Recent New Drug Approvals. Part 1: Drugs with Pediatric Indications

Shelton, Chasity M.; Chhim, Rebecca F.; Christensen, Michael L.

doi:10.5863/1551-6776-17.4.329

Cited by 4 publications

(3 citation statements)

References 23 publications

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“…The objective of this study was to collect additional safety information on asparaginase Erwinia chrysanthemi in a systematic manner in a large compassionate‐use trial and to support Food and Drug Administration (FDA) approval of the product. Asparaginase Erwinia chrysanthemi was subsequently approved by the FDA in November 2011 for patients with ALL who develop a hypersensitivity reaction to E. coli ‐derived asparaginase .…”

Section: Introductionmentioning

confidence: 99%

Safety profile of asparaginase Erwinia chrysanthemi in a large compassionate‐use trial

Plourde

Jeha

Hijiya

et al. 2014

Pediatric Blood & Cancer

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Background L‐Asparaginase is an integral component of standard chemotherapy regimens for the treatment of acute lymphoblastic leukemia (ALL). Clinical hypersensitivity, a common reason for treatment discontinuation, has been reported in 10–30% of patients receiving Escherichia coli‐derived asparaginase. After hypersensitivity, E. coli‐derived asparaginase should be discontinued and an alternative asparaginase preparation, such as asparaginase Erwinia chrysanthemi, may be initiated. We conducted a compassionate‐use study to collect additional safety information on asparaginase Erwinia chrysanthemi and to support FDA approval of the product. Procedure Patients with ALL or lymphoblastic lymphoma (LBL; N = 1368) who developed a hypersensitivity reaction (grade ≥2) to an E. coli‐derived asparaginase participated in this trial. The recommended asparaginase Erwinia chrysanthemi dose was 25,000 IU/m2 three days per week (Monday/Wednesday/Friday) for two consecutive weeks for each missed pegylated E. coli‐derived asparaginase dose and 25,000 IU/m2 for each missed nonpegylated asparaginase dose for the completion of their planned asparaginase treatment. Results Adverse event reports and/or case report forms were completed for 940 patients. The most common adverse event (AE) was hypersensitivity (13.6%). Eighteen patients (1.9%) died during the study. Most patients (77.6%) completed their planned asparaginase treatment with asparaginase Erwinia chrysanthemi. There was no apparent difference in the incidence of the most commonly reported AEs with asparaginase treatment by age, administration, or disease state. Conclusions This study further established the safety profile of asparaginase Erwinia chrysanthemi in patients with ALL or LBL who had a hypersensitivity reaction to an E. coli‐derived asparaginase. Pediatr Blood Cancer 2014;61:1232–1238. © 2014 Wiley Periodicals, Inc.

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Section: Introductionmentioning

confidence: 99%

Safety profile of asparaginase Erwinia chrysanthemi in a large compassionate‐use trial

Plourde

Jeha

Hijiya

et al. 2014

Pediatric Blood & Cancer

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“…The objective of this study was to collect additional safety information on asparaginase Erwinia chrysanthemi in a systematic manner in a large compassionate-use trial and to support Food and Drug Administration (FDA) approval of the product. Asparaginase Erwinia chrysanthemi was subsequently approved by the FDA in November 2011 for patients with ALL who develop a hypersensitivity reaction to E. coli-derived asparaginase [25].…”

Section: Introductionmentioning

confidence: 99%

L-Asparaginase (L-ASP) Related Toxicities with Asparaginase Erwinia Chrysanthemi in a Large Compassionate Use Protocol.

Plourde

Jeha

Hijiya

et al. 2012

Blood

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2568 Background: L-asparaginase (L-ASP) is an important component of multi-agent chemotherapy for treatment of Acute Lymphoblastic Leukemia (ALL) in children and young adults. The pegylated E. coli derived form, Oncaspar® (PEG-ASP), is most commonly used because of its longer half-life and lower immunogenicity compared to the native enzyme; however, clinical hypersensitivity reactions still occurs in 10–30% of patients (pts) requiring its discontinuation. Asparaginase Erwinia Chrysanthemi (Erwinaze™) is an L-ASP derived from a different bacterium and is immunologically distinct from the E. coli L-ASP. We conducted a compassionate use trial of Erwinaze in pts with ALL and hypersensitivity to native E. coli or PEG-ASP to collect safety information. Patients and Methods: Pts of any age with ALL or lymphoblastic lymphoma (LBL) who developed a Grade ≥2 clinical hypersensitivity reaction to PEG-ASP or native E. coli ASP (Elspar®) were eligible. Pts with a history of pancreatitis, previous allergic reaction to Erwinaze, or pregnant, were excluded. The study was IRB approved at each institution and pts/family provided informed consent/assent. Safety information on Erwinaze-related adverse events (AEs) were captured on Case Report Forms (CRFs) submitted to the Sponsor when the pt completed his/her entire Erwinaze treatment plan. AEs may have also been reported directly by the investigational sites. Results: Between February 2006 and November 2011, 1,368 pts were treated with Erwinaze. CRFs were received in 893 pts and 47 additional AEs were received from patients for which a CRF was not obtained. The average age was 9.6 years (range 1–66). The majority of patients (63.5%) were male. Of the pts for which CRFs were received (893); 77.6% were able to conclude their Erwinaze treatment. Discontinuation was due to allergic reaction in 8.8%; other AEs 4.7%; at the physician or patient discretion 7.5%; and missing information in 1.3%. Anaphylaxis was reported in 8 pts (0.9%) and Grade ≥2 clinical hypersensitivity reactions in 130 (13.8%). The incidence of pancreatitis was 3.9%, hemorrhagic or thrombosis abnormalities 2.4%, hyperglycemia 3.6% and elevation in liver enzymes 3.5%. There were 18 deaths on study; 11 disease progression, 3 intracranial hemorrhage, 4 other individual reports. Conclusion: This is the largest study of pts treated with Erwinaze to determine the toxicity profile. Erwinaze was well tolerated with no unexpected toxicities identified beyond those associated with L-Asp treatment. This compassionate use trial permitted the continuation and completion of asparaginase treatment in 77.6% of pts with hypersensitivity reactions to E. coli formulations. Final results will be available for presentation. Disclosures: Plourde: Jazz Pharmaceuticals: Employment, Equity Ownership. Mercedes:Jazz Pharmaceuticals: Employment. Corn:EUSA Pharma: Employment.

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“…Recently, a Michigan consortia consisting of professional societies, university, and healthcare organizations created a website to improve patient safety through standardized oral liquid concentrations [16]. While the number of new molecular entities with either pediatric nosology or posology in official product labeling has increased exponentially since enactment of BPCA [17], scientific evidence for the use, preparation, and monitoring of currently marketed drugs remains elusive [18][19][20].…”

Section: Statement Of the Problemmentioning

confidence: 99%

Optimizing Effectiveness in Electronic Prescriptions for Pediatric Outpatients: A Call for Responsive Action

et al. 2014

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A pediatric compounded non-sterile products repository (pCNP) to optimize the effectiveness and safety of "off-label" use of compounded pharmacotherapy through complete transmission of electronic prescriptions across the continuum of care is described. The advent of electronic prescribing has the potential to refocus and resolve long-standing issues of prescription therapy for pediatric patients related to formulation, indication, dosing, and outcomes follow-up, among others. This white paper describes the architecture and function of the pCNP repository. Further, it calls on professional societies, health information technology (HIT) and pharmaceutical industries, universities, and government to create a safer pediatric pharmacotherapy system across the continuum of care. This system would include pCNPs within the existing federal and corporate database structures for medical language, and integrates advanced system safety features as requirements for prescribing, compounding, and dispensing non-mass produced prescription therapies for children.

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Recent New Drug Approvals. Part 1: Drugs with Pediatric Indications

Cited by 4 publications

References 23 publications

Safety profile of asparaginase Erwinia chrysanthemi in a large compassionate‐use trial

Safety profile of asparaginase Erwinia chrysanthemi in a large compassionate‐use trial

L-Asparaginase (L-ASP) Related Toxicities with Asparaginase Erwinia Chrysanthemi in a Large Compassionate Use Protocol.

Optimizing Effectiveness in Electronic Prescriptions for Pediatric Outpatients: A Call for Responsive Action

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