bims-actimu 2025-12-07 papers

Blood Adv. 2025 Dec 04. pii: bloodadvances.2025017662. [Epub ahead of print]

Hematopoietic Cell Transplantation for Wiskott-Aldrich syndrome: A PIDTC Report.

Jessie L Alexander, Blachy J Dávila Saldaña, Ruta Brazauskas, Sravya Gethika Dammalapati, Linda M Griffith, Ami J Shah, Kristin A Shimano, Hans D Ochs, Jack Bleesing, Christen L Ebens, Malika Kapadia, Andrea Bauchat, Neena Kapoor, Joseph H Oved, Hesham Eissa, Hannah Lust, Michael D Keller, Hilary Haines, Shanmuganathan Chandrakasan, Julie-An M Talano, Ahmad Rayes, Lisa M Madden, Evan B Shereck, Holly K Miller, Lisa R Forbes Satter, Caridad A Martinez, Jacob Rozmus, Jeffrey J Bednarski, Lolie C Yu, Deepakbabu Chellapandian, Victor M Aquino, Alan P Knutsen, Hey Chong, Ashley Chopek, Alfred P Gillio, Avni Joshi, Hemalatha G Rangarajan, Theodore B Moore, Jeffrey R Andolina, Kenneth DeSantes, Mark T Vander Lugt, Susan E Prockop, David Shyr, Kathleen E Sullivan, Suhag H Parikh, Katja G Weinacht, Troy R Torgerson, Rebecca A Marsh, Christopher C Dvorak, Alice Y Chan, Elie Haddad, Jennifer Heimall, Michael A Pulsipher, Jennifer W Leiding, Donald B Kohn, Jennifer M Puck, Luigi D Notarangelo, David J Rawlings, Morton J Cowan, Aleksandra Petrovic, Sung-Yun Pai, Lauri M Burroughs.

Wiskott-Aldrich syndrome (WAS), an X-linked disorder characterized by immunodeficiency, thrombocytopenia, autoimmunity, and malignancy, can be effectively treated with allogeneic hematopoietic cell transplantation (HCT). Older age at HCT and mismatched donors are known to impact overall survival (OS). The influence of specific clinical manifestations or WAS variant class on OS and factors associated with event-free survival (EFS) remain incompletely defined. We analyzed outcomes of 308 patients with WAS who underwent HCT at 37 institutions of the Primary Immune Deficiency Treatment Consortium (PIDTC) from 1990-2018. With a median follow-up of 5.3 years, the 5-year OS and EFS were 87.2% and 79.7%, respectively. Age ≥5 years, donor type, and a pre-HCT history of severe infection had a negative impact on OS and EFS, whereas pre-HCT autoimmunity had no impact. Reduced intensity regimens were associated with lower T cell and myeloid donor chimerism, particularly when non-busulfan-based regimens were used. Low myeloid donor chimerism was associated with lower platelet counts. Mixed chimerism was not consistently associated with post-HCT autoimmunity. Patients with class I (exon 1-2 missense and intron 5 hotspot variants) and class II variants (all others) had similar pre-HCT clinical symptom severity and no difference in OS, EFS or platelet recovery post-HCT. In conclusion, our study showed excellent long-term OS and EFS following HCT for WAS, highlighting the importance of early HCT, before the development of severe infections. We confirmed that HCT using busulfan-based conditioning was associated with improved donor chimerism and platelet recovery. This study was registered at www.clinicaltrials.gov as #NCT02064933.

DOI: https://doi.org/10.1182/bloodadvances.2025017662

BMC Immunol. 2025 Dec 04. 26(1): 97

Comprehensive clinical and immunologic characterization of Wiskott-Aldrich syndrome in Iran: a 10-year cohort study.

Hossein Esmaeilzadeh, Mohammad Amin Gholami, Seyed Sina Dehghani, Hesamedin Nabavizadeh, Soheila Alyasin, Nima Rezaei, Samaneh Delavari, Hassan Abolhassani, Farahnaz DorriMoghaddam, Farnia Ghasemi.

Keywords: Clinical manifestations; Cohort study; Immunoglobulin; Immunology profile; WAS; Wiskott-Aldrich

DOI: https://doi.org/10.1186/s12865-025-00779-4

Front Cell Dev Biol. 2025 ;13 1631187

The role of actin cytoskeleton component Arp2/3 in inflammatory response.

Jianxiao Xing, Ying Wang, Yanyang Liang, Jiao Li, Yuanjun Yao, Junqin Li, Kaiming Zhang.

Actin regulatory protein plays an important role in immune-related diseases and affects cellular behavior by regulating the dynamic changes of the cytoskeleton. This regulation is crucial for maintaining the fine balance in the body's biological processes, and can effectively prevent and control the occurrence and development of acute or chronic inflammation, thereby avoiding the appearance of various diseases. The Arp2/3 complex, an evolutionarily conserved molecular machinery, regulates actin cytoskeleton dynamics and nucleates branched actin networks. Upon activation, the Arp2/3 complex binds to the lateral face of pre-existing actin filaments and nucleates daughter filament assembly, generating branched actin networks through this Y-junction formation mechanism. However, the research on how actin is involved in regulating the inflammatory process has only gradually become clear recently. The article mainly summarizes the functions of the actin cytoskeleton, the role of the Arp2/3 complex, and its specific functions in the inflammatory response.

Keywords: ARP2/3; WASP; actin cytoskeleton; cell migration; inflammation

DOI: https://doi.org/10.3389/fcell.2025.1631187