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FDA grants orphan drug, rare pediatric disease status to entrectinib for neuroblastoma

December 29, 2014

The FDA has granted both orphan drug designation and rare pediatric disease designation to entrectinib for the treatment of neuroblastoma, according to the drug’s manufacturer.

Entrectinib (Ignyta Inc.), a selective tyrosine kinase inhibitor, is a targeted therapeutic candidate intended for patients with cancers that contain activating alterations to TrkA, TrkB, TrkC, ROS1 or ALK. Based on prior mouse xenograft models, entrectinib has demonstrated in vivo antitumor activity against multiple TrkA, ROS1 or ALK-driven cancers.

David Bundy In the Journals

Quality improvement efforts reduced pediatric CLABSI rates

December 9, 2014
Implementing a collaborative quality improvement program yielded significant reductions in the rate of central line-associated bloodstream infections among pediatric…
In the Journals

Telephone counseling increased cardiomyopathy screenings in childhood cancer survivors

December 4, 2014
The addition of telephone counseling to a printed survivorship care plan increased the frequency with which survivors of childhood cancer at risk for cardiovascular…
Sally E. Carty, MD In the Journals

High radioactive iodine-131 exposure dose linked to aggressive thyroid cancer

December 2, 2014
Children and adolescents exposed to high doses of radioactive iodine-131 after the Chernobyl nuclear power plant accident experienced greater rates of more aggressive…
CME
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Targeted Therapy and Immunotherapy in the Management of Metastatic Melanoma: 2014 Updates

This activity is supported by an educational grant from Merck & Co., Inc.

Melanoma remains a challenging condition for clinicians. An array of targeted therapies and immunotherapy are in…
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CME CNE
Stem Cell Transplant for AAMDS

Treatment Decision-Making through the Spectrum of MDS:
Case 3 Stem Cell Transplant for MDS

An educational presentation from Aplastic Anemia & MDS International Foundation.

MDS is a difficult disease to diagnose and treat. Frequently it is challenging for healthcare providers to explain to…
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FDA News

FDA grants orphan drug, rare pediatric disease status to entrectinib for neuroblastoma

December 29, 2014
The FDA has granted both orphan drug designation and rare pediatric disease designation to entrectinib for the treatment of neuroblastoma, according…
David Bundy In the Journals

Quality improvement efforts reduced pediatric CLABSI rates

December 9, 2014
Implementing a collaborative quality improvement program yielded significant reductions in the rate of central line-associated bloodstream infections…
In the Journals

Telephone counseling increased cardiomyopathy screenings in childhood cancer survivors

December 4, 2014
The addition of telephone counseling to a printed survivorship care plan increased the frequency with which survivors of childhood cancer at risk for…
Sally E. Carty, MD In the Journals

High radioactive iodine-131 exposure dose linked to aggressive thyroid cancer

December 2, 2014
Children and adolescents exposed to high doses of radioactive iodine-131 after the Chernobyl nuclear power plant accident experienced greater rates…
In the Journals

AAP, ACIP revise recommendation for PCV13 among children at high risk for IPD

December 1, 2014
The CDC’s Advisory Committee on Immunization Practices and the AAP have updated recommendations for use of the 13-valent pneumococcal conjugate…
In the Journals

Model predicted risk for heart failure in childhood cancer survivors

November 14, 2014
Models that included factors such as age at diagnosis, anthracycline doses, and radiation fields and doses accurately predicted risk for congestive…
Norman E. Breslow, PhD In the Journals

Female survivors of Wilms tumor may be at increased risk for breast cancer

November 13, 2014
Women who survived Wilms tumor, particularly those who underwent chest radiotherapy, demonstrated increased risk for invasive breast cancer by age 40…
In the Journals

Nearly one-third of AYA patients do not receive adequate cancer therapy

November 7, 2014
About 30% of adolescents and young adults with cancer do not receive appropriate initial treatment, according to study results.Arnold L. Potosky
Earlier hepatoblastoma surgery may reduce chemotherapy complications In the Journals

Earlier hepatoblastoma surgery may reduce chemotherapy complications

November 7, 2014
Many pediatric patients with hepatoblastoma achieved radiographic resectability after two cycles of chemotherapy compared with four cycles, creating…
John Wagner In the JournalsPerspective

Double-unit cord-blood transplant failed to extend survival in blood cancers

October 29, 2014
Children and adolescents with hematologic malignancies who underwent double-unit cord-blood transplantation experienced similar survival rates as…
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