Novel treatment for severe congenital neutropenia with pegfilgrastim

Choi, Leslie; Guelcher, Christine; Guerrera, Michael F.

doi:10.1182/blood-2007-08-106807

Cited by 10 publications

(12 citation statements)

References 8 publications

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“…The use of pegfilgrastim instead of filgrastim is theoretically attractive, and has been tried in isolated cases [91–94], but has not been routinely accepted, and may in fact lead to an increase in adverse events [95,96]. One obstacle to the use of this agent in the United States is the packaging of the commercially available product in a syringe prefilled with the adult dose of 6 mg.…”

Section: Treatment Of Neutropenia In Primary Immunodeficiency Diseasementioning

confidence: 99%

Neutropenia in primary immunodeficiency

Sokolic

2013

Current Opinion in Hematology

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Purpose of review Neutropenia is a feature of several primary immunodeficiency diseases (PIDDs). Because of the diverse pathophysiologies of the PIDDs and the rarity of each disorder, data are often lacking, leading to the necessity of empiric treatment. Recent developments in the understanding of neutropenia in several of the PIDDs make a review of the data timely. Recent findings The category of severe congenital neutropenia continues to expand. Mutations in G6PC3 have been identified as the cause of neutropenia in a minority of previously molecularly undefined cases. Recent advances have broadened our understanding of the pathophysiology and the clinical expression of this disorder. A possible function of the C16orf57 gene has been hypothesized that may explain the clinical overlap between Clerucuzio-type poikiloderma with neutropenia and other marrow diseases. Plerixafor has been shown to be a potentially useful treatment in the warts, hypogammaglobulinemia, infection, and myelokathexis syndrome. Investigations of patients with adenosine deaminase deficient severe combined immunodeficiency have identified neutropenia, and particularly susceptibility to myelotoxins, as a feature of this disorder. Granulocyte-colony stimulating factor is the treatment of choice for neutropenia in PIDD, whereas hematopoietic cell transplantation is the only curative option. Summary The number of PIDDs associated with neutropenia has increased, as has our understanding of the range of phenotypes. Additional data and hypotheses have been generated helping to explain the diversity of presentations of neutropenia in PIDDs.

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Section: Treatment Of Neutropenia In Primary Immunodeficiency Diseasementioning

confidence: 99%

Neutropenia in primary immunodeficiency

Sokolic

2013

Current Opinion in Hematology

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“…As filgrastim requires daily injections, clinical benefit obtained with pegfilgrastim may be due to better compliance to treatment with longer injection intervals. 21,22 Further studies are required to understand the efficacy of pegfilgrastim on neutrophil functions and long-term outcome of IBD in patients with G6PC3 deficiency.…”

Section: Discussionmentioning

confidence: 99%

Resolution of Inflammatory Colitis With Pegfilgrastim Treatment in a Case of Severe Congenital Neutropenia Due to Glucose 6 Phosphatase Catalytic Subunit-3 Deficiency

Kaya

Eğritaş

Albayrak

et al. 2014

Journal of Pediatric Hematology/Oncology

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Glucose 6 phosphatase catalytic subunit-3 (G6PC3) deficiency is a heterogenous disorder characterized by severe congenital neutropenia and a variety of extrahematopoietic manifestations. Inflammatory bowel disease like colitis is an uncommon complication of G6PC3 deficiency, described only in adolescent and adults. Herein, we describe inflammatory colitis in a 10-year-old girl with severe congenital neutropenia due to G6PC3 deficiency while she was on a high-dose filgrastim. Switching from filgrastim to (pegylated filgrastim) Pegfilgrastim led to rapid resolution of colitis, weight gain, and decreased infections. Pegfilgrastim seems to be a better remedy for treatment of G6PC3 deficiency complicated with inflammatory bowel disease.

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“…However, the expert agreed on the non‐superiority of lenograstim vs. filgrastim (II, 8.1, C) as the number of infections was similar [16] in patients treated with the two preparations. The use of pegylated G‐CSF in neutropenia has been rarely reported in pediatric age [17–20].…”

Section: Therapymentioning

confidence: 99%

Congenital and acquired neutropenias consensus guidelines on therapy and follow‐up in childhood from the Neutropenia Committee of the Marrow Failure Syndrome Group of the AIEOP (Associazione Italiana Emato‐Oncologia Pediatrica)

et al. 2011

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ning Studies for Rare Thrombotic and Hemostatic Disorders (U34) (RFA-HL-12-023) and the NHLBI Clinical Trial Pilot Studies (R34) (PAR-10-005) awards.You may find more information for this FOA at: http://grants.nih.gov/ grants/guide/rfa-files/RFA-HL-12-016.html.The NHLBI also has a program for NHLBI Clinical Trial Pilot Studies (R34), PAR-10-005, which supports pilot studies to obtain data critical for the design of robust clinical trials. Additional information is at: http://grants.-nih.gov/grants/guide/pa-files/PAR-10-005.html.In addition, NHLBI continues the program for Ancillary Studies in Clinical Trials (R01), RFA-HL-12-012, which is for the conduct of time-sensitive ancillary studies related to heart, lung and blood diseases and sleep disorders in conjunction with ongoing clinical trials. Additional information is at: http:// grants.nih.gov/grants/guide/rfa-files/RFA-HL-12-012.html.

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Novel treatment for severe congenital neutropenia with pegfilgrastim

Cited by 10 publications

References 8 publications

Neutropenia in primary immunodeficiency

Neutropenia in primary immunodeficiency

Resolution of Inflammatory Colitis With Pegfilgrastim Treatment in a Case of Severe Congenital Neutropenia Due to Glucose 6 Phosphatase Catalytic Subunit-3 Deficiency

Congenital and acquired neutropenias consensus guidelines on therapy and follow‐up in childhood from the Neutropenia Committee of the Marrow Failure Syndrome Group of the AIEOP (Associazione Italiana Emato‐Oncologia Pediatrica)

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