Miscellaneous Hematological Agents

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.

Blood Transfusions from Ever-Pregnant Donors and Recipient Mortality: What's the Link?

October 18, 2017

Receipt of red blood cell transfusion from ever-pregnant donor tied to increased risk for males.

Phase 3 Results Announced for Lusutrombopag for Thrombocytopenia

By September 25, 2017

The trial, L-PLUS2, included 215 patients who were randomized 1:1 to either 3mg of lusutrombopag or placebo for up to 7 days.

NDA Submitted for Thrombocytopenia Treatment

By September 22, 2017

The results from Phase 3 trials demonstrated efficacy and safety in patients with severe thrombocytopenia and with CLD.

Hemotopoietic Stem Cell Transplant Examined for Inherited Blood Disorder

September 13, 2017

The researchers found that in transplanted patients, the 30-year overall survival (OS) was 82.6±2.7% and thalassemia-free survival (TFS) was 77.8±2.9% compared with OS of 85.3±2.7% in CT patients (P=NS).

Blood Disorder ID'd as Possible Reason for Vampire Folklore

By September 08, 2017

Erythropoietic protoporphyria (EPP), the most common form of porphyria (a group of blood disorders), affects the body's ability to make heme resulting in symptoms of chronic anemia. A patient's skin can become very sensitive to light, resulting in disfiguring blisters.

BLA Submitted for Novel X-Linked Hypophosphatemia Tx

By August 24, 2017

Burosumab is being developed in a collaboration between Ultragenyx and Kyowa Hakko Kirin Pharmaceuticals, based on a license agreement between both companies.

Novel Reversal Agent for Factor Xa Inhibitors Under FDA Review

By August 17, 2017

The FDA has accepted for review the BLA resubmission for AndexXa (andexanet alfa; Portola) for the reversal of the anticoagulant effects of Factor Xa inhibitors in patients experiencing uncontrolled or life-threatening bleeding.

'Groundbreaking' Technology Heals Serious Injuries with a Single Touch

By August 09, 2017

"By using our novel nanochip technology, injured or compromised organs can be replaced. We have shown that skin is a fertile land where we can grow the elements of any organ that is declining," said Dr. Chandan Sen.

FDA Grants Zelboraf Priority Review for Rare Blood Disease

By August 07, 2017

The application includes data from the open-label, Phase 2 VE-BASKET study; non-randomized, basket study investigating the use of Zelboraf in patients with BRAF V600 mutation-positive cancers and other diseases, including 22 patients with ECD.

FDA Approves New Sickle Cell Disease Treatment

By July 07, 2017

The safety and efficacy of Endari were evaluated in a randomized clinical trial involving patients aged 5-58 years with sickle cell disease who had ≥2 painful crises within 12 months prior to trial enrollment.

Novel Synthetic Bio Drug for Hyperammonemia Granted Fast Track

By June 26, 2017

Synthetic Biotic medicines utilize synthetic biology to reprogram probiotic bacteria to perform critical functions that compensate for those missing or damaged due to a particular disease.

Dosing Errors Common with Certain Blood-Thinning Medications

June 06, 2017

Study of newer medications finds 16 percent receive too much or too little

Mepolizumab Evaluated in Eosinophilic Vasculitis

May 26, 2017

The researchers found that, compared with placebo, mepolizumab treatment correlated with significantly more accrued weeks of remission.

New Formulation of Jadenu Approved

By May 22, 2017

Both the tablets and granules should not be taken with aluminum-containing antacid products. The drug labeling contains additional information for patients who are on current chelation therapy with Exjade (deferasirox) tablets for oral suspension and converting to Jadenu.

Mepolizumab or Placebo for Eosinophilic Granulomatosis with Polyangiitis

By May 18, 2017

Wechsler, ME et al. "Mepolizumab or Placebo for Eosinophilic Granulomatosis with Polyangiitis". DOI: 10.1056/NEJMOA1702079

Delaying Anticoagulation Tx in Afib May Increase Dementia Risk

May 15, 2017

Even patients with a low risk of stroke could benefit, researchers say

Blood Type May be Factor in Heart Attack Risk

By May 02, 2017

The odds ratio for all coronary events and combined cardiovascular events was found to be significantly higher in carriers of a non-O blood group.

Rydapt Approved for Acute Myeloid Leukemia, Mastocytosis

By April 28, 2017

Rydapt works by inhibiting multiple receptor tyrosine kinases.

FDA Approves Test for Suspected Polycythemia Vera Evaluation

By April 07, 2017

The ipsogen JAK2 RGQ PCR Kit test detected polycythemia vera with 94.6% sensitivity and 98.1% specificity, in the study the approval was based on.

DAPT Cessation Significantly Lower in Diabetic Patients

March 29, 2017

Risk of major adverse cardiovascular events up for DAPT disruption, regardless of diabetes status

Levosimendan Treatment Assessed in Post-Cardiac Sx Patients

March 24, 2017

"In patients who required perioperative hemodynamic support after cardiac surgery, low-dose levosimendan in addition to standard care did not result in lower 30-day mortality than placebo," the authors write.

ASA Hyporesponsiveness After CABG May Be Overcome by Multiple Dosing

March 22, 2017

Multiple dosing more effective than ASA 81 or 325 mg once-daily for suppressing serum TXB2 formation

FDA Fast Tracks Recombinant ADAMTS13 Enzyme Replacement Therapy

By March 22, 2017

Shire announced that the FDA has granted Fast Track designation to SHP655 (historically BAX930) for the treatment of acute episodes of hereditary thrombotic thrombocytopenic purpura (hTTP) in patients with a constitutional ADAMTS13 deficiency.

Stem Cell Transplant Cures First Adult Patient of Rare Blood Disorder

By March 21, 2017

The patient underwent a non-myeloablative (NMA) peripheral blood stem cell transplant from a 10/10 HLA Ag matched, ABO incompatible unrelated donor.

Risks Following RFA for Liver Cancer in ESRD Patients on Hemodialysis Investigated

March 20, 2017

Dialyzed patients have higher in-hospital mortality, hemorrhagic complications after RFA for HCC