{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,12,29]],"date-time":"2025-12-29T11:06:25Z","timestamp":1767006385153},"reference-count":34,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2009,7,17]],"date-time":"2009-07-17T00:00:00Z","timestamp":1247788800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"name":"Inserm AFM for Rare Diseases","award":["RAF0007"],"award-info":[{"award-number":["RAF0007"]}]},{"name":"Soci\u00e9t\u00e9 d'H\u00e9matologie et Immunologie P\u00e9diatrique"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Pediatric Blood &amp; Cancer"],"published-print":{"date-parts":[[2009,12]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Aims<\/jats:title><jats:p>To examine the efficacy and safety of pegfilgrastim in patients with congenital neutropenia (CN).<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Seventeen patients enrolled in the French Severe CN Register received pegfilgrastim.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Median age at pegfilgrastim introduction was 19.1 years (range 3.9\u201352.3 years). In 14 cases pegfilgrastim replaced GCSF (filgrastim or lenograstim), after a median of 6.9 years of GCSF therapy. The dose of pegfilgrastim was usually one full vial per injection (except in five children, who received 1\/6 to 1\/2 a vial), resulting in a dose of between 50 and 286\u2009\u00b5g\/kg. The pegfilgrastim schedule ranged from two injections every 7 days to one injection every 30 days, with treatment\u2010free periods. The median interval between the first and last dose of pegfilgrastim was 0.8 years (0.01\u20134.1 years). The absolute neutrophil count tended to increase more strongly on pegfilgrastim than on GCSF, but the difference was not statistically significant. During pegfilgrastim therapy, a severe infection occurred in two patients and recurrent ENT infections in two other patients. Bone pain was reported by nine patients, anemia and thrombocytopenia occurred in one patient (WHO grade III), chronic urticaria occurred in one patient (WHO grade III), and a single pegfilgrastim injection was followed by respiratory distress and death 15 days later in a patient with GDSIb. At the last update, 10 patients had stopped receiving pegfilgrastim and seven patients were still receiving pegfilgrastim.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>Compared to conventional GCSF, pegfilgrastim is more difficult to use in congenital neutropenia, with more frequent adverse events and sometimes poor efficacy. Pediatr Blood Cancer 2009;53:1068\u20131073. \u00a9 2009 Wiley\u2010Liss, Inc.<\/jats:p><\/jats:sec>","DOI":"10.1002\/pbc.22147","type":"journal-article","created":{"date-parts":[[2009,7,18]],"date-time":"2009-07-18T07:54:20Z","timestamp":1247903660000},"page":"1068-1073","source":"Crossref","is-referenced-by-count":19,"title":["Is pegfilgrastim safe and effective in congenital neutropenia? An analysis of the French Severe Chronic Neutropenia registry"],"prefix":"10.1002","volume":"53","author":[{"given":"Blandine","family":"Beaupain","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Thierry","family":"Leblanc","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Oum\u00e9daly","family":"Reman","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Olivier","family":"Hermine","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jean\u2010Pierre","family":"Vannier","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Felipe","family":"Suarez","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Patrick","family":"Lutz","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Pierre","family":"Bordigoni","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Anne","family":"Jourdain","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Michele","family":"Schoenvald","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Marie","family":"Ouachee","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sylvie","family":"Fran\u00e7ois","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Fr\u00e9deric","family":"Kohser","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Fabrice","family":"Jardin","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Gilles","family":"Devouassoux","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yves","family":"Bertrand","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Raphaele","family":"Nove\u2010Josserand","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jean","family":"Donadieu","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2009,7,17]]},"reference":[{"key":"e_1_2_7_2_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM198906153202402"},{"key":"e_1_2_7_3_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.leukres.2006.11.016"},{"key":"e_1_2_7_4_2","doi-asserted-by":"publisher","DOI":"10.1038\/sj.leu.2405038"},{"key":"e_1_2_7_5_2","doi-asserted-by":"publisher","DOI":"10.1177\/0091270006288731"},{"key":"e_1_2_7_6_2","doi-asserted-by":"publisher","DOI":"10.1592\/phco.23.9.9S.32888"},{"key":"e_1_2_7_7_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2007-08-106807"},{"key":"e_1_2_7_8_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jaad.2004.12.028"},{"key":"e_1_2_7_9_2","doi-asserted-by":"publisher","DOI":"10.1111\/j.1600-0609.2008.01145.x"},{"key":"e_1_2_7_10_2","first-page":"45","article-title":"Analysis of risk factors for myelodysplasias, leukemia and death from infection among patients with congenital neutropenia. Experience of the French Severe Chronic Neutropenia Study Group","volume":"90","author":"Donadieu J","year":"2005","journal-title":"Haematologica"},{"key":"e_1_2_7_11_2","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2008.005330"},{"key":"e_1_2_7_12_2","first-page":"715","article-title":"A combined analysis of two pivotal randomized trials of a single dose of pegfilgrastim per chemotherapy cycle and daily Filgrastim in patients with stage II\u2010IV breast cancer","volume":"10","author":"Siena S","year":"2003","journal-title":"Oncol Rep"},{"key":"e_1_2_7_13_2","doi-asserted-by":"publisher","DOI":"10.1200\/JCO.2003.03.040"},{"key":"e_1_2_7_14_2","doi-asserted-by":"publisher","DOI":"10.1097\/CAD.0b013e328011a532"},{"key":"e_1_2_7_15_2","doi-asserted-by":"publisher","DOI":"10.1097\/01.mph.0000188631.41510.23"},{"key":"e_1_2_7_16_2","doi-asserted-by":"publisher","DOI":"10.1097\/01.mph.0000175711.73039.63"},{"key":"e_1_2_7_17_2","doi-asserted-by":"publisher","DOI":"10.1345\/aph.1K093"},{"key":"e_1_2_7_18_2","doi-asserted-by":"publisher","DOI":"10.1111\/j.1537-2995.2007.01241.x"},{"key":"e_1_2_7_19_2","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2141.1994.tb05110.x"},{"key":"e_1_2_7_20_2","doi-asserted-by":"publisher","DOI":"10.1053\/shem.2002.31914"},{"key":"e_1_2_7_21_2","doi-asserted-by":"publisher","DOI":"10.1378\/chest.107.1.276"},{"key":"e_1_2_7_22_2","first-page":"341","article-title":"Spontaneous splenic rupture following administration of granulocyte colony\u2010stimulating factor (G\u2010CSF): Occurrence in an allogeneic donor of peripheral blood stem cells","volume":"3","author":"Brown SL","year":"1997","journal-title":"Biol Blood Marrow Transplant"},{"key":"e_1_2_7_23_2","first-page":"ECR08","article-title":"Spontaneous splenic rupture in a healthy allogeneic donor of peripheral\u2010blood stem cell following the administration of granulocyte colony\u2010stimulating factor (g\u2010csf). A case report and review of the literature","volume":"91","author":"Nuamah NM","year":"2006","journal-title":"Haematologica"},{"key":"e_1_2_7_24_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2005-11-4370"},{"key":"e_1_2_7_25_2","first-page":"176","article-title":"A comparison of pegfilgrastim and filgrastim","volume":"3","author":"Grigg AP","year":"2005","journal-title":"Clin Adv Hematol Oncol"},{"key":"e_1_2_7_26_2","doi-asserted-by":"publisher","DOI":"10.3816\/CBC.2003.n.003"},{"key":"e_1_2_7_27_2","first-page":"529","article-title":"Recombinant human G\u2010CSF (Lenograstim) for infectious complications in glycogen storage disease type Ib. Report of 7 cases","volume":"35","author":"Donadieu J","year":"1994","journal-title":"Nouv Rev Fr Hematol"},{"key":"e_1_2_7_28_2","doi-asserted-by":"publisher","DOI":"10.1080\/10428190500144615"},{"key":"e_1_2_7_29_2","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2141.2005.05839.x"},{"key":"e_1_2_7_30_2","doi-asserted-by":"publisher","DOI":"10.1038\/sj.bmt.1705382"},{"key":"e_1_2_7_31_2","doi-asserted-by":"publisher","DOI":"10.1200\/JCO.2005.04.1012"},{"key":"e_1_2_7_32_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.ajem.2006.10.005"},{"key":"e_1_2_7_33_2","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2257.2006.00819.x"},{"key":"e_1_2_7_34_2","doi-asserted-by":"publisher","DOI":"10.1007\/s00277-007-0364-9"},{"key":"e_1_2_7_35_2","doi-asserted-by":"publisher","DOI":"10.1002\/ajh.20854"}],"container-title":["Pediatric Blood &amp; Cancer"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.wiley.com\/onlinelibrary\/tdm\/v1\/articles\/10.1002%2Fpbc.22147","content-type":"unspecified","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.wiley.com\/onlinelibrary\/tdm\/v1\/articles\/10.1002%2Fpbc.22147","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/pbc.22147","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,10,18]],"date-time":"2023-10-18T18:17:08Z","timestamp":1697653028000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/pbc.22147"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2009,7,17]]},"references-count":34,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2009,12]]}},"alternative-id":["10.1002\/pbc.22147"],"URL":"https:\/\/doi.org\/10.1002\/pbc.22147","archive":["Portico"],"relation":{},"ISSN":["1545-5009","1545-5017"],"issn-type":[{"value":"1545-5009","type":"print"},{"value":"1545-5017","type":"electronic"}],"subject":[],"published":{"date-parts":[[2009,7,17]]}}}