{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,12,29]],"date-time":"2025-12-29T05:54:42Z","timestamp":1766987682680,"version":"3.48.0"},"reference-count":29,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2025,12,10]],"date-time":"2025-12-10T00:00:00Z","timestamp":1765324800000},"content-version":"vor","delay-in-days":9,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["eJHaem"],"published-print":{"date-parts":[[2025,12]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>Severe congenital neutropenia (SCN) is commonly treated with granulocyte colony\u2010stimulating factor (G\u2010CSF) to reduce neutropenia and the associated risk of infections. Pegfilgrastim, a long\u2010acting form of G\u2010CSF, provides the benefit of less frequent dosing; however, its application in SCN has not been extensively studied. This study aims to assess the long\u2010term safety and effectiveness of transitioning SCN patients from standard G\u2010CSF therapy to pegfilgrastim.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>We followed eight patients with severe congenital neutropenia (five males, average age 17.8 years) who had been on G\u2010CSF treatment for at least five years. They were switched to pegfilgrastim injections (3\u20136\u00a0mg every 7\u201314 days). Dosing was individualized to obtain an absolute neutrophil count (ANC) above 1000\/\u00b5L in all patients. Over a five\u2010year follow\u2010up under treatment with pegfilgrastim, patients were evaluated for ANC levels, infection frequency, adverse effects, and quality of life using the Functional Assessment of Cancer Therapy\u2010Neutropenia (FACT\u2010N) questionnaire. In addition, yearly abdominal ultrasounds, bone density every two years, and genetic screening for RUNX1 and the G\u2010CSF receptor mutations were performed.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      Pegfilgrastim significantly improved ANC levels compared to prior G\u2010CSF treatment (\n                      <jats:italic>p\u00a0=\u00a0<\/jats:italic>\n                      0.008). Quality of life (QoL) significantly improved in six patients (\n                      <jats:italic>p\u00a0=\u00a0<\/jats:italic>\n                      0.016). Varying maintenance dosages were required based on the therapeutic response. Bone pain was common, leading to discontinuation in one patient. Two patients developed hip osteopenia, and one showed progressive splenomegaly. Hospitalization rates and infection frequency remained unchanged. Oral ulcers were decreased overall.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>Pegfilgrastim can be used as an alternative to daily G\u2010CSF in SCN, providing stable ANC and QoL improvements with individualized dosing. Larger studies are warranted to evaluate its long\u2010term safety and efficacy.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Clinical trial registration<\/jats:title>\n                    <jats:p>\n                      This trial is registered at\n                      <jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" xlink:href=\"https:\/\/irct.behdasht.gov.ir\/\">https:\/\/irct.behdasht.gov.ir\/<\/jats:ext-link>\n                      in Iranian Registry of Clinical Trials (IRCT) # IRCT20150125020786N3.\n                    <\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/jha2.70198","type":"journal-article","created":{"date-parts":[[2025,12,10]],"date-time":"2025-12-10T14:32:20Z","timestamp":1765377140000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Efficacy and Safety of Pegfilgrastim in Patients With Severe Congenital Neutropenia"],"prefix":"10.1002","volume":"6","author":[{"given":"Mahdiyeh","family":"Movahedi","sequence":"first","affiliation":[{"name":"Immunology, Asthma and Allergy Research Institute Tehran University of Medical Sciences  Tehran Iran"},{"name":"Children's Medical Center Pediatrics Center of Excellence Tehran University of Medical Sciences  Tehran Iran"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Leila","family":"Moradi","sequence":"additional","affiliation":[{"name":"Immunology, Asthma and Allergy Research Institute Tehran University of Medical Sciences  Tehran Iran"},{"name":"Children's Medical Center Pediatrics Center of Excellence Tehran University of Medical Sciences  Tehran Iran"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Zahra","family":"Alizadeh","sequence":"additional","affiliation":[{"name":"Immunology, Asthma and Allergy Research Institute Tehran University of Medical Sciences  Tehran Iran"},{"name":"Children's Medical Center Pediatrics Center of Excellence Tehran University of Medical Sciences  Tehran Iran"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mojtaba Tabatabaei","family":"Yazdi","sequence":"additional","affiliation":[{"name":"Department of Pharmaceutical Biotechnology Faculty of Pharmacy Tehran University of Medical Sciences  Tehran Iran"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Shirin","family":"Dehbashi","sequence":"additional","affiliation":[{"name":"Department of Pharmaceutical Biotechnology Faculty of Pharmacy Tehran University of Medical Sciences  Tehran Iran"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Raheleh Shokouhi","family":"Shoormasti","sequence":"additional","affiliation":[{"name":"Immunology, Asthma and Allergy Research Institute Tehran University of Medical Sciences  Tehran Iran"},{"name":"Children's Medical Center Pediatrics Center of Excellence Tehran University of Medical Sciences  Tehran Iran"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Marzieh","family":"Maddah","sequence":"additional","affiliation":[{"name":"Clinical Research Development Center Mahdieh Educational Hospital Shahid Beheshti University of Medical Sciences  Tehran Iran"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Anahita","family":"Razaghian","sequence":"additional","affiliation":[{"name":"Immunology, Asthma and Allergy Research Institute Tehran University of Medical Sciences  Tehran Iran"},{"name":"Department of Pediatrics Division of Allergy and Clinical Immunology Hakim Children's Hospital Tehran University of Medical Sciences Tehran Iran"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Amir Ali","family":"Hamidieh","sequence":"additional","affiliation":[{"name":"Pediatric Cell and Gene Therapy Research Center Gene, Cell &amp; Tissue Research Institute Tehran University of Medical Sciences  Tehran Iran"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mansoureh","family":"Shariat","sequence":"additional","affiliation":[{"name":"Department of Immunology and Allergy Children Medical Center Tehran University of Medical Sciences  Tehran Iran"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Massoud","family":"Houshmand","sequence":"additional","affiliation":[{"name":"Department of Medical Genetics National Institute of Genetic Engineering and Biotechnology (NIGEB)  Tehran Iran"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Atefe","family":"Alirezaee","sequence":"additional","affiliation":[{"name":"Immunology, Asthma and Allergy Research Institute Tehran University of Medical Sciences  Tehran Iran"},{"name":"Children's Medical Center Pediatrics Center of Excellence Tehran University of Medical Sciences  Tehran Iran"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Maryam","family":"Akbari","sequence":"additional","affiliation":[{"name":"Immunology, Asthma and Allergy Research Institute Tehran University of Medical Sciences  Tehran Iran"},{"name":"Children's Medical Center Pediatrics Center of Excellence Tehran University of Medical Sciences  Tehran Iran"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mohammad Reza","family":"Fazlollahi","sequence":"additional","affiliation":[{"name":"Immunology, Asthma and Allergy Research Institute Tehran University of Medical Sciences  Tehran Iran"},{"name":"Children's Medical Center Pediatrics Center of Excellence Tehran University of Medical Sciences  Tehran Iran"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Zahra","family":"Pourpak","sequence":"additional","affiliation":[{"name":"Immunology, Asthma and Allergy Research Institute Tehran University of Medical Sciences  Tehran Iran"},{"name":"Children's Medical Center Pediatrics Center of Excellence Tehran University of Medical Sciences  Tehran Iran"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2025,12,10]]},"reference":[{"key":"e_1_2_13_2_1","doi-asserted-by":"publisher","DOI":"10.1111\/pai.12654"},{"key":"e_1_2_13_3_1","doi-asserted-by":"publisher","DOI":"10.1038\/nrdp.2017.32"},{"key":"e_1_2_13_4_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V96.7.2317"},{"issue":"1","key":"e_1_2_13_5_1","first-page":"86","article-title":"Different Pattern of Gene Mutations in Iranian Patients With Severe Congenital Neutropenia (Including 2 New Mutations)","volume":"12","author":"Alizadeh Z.","year":"2013","journal-title":"Iranian Journal of Allergy, Asthma and Immunology"},{"key":"e_1_2_13_6_1","doi-asserted-by":"publisher","DOI":"10.1002\/pbc.22350"},{"key":"e_1_2_13_7_1","doi-asserted-by":"publisher","DOI":"10.1002\/pbc.22147"},{"issue":"17","key":"e_1_2_13_8_1","first-page":"2178","article-title":"Long\u2010Term Use of Pegfilgrastim in Children With Severe Congenital Neutropenia: Clinical and Pharmacokinetic Data. 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