH1 Mutation

By July 20, 2018

The approval of Tibsovo was based on an open-label, single-arm, multicenter clinical trial (Study AG120-C-001) involving 174 patients with relapsed or refractory AML with an IDH1 miutation.

Galinpepimut-S Fast-Tracked for Multiple Myeloma Treatment

By July 20, 2018

As WT1 is present in an array of tumor types, the Company believes GPS has the potential to be a broad immunotherapy.

<small>L</small>-Glutamine Reduces Number of Pain Crises in Sickle Cell Disease

July 19, 2018

The researchers found that there were significantly fewer pain crises in the L-glutamine group than in the placebo group, with a median of 3.0 and 4.0, respectively.

Bone Marrow Transplant Alternative Granted Orphan Drug Designation

By July 17, 2018

It is also the first bone marrow transplant alternative to be granted Breakthrough Therapy status by the FDA.

Dilanubicel Gets Orphan Drug Status for Reduction of Morbidity/Mortality Associated With HSCT

By July 17, 2018

Dilanubicel is a universal donor, off-the-shelf-, ex vivo expanded hematopoietic stem and progenitor cell investigational product that provides rapid, transient hematopoiesis while also inducing long-term immunologic benefits.

FDA Drops Zika Testing for Blood Donors

July 10, 2018

"Now, given the significant decrease in cases of Zika virus infection in the United States and its territories, we are moving away from testing each individual donation to testing pooled donations."

Red Cross Issues Nationwide Call for Blood Donations

July 10, 2018

"Blood donations are currently being distributed to hospitals faster than donations are coming in - we need both new and current blood donors to make an appointment as soon as possible to help patients battling illness and injury," said Chris Hrouda, president of Red Cross Blood Services.

Glasdegib NDA Granted Priority Review for Acute Myeloid Leukemia

By June 28, 2018

Glasdegib, an investigational oral smoothened (SMO) inhibitor, is a once-daily therapy that is thought to work by disrupting the Hedgehog pathway.

Combination Therapy for Waldenström's Macroglobulinemia Gets Priority Review

By June 26, 2018

The sNDA is supported by data from the Phase 3 iNNOVATE (PCYC-1127) trial which assessed ibrutinib in combination with rituximab vs rituximab alone in 150 patients with previously untreated and relapsed/refractory WM.

FDA to Review Afamelanotide NDA for Erythropoietic Protoporphyria

By June 25, 2018

Scenesse (Clinuvel Pharmaceuticals) is a bimonthly, subcutaneous dose of afamelanotide 16mg.

Keytruda Approved for Primary Mediastinal Large B-Cell Lymphoma

By June 14, 2018

The approval was based on data from the KEYNOTE-170 study, a multicenter, open-label, single-arm trial that included 53 patients with relapsed or refractory PMBCL.

Kyprolis Labeling Updated With Overall Survival Data From Multiple Myeloma Trial

By June 11, 2018

ASPIRE (CArfilzomib, Lenalidomide, and DexamethaSone versus Lenalidomide and Dexamethasone for the treatment of PatIents with Relapsed Multiple MyEloma) was a Phase 3 trial that evaluated the triplet regimen Kyprolis, lenalidomide, and dexamethasone (KRd) vs lenalidomide and dexamethasone (Rd) alone, in patients with relapsed or refractory multiple myeloma who received 1-3 prior regimens.

Combo Tx Approved for Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma

By June 11, 2018

The new approval was based results from the Phase 3 MURANO trial which included 389 patients with CLL who had received ≥1 prior line of therapy.

Oral Propranolol Seems Safe for Infantile Hemangioma

By May 31, 2018

"In this study on a large continuous nationwide claims database, we confirm the safety profile of oral propranolol in healthy children to be good," the authors write.

CDC Warns of Serious Coagulopathy Outbreak Linked to Synthetic Cannabinoids

By May 29, 2018

Since brodifacoum - a long-acting vitamin K-dependent antagonist, used as a rodenticide - was detected in drug and biologic samples from patients using synthetic cannabinoids, there have been 202 reported cases of coagulopathy in 9 states (mostly Illinois; N=164)

FDA Committees Vote Against Drug Tx for Severe Neonatal Jaundice

By May 04, 2018

Stannsoporfin, a heme oxygenase inhibitor, works by inhibiting bilirubin production at its source.

Pharmacologic Tx for Severe Neonatal Jaundice to Be Reviewed by FDA

By February 23, 2018

The FDA has set an action date of August 22, 2018 to respond to the NDA

Treating Endothelial Function May Target Root Disease in Marfan Syndrome

February 13, 2018

The researchers found that the MFS/ATR1a-null mice showed unabated aortic root enlargement and remained responsive to losartan, despite being hypotensive and resistant to AngII vasopressor effects.

Antibiotic Prophylaxis Rates Low for Pediatric Sickle Cell Patients

February 06, 2018

Data were included for 2,821 children with 5,014 person-years. The researchers found that 18 percent of children received ≥300 days of antibiotics.

Results Released in Multi-Year IV Bevacizumab Study for HHT-Related Bleeding

February 05, 2018

After completion of the initial bevacizumab treatment cycle, there was a significant reduction in epistaxis severity scores and RBC transfusion requirements.

Should all Syncope Patients Undergo Evaluation for Pulmonary Embolism?

January 30, 2018

At 90 days, the prevalence of venous thromboembolism varied from 0.3 to 1.37% for all patients and from 0.75 to 3.86% for hospitalized patients.

New Guidelines Available on Anticoagulation Tx for Cardiopulmonary Bypass

January 22, 2018

Recommendations for heparin dosing and monitoring, contraindications and alternatives, reversal

Surgicel Powder Absorbable Hemostat Available for Bleeding Management

By January 09, 2018

Surgicel Powder Absorbable Hemostat is a powdered adjunctive hemostat used to control disruptive bleeding.

Siklos Approved for Pediatric Patients With Sickle Cell Anemia

By December 21, 2017

This is the first FDA approval of hydroxyurea for use in pediatric patients with sickle cell disease.

Case Study IDs Calciphylaxis Risk Factors, May Aid in Future Prevention

December 14, 2017

The researchers observed significant associations for lupus anticoagulant (48% positive in cases versus 5% in controls), protein C deficiency (50 versus 8%), and combined thrombophilias (62 versus 31%) with calciphylaxis among all patients.

Sickle Cell Disease Treatment Endari Soon to Be Available

By December 11, 2017

Endari is indicated to reduce the acute complications of sickle cell disease in adult and pediatric patients 5 years of age and older.

NDA Under Priority Review for Thrombocytopenia in Chronic Liver Disease

By November 28, 2017

A Prescription Drug User Free Act (PDUFA) date has been set for May 21, 2018.

Anticoagulant Linked to Reduced Cancer Incidence

November 08, 2017

"Warfarin use may have broad anticancer potential in a large, population-based cohort of persons older than 50 years," the authors write.

Lenalidomide Assessed for Hematologic Response in Patients With MDS/MPN-RS-T

November 01, 2017

"While lenalidomide therapy does produce hematological responses in patients with MDS/MPN-RS-T, these are often suboptimal, without significant changes in bone marrow morphology," the authors found.

AAP Revise Policy Statement on Cord Blood Banking

October 30, 2017

Physicians need to be able to explain concepts of autologous and allogeneic use of cord blood units, as well as benefits and limitations of blood banking and transplantation.