Hematological Disorders

ESA Biosimilar Effective for Anemia in Hemodialysis Patients

July 13, 2018

Researchers found "no clinically meaningful differences" in efficacy and safety between the biosimilar epoetin alfa-epbx and epoetin alfa.

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.

Most Bleeding Events in Non-CVD Patients Are GI-Related

June 28, 2018

The risk of a nonfatal GI bleeding event was 2.19 per 1,000 person-years in the baseline cohort, 1.77 in the non-high-risk cohort, and 1.61 in the nonmedication cohort.

Clotting Time in Transfemoral PCI Linked to Bleeding Risk

June 27, 2018

Researchers related maximal ACT to the risk of major bleeding in 14,634 patients undergoing TR or TF PCI with unfractionated heparin monotherapy.

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.

Cinryze Approved for Pediatric Patients With Hereditary Angioedema

By June 22, 2018

The approval was based on data from a randomized, single-blind, multicenter, dose-ranging, crossover study which evaluated the safety and efficacy of Cinryze in 12 pediatric patients (7 to 11 years old).

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.

Isolated mild/moderate thrombocytopenia may not require intervention

June 11, 2018

In a review of children who presented to a specialist hematology clinic with isolated mild or moderate low platelet counts, though causes were variable, most required no interventions.

Bleeding Disorder Treatments: Hemophilia B

June 08, 2018

Treatment options for Hemophilia B.

Olinciguat Granted Orphan Drug Status for Sickle Cell Disease

By June 08, 2018

Olinciguat is a soluble guanylate cyclase stimulator currently in Phase 2 development.

Bleeding Disorder Treatments: von Willebrand Disease

June 08, 2018

Treatment options for von Willebrand Disease (VWD).

Mircera Approved to Treat Anemia in Pediatric Patients With CKD

By June 07, 2018

The approval was based on an open-label, multiple dose, multicenter, dose-finding study in 64 pediatric patients with CKD who were on hemodialysis and who had stable hemoglobin levels while previously receiving another ESA.

Bleeding Disorder Treatments: Hemophilia A

June 06, 2018

Treatment options for Hemophilia A including factors and other biologics.

First Biosimilar Pegfilgrastim Gets FDA Approval

By June 05, 2018

Fulphila will be available as a 6mg/0.6mL single-dose prefilled syringe for subcutaneous (SC) injection.

Doptelet Now Available for Thrombocytopenia in Chronic Liver Disease

By May 31, 2018

The drug works by stimulating the proliferation and differentiation of megakaryocytes from bone marrow progenitor cells resulting in an increased production of platelets.

Larger Vial Size Approved for Hemophilia B Treatment Idelvion

By May 31, 2018

The new 3500 IU vial size will help shorten the reconstitution time needed to prepare multiple vials for a similar dose.

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.

FDA to Review Promacta for First-Line Treatment of Severe Aplastic Anemia

By May 30, 2018

The FDA decision to grant Priority Review was based on results from a National Institute of Health (NIH) study which demonstrated a 52% complete response rate and an 85% overall response rate in treatment-naïve SAA patients treated with eltrombopag plus standard IST.

Tavalisse Now Available for Chronic Immune Thrombocytopenia

By May 29, 2018

Treatment with Tavalisse should be discontinued after 12 weeks if the platelet count does not increase to a level sufficient to avoid clinically important bleeding.

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)

Extended INR Test Intervals Safe for Warfarin-Treated Patients

May 23, 2018

Similar number of out-of-range next INR values for patients with, without extended testing interval

Doptelet Approved for Chronic Liver Disease Patients With Thrombocytopenia

By May 21, 2018

The approval of Doptelet was based on results from the ADAPT-1 (N=231) and ADAPT-2 trials (N=204), 2 identically-designed multicenter, randomized, double-blind, placebo-controlled studies.

FDA Approves Epoetin Alfa Biosimilar Retacrit

By May 15, 2018

The approval of Retacrit was based on a review of evidence that included human pharmacokinetic/pharmacodynamic data and clinical immunogenicity data which demonstrated a high degree of similarity between Retacrit and its reference product.

First Generic Alternative for Mephyton Now Available

By May 15, 2018

Amneal's generic product is available in a 5mg strength tablet supplied in 100-count bottles.

FDA Approves Hemospray to Treat Non-Variceal GI Bleeds

By May 09, 2018

The product is a single-use device that delivers hemostatic powder through the channel of an endoscope.