Andolina, Jeffrey R
Mayo Clinic
Department of Pediatrics, Golisano Children's Hospital, University of Rochester Medical Center, Rochester, NY, USA.
Citations 2
Newborn screening reduces survival disparities in SCID after stem cell transplant: A PIDTC report
Winestone, Lena E. , Logan, Brent R. , Liu, Xuerong , Williams, Olatundun , Dunn, Elizabeth A. , Thakar, Monica S. , Kidd, Sharon A. , Dorsey, Morna J. , O'Reilly, R, Kapoor, Neena , Kapadia, Malika , Jyonouchi, Soma , Chandra, Sharat , Chellapandian, Deepak , Chaudhury, Sonali , Chen, Karin , Rayes, Ahmad , Quigg, Troy C. , Teira, Pierre , Gillio, Alfred P. , Eissa, Hesham, Knutsen, Alan P., Aquino, Victor M. , Shereck, Evan , Lugt, Mark T. Vander , Rozmus, Jacob, Yu, Lolie C. , Shah, Ami J. , Joshi, Avni Y. , Buckley, Rebecca H. , Oved, Joseph H. , Azim, Hisham Abdel, Martinez, Caridad , Sullivan, Kathleen E. , Bleesing, Jack J., Madden, Lisa, Shenoy, Shalini , Seroogy, Christine M. , Rubin, Tamar N. , Licata, Jennifer, Andolina, Jeffrey R. , Burroughs, Lauri M. , Leiding, Jennifer W. , Marsh, Rebecca , Pai, Sung‐Yun , Torgerson, Troy R. , Heimall, Jennifer , Griffith, Linda M. , Puck, Jennifer M. , Dvorak, Christopher C. , Cowan, Morton J. , Liu, Xuerong , Mousallem, Talal , Dorsey, Morna J , O'Reilly, Richard J , Kapoor, Neena , Forbes Satter, Lisa R , Kapadia, Malika , Chandra, Sharat , Ebens, Christen L , Chellapandian, Deepakbabu , Chaudhury, Sonali , Chen, Karin , Dávila Saldaña, Blachy J , Rayes, Ahmad , Quigg, Troy C , Chandrakasan, Shanmuganathan , Bednarski, Jeffrey J , DeSantes, Kenneth B , Teira, Pierre , Gillio, Alfred P , Eissa, Hesham , Aquino, Victor M , Shereck, Evan B , Moore, Theodore Bruce , Caywood, Emi H , Vander Lugt, Mark T , Rozmus, Jacob , Broglie, Larisa A , Yu, Lolie C. , Shah, Ami J , Joshi, Avni Y , Buckley, Rebecca H , Dara, Jasmeen , Oved, Joseph H , Abdel Azim, Hisham , Martinez, Caridad , Prockop, Susan E , Sullivan, Kathleen E , Bleesing, Jack J , Keller, Michael D , Madden, Lisa M , Parikh, Suhag , Shenoy, Shalini , Seroogy, Christine M , Rubin, Tamar , Licata, Jennifer , Andolina, Jeffrey R , Kohn, Donald B , Burroughs, Lauri M , Leiding, Jennifer W , Marsh, Rebecca A , Notarangelo, Luigi D , Pai, Sung-Yun , Torgerson, Troy R , Pulsipher, Michael A. , Heimall, Jennifer , Cuvelier, Geoffrey D E , Griffith, Linda M , Puck, Jennifer M , Dvorak, Christopher C , Cowan, Morton J , Haddad, Elie , Winestone, Lena E , Satter, Lisa Forbes , Ebens, Christen L. , Saldaña, Blachy J. Dávila , Chandrakasan, Shanmuganathan , Knutsen, Alan P. , Broglie, Larisa , Dara, Jasmeen , Abdel‐Azim, Hisham , Prockop, Susan E. , Bleesing, Jack , Keller, Michael D. , Parikh, Suhag , Kohn, Donald B. , Cuvelier, Geoff D.E. , Haddad, Élie
Journal of Human Immunity | July 7, 2026 | Volume: 2 | Issue: 5 | Pages: No page available
Black race and Hispanic ethnicity are associated with higher mortality in severe combined immunodeficiency (SCID) following hematopoietic cell transplantation (HCT), though mechanisms remain unclear. We evaluated 796 children with SCID who received nonsibling HCT between 1982 and 2020 using data from the Primary Immune Deficiency Treatment Consortium. Overall survival for Black (aHR 2.47, 95%CI 1.64, 3.71) and Asian/Pacific Islander patients (aHR 1.82, 95%CI 1.00, 3.30) was significantly lower compared with non-Hispanic White patients, while Hispanic patients had lower event-free survival (aHR 1.83, 95%CI 1.27, 2.63) compared with non-Hispanic White patients. Even after adjusting for age and infection, Black patients with SCID had more than twofold hazard of death compared with non-Hispanic White patients. NBS was associated with earlier diagnosis, reduced infection at HCT, and elimination of survival disparities between Black and non-Hispanic White patients. These findings suggest that universal, system-level interventions such as NBS can mitigate disparities in outcomes for children with SCID.
Hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report
Alexander, Jessie L. , Saldaña, Blachy J. Dávila , Brazauskas, Ruta , Dammalapati, Sravya Gethika, Griffith, Linda M. , Shah, Ami J. , Shimano, Kristin A. , Ochs, Hans D., Bleesing, Jack , Ebens, Christen L. , Kapadia, Malika, Bauchat, Andrea , Oved, Joseph H. , Eissa, Hesham, Lust, Hannah , Haines, Hilary , Rayes, Ahmad , Madden, Lisa, Shereck, Evan , Miller, Holly, Martinez, Caridad , Rozmus, Jacob, Yu, Lolie C. , Chellapandian, Deepak , Aquino, Victor M., Knutsen, Alan P. , Chong, Hey , Chopek, Ashley, Gillio, Alfred P. , Joshi, Avni Y. , Rangarajan, Hemalatha G. , Andolina, Jeffrey R. , Lugt, Mark T. Vander , Prockop, Susan E. , Shyr, David C. , Sullivan, Kathleen E. , Weinacht, Katja G. , Torgerson, Troy R. , Marsh, Rebecca , Dvorak, Christopher C. , Heimall, Jennifer , Leiding, Jennifer W. , Puck, Jennifer M. , Rawlings, David J. , Cowan, Morton J. , Petrović, Aleksandra , Pai, Sung‐Yun , Burroughs, Lauri M. , Dávila Saldaña, Blachy J , Oved, Joseph H , Lust, Hannah , Rayes, Ahmad , Forbes Satter, Lisa R , Bednarski, Jeffrey J , Chellapandian, Deepakbabu , Joshi, Avni, Rangarajan, Hemalatha G , Andolina, Jeffrey R , Prockop, Susan E , Parikh, Suhag , Torgerson, Troy R , Chan, Alice Y , Haddad, Elie , Pulsipher, Michael A. , Leiding, Jennifer W , Kohn, Donald B , Puck, Jennifer M , Notarangelo, Luigi D , Pai, Sung-Yun , Burroughs, Lauri M , Ebens, Christen L , Dvorak, Christopher C , Shimano, Kristin A , Shah, Ami J , Ochs, Hans D., Kapoor, Neena R. , Keller, Michael D. , Chandrakasan, Shanmuganathan , Talano, Julie‐An , Satter, Lisa Forbes , Moore, Theodore Bruce , DeSantes, Kenneth B , Parikh, Suhag , Haddad, Élie , Kohn, Donald B.
Blood Advances | December 5, 2025 | Volume: 10 | Issue: 5 | Pages: 1783-1798
10.1182/bloodadvances.2025017662
Abstract 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 affect overall survival (OS). However, 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 from 1990 to 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 hot spot 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 after 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 trial was registered at www.clinicaltrials.gov as NCT02064933.