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1. Intestinal microbiome and metabolome signatures in patients with chronic granulomatous disease.

Author

Chandrasekaran, Prabha, Han, Yu, Zerbe, Christa, Heller, Theo, DeRavin, Suk, Kreuzberg, Samantha, Marciano, Beatriz, Siu, Yik, Jones, Drew, Abraham, Roshini, Stephens, Michael, Tsou, Amy, Snapper, Scott, Conlan, Sean, Subramanian, Poorani, Quinones, Mariam, Grou, Caroline, Calderon, Virginie, Deming, Clayton, Leiding, Jennifer, Arnold, Danielle, Logan, Brent, Griffith, Linda, Petrovic, Aleksandra, Mousallem, Talal, Kapoor, Neena, Heimall, Jennifer, Barnum, Jessie, Kapadia, Malika, Wright, Nicola, Rayes, Ahmad, Chandra, Sharat, Broglie, Larisa, Chellapandian, Deepak, Deal, Christin, Grunebaum, Eyal, Lim, Stephanie, Mallhi, Kanwaldeep, Marsh, Rebecca, Murguia-Favela, Luis, Parikh, Suhag, Touzot, Fabien, Cowan, Morton, Dvorak, Christopher, Haddad, Elie, Kohn, Donald, Notarangelo, Luigi, Pai, Sung-Yun, Puck, Jennifer, Pulsipher, Michael, Torgerson, Troy, Kang, Elizabeth, Malech, Harry, Segre, Julia, Bryant, Clare, Holland, Steven, and Falcone, Emilia

Subjects
CGD, Chronic granulomatous disease, IBD, NADPH oxidase, dysbiosis, inborn errors of immunity, inflammatory bowel disease, intestinal inflammation, metabolome, microbiome, primary immune deficiency, Humans, Granulomatous Disease, Chronic, Gastrointestinal Microbiome, NADPH Oxidases, Cross-Sectional Studies, Inflammatory Bowel Diseases
Abstract

BACKGROUND: Chronic granulomatous disease (CGD) is caused by defects in any 1 of the 6 subunits forming the nicotinamide adenine dinucleotide phosphate oxidase complex 2 (NOX2), leading to severely reduced or absent phagocyte-derived reactive oxygen species production. Almost 50% of patients with CGD have inflammatory bowel disease (CGD-IBD). While conventional IBD therapies can treat CGD-IBD, their benefits must be weighed against the risk of infection. Understanding the impact of NOX2 defects on the intestinal microbiota may lead to the identification of novel CGD-IBD treatments. OBJECTIVE: We sought to identify microbiome and metabolome signatures that can distinguish individuals with CGD and CGD-IBD. METHODS: We conducted a cross-sectional observational study of 79 patients with CGD, 8 pathogenic variant carriers, and 19 healthy controls followed at the National Institutes of Health Clinical Center. We profiled the intestinal microbiome (amplicon sequencing) and stool metabolome, and validated our findings in a second cohort of 36 patients with CGD recruited through the Primary Immune Deficiency Treatment Consortium. RESULTS: We identified distinct intestinal microbiome and metabolome profiles in patients with CGD compared to healthy individuals. We observed enrichment for Erysipelatoclostridium spp, Sellimonas spp, and Lachnoclostridium spp in CGD stool samples. Despite differences in bacterial alpha and beta diversity between the 2 cohorts, several taxa correlated significantly between both cohorts. We further demonstrated that patients with CGD-IBD have a distinct microbiome and metabolome profile compared to patients without CGD-IBD. CONCLUSION: Intestinal microbiome and metabolome signatures distinguished patients with CGD and CGD-IBD, and identified potential biomarkers and therapeutic targets.

Published
2023

2. Measuring the effect of newborn screening on survival after haematopoietic cell transplantation for severe combined immunodeficiency: a 36-year longitudinal study from the Primary Immune Deficiency Treatment Consortium.

Author

Thakar, Monica, Logan, Brent, Puck, Jennifer, Dunn, Elizabeth, Buckley, Rebecca, Cowan, Morton, OReilly, Richard, Kapoor, Neena, Satter, Lisa, Pai, Sung-Yun, Heimall, Jennifer, Chandra, Sharat, Ebens, Christen, Chellapandian, Deepak, Williams, Olatundun, Burroughs, Lauri, Saldana, Blachy, Rayes, Ahmad, Madden, Lisa, Chandrakasan, Shanmuganathan, Bednarski, Jeffrey, DeSantes, Kenneth, Cuvelier, Geoffrey, Teira, Pierre, Gillio, Alfred, Eissa, Hesham, Knutsen, Alan, Goldman, Frederick, Aquino, Victor, Shereck, Evan, Moore, Theodore, Caywood, Emi, Lugt, Mark, Rozmus, Jacob, Broglie, Larisa, Yu, Lolie, Shah, Ami, Andolina, Jeffrey, Liu, Xuerong, Parrott, Roberta, Dara, Jasmeen, Prockop, Susan, Martinez, Caridad, Kapadia, Malika, Jyonouchi, Soma, Sullivan, Kathleen, Bleesing, Jack, Chaudhury, Sonali, Petrovic, Aleksandra, Keller, Michael, Quigg, Troy, Parikh, Suhag, Shenoy, Shalini, Seroogy, Christine, Rubin, Tamar, Decaluwe, Hélène, Routes, John, Torgerson, Troy, Leiding, Jennifer, Pulsipher, Michael, Kohn, Donald, Griffith, Linda, Haddad, Elie, Dvorak, Christopher, and Notarangelo, Luigi

Subjects
Humans, Infant, Newborn, Graft vs Host Disease, Hematopoietic Stem Cell Transplantation, Longitudinal Studies, Neonatal Screening, Proportional Hazards Models, Severe Combined Immunodeficiency
Abstract

BACKGROUND: Severe combined immunodeficiency (SCID) is fatal unless durable adaptive immunity is established, most commonly through allogeneic haematopoietic cell transplantation (HCT). The Primary Immune Deficiency Treatment Consortium (PIDTC) explored factors affecting the survival of individuals with SCID over almost four decades, focusing on the effects of population-based newborn screening for SCID that was initiated in 2008 and expanded during 2010-18. METHODS: We analysed transplantation-related data from children with SCID treated at 34 PIDTC sites in the USA and Canada, using the calendar time intervals 1982-89, 1990-99, 2000-09, and 2010-18. Categorical variables were compared by χ2 test and continuous outcomes by the Kruskal-Wallis test. Overall survival was estimated by the Kaplan-Meier method. A multivariable analysis using Cox proportional hazards regression models examined risk factors for HCT outcomes, including the variables of time interval of HCT, infection status and age at HCT, trigger for diagnosis, SCID type and genotype, race and ethnicity of the patient, non-HLA-matched sibling donor type, graft type, GVHD prophylaxis, and conditioning intensity. FINDINGS: For 902 children with confirmed SCID, 5-year overall survival remained unchanged at 72%-73% for 28 years until 2010-18, when it increased to 87% (95% CI 82·1-90·6; n=268; p=0·0005). For children identified as having SCID by newborn screening since 2010, 5-year overall survival was 92·5% (95% CI 85·8-96·1), better than that of children identified by clinical illness or family history in the same interval (79·9% [69·5-87·0] and 85·4% [71·8-92·8], respectively [p=0·043]). Multivariable analysis demonstrated that the factors of active infection (hazard ratio [HR] 2·41, 95% CI 1·56-3·72; p

Published
2023

3. Effect of Autograft CD34+ Dose on Outcome in Pediatric Patients Undergoing Autologous Hematopoietic Stem Cell Transplant for Central Nervous System Tumors.

Author

Knight, Tristan, Ahn, Kwang, Hebert, Kyle, Atshan, Rasha, Wall, Donna, Chiengthong, Kanhatai, Rotz, Seth, Fraint, Ellen, Rangarajan, Hemalatha, Auletta, Jeffery, Sharma, Akshay, Kitko, Carrie, Hashem, Hasan, Williams, Kirsten, Wirk, Baldeep, Myers, Kasiani, Pulsipher, Michael, Warwick, Anne, Schultz, Kirk, Qayed, Muna, Broglie, Larisa, Eapen, Mary, Yanik, Gregory, Dvorak, Christopher, and Lalefar, Nahal

Subjects
Autograft, Autologous hematopoietic stem cell transplant, CD34+, Central nervous system, Medulloblastoma, TNC, Humans, Child, Retrospective Studies, Autografts, Neoplasm Recurrence, Local, Hematopoietic Stem Cell Transplantation, Antigens, CD34, Central Nervous System Neoplasms
Abstract

Consolidation with autologous hematopoietic stem cell transplantation (HSCT) has improved survival for patients with central nervous system tumors (CNSTs). The impact of the autologous graft CD34+ dose on patient outcomes is unknown. We wanted to analyze the relationship between CD34+ dose, total nucleated cell (TNC) dose, and clinical outcomes, including overall survival (OS), progression-free survival (PFS), relapse, non-relapse mortality (NRM), endothelial-injury complications (EIC), and time to neutrophil engraftment in children undergoing autologous HSCT for CNSTs. A retrospective analysis of the CIBMTR database was performed. Children aged 3.6×106/kg CD34+ cells experienced superior PFS (p = .04) and OS (p = .04) compared to children receiving ≤3.6 × 106/kg. Relapse rates were lower in patients receiving >3.6 × 106/kg CD34+ cells (p = .05). Higher CD34+ doses were not associated with increased NRM (p = .59). Stratification of CD34+ dose by quartile did not reveal any statistically significant differences between quartiles for 3-year PFS (p = .66), OS (p = .29), risk of relapse (p = .57), or EIC (p = .87). There were no significant differences in patient outcomes based on TNC, and those receiving a TNC >4.4 × 108/kg did not experience superior PFS (p = .26), superior OS (p = .14), reduced risk of relapse (p = .37), or reduced NRM (p = .25). Children with medulloblastoma had superior PFS (p < .001), OS (p = .01), and relapse rates (p = .001) compared to those with other CNS tumor types. Median time to neutrophil engraftment was 10 days versus 12 days in the highest and lowest infused CD34+ quartiles, respectively. For children undergoing autologous HSCT for CNSTs, increasing CD34+ cell dose was associated with significantly improved OS and PFS, and lower relapse rates, without increased NRM or EICs.

Published
2023

4. The diagnosis of severe combined immunodeficiency: Implementation of the PIDTC 2022 Definitions.

Author

Pai, Sung-Yun, Kapoor, Neena, Satter, Lisa, Buckley, Rebecca, OReilly, Richard, Chandra, Sharat, Bednarski, Jeffrey, Williams, Olatundun, Rayes, Ahmad, Moore, Theodore, Ebens, Christen, Davila Saldana, Blachy, Petrovic, Aleksandra, Chellapandian, Deepak, Cuvelier, Geoffrey, Vander Lugt, Mark, Caywood, Emi, Chandrakasan, Shanmuganathan, Eissa, Hesham, Goldman, Frederick, Shereck, Evan, Aquino, Victor, Desantes, Kenneth, Madden, Lisa, Miller, Holly, Yu, Lolie, Broglie, Larisa, Gillio, Alfred, Shah, Ami, Knutsen, Alan, Andolina, Jeffrey, Joshi, Avni, Szabolcs, Paul, Kapadia, Malika, Martinez, Caridad, Parrot, Roberta, Sullivan, Kathleen, Prockop, Susan, Abraham, Roshini, Thakar, Monica, Leiding, Jennifer, Haddad, Elie, Heimall, Jennifer, Dunn, Elizabeth, Pulsipher, Michael, Griffith, Linda, Notarangelo, Luigi, Dvorak, Christopher, Puck, Jennifer, Cowan, Morton, and Kohn, Donald

Subjects
Omenn syndrome, SCID, Severe combined immunodeficiency, leaky/atypical SCID, newborn screening, typical SCID, Infant, Newborn, Humans, Infant, Severe Combined Immunodeficiency, Retrospective Studies, Prospective Studies, Homeodomain Proteins
Abstract

BACKGROUND: Shearer et al in 2014 articulated well-defined criteria for the diagnosis and classification of severe combined immunodeficiency (SCID) as part of the Primary Immune Deficiency Treatment Consortiums (PIDTCs) prospective and retrospective studies of SCID. OBJECTIVE: Because of the advent of newborn screening for SCID and expanded availability of genetic sequencing, revision of the PIDTC 2014 Criteria was needed. METHODS: We developed and tested updated PIDTC 2022 SCID Definitions by analyzing 379 patients proposed for prospective enrollment into Protocol 6901, focusing on the ability to distinguish patients with various SCID subtypes. RESULTS: According to PIDTC 2022 Definitions, 18 of 353 patients eligible per 2014 Criteria were considered not to have SCID, whereas 11 of 26 patients ineligible per 2014 Criteria were determined to have SCID. Of note, very low numbers of autologous T cells (

Published
2023

6. Outcomes after Second Hematopoietic Cell Transplantation in Children and Young Adults with Relapsed Acute Leukemia

Author

Lund, Troy C, Ahn, Kwang Woo, Tecca, Heather R, Hilgers, Megan V, Abdel-Azim, Hisham, Abraham, Allistair, Diaz, Miguel Angel, Badawy, Sherif M, Broglie, Larisa, Brown, Valerie, Dvorak, Christopher C, Gonzalez-Vicent, Marta, Hashem, Hasan, Hayashi, Robert J, Jacobsohn, David A, Kent, Michael W, Li, Chi-Kong, Margossian, Steven P, Martin, Paul L, Mehta, Parinda, Myers, Kasiani, Olsson, Richard, Page, Kristin, Pulsipher, Michael A, Shaw, Peter J, Smith, Angela R, Triplett, Brandon M, Verneris, Michael R, and Eapen, Mary

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Cancer, Transplantation, Hematology, Childhood Leukemia, Regenerative Medicine, Pediatric Cancer, Rare Diseases, Orphan Drug, Pediatric, Acute Disease, Adolescent, Adult, Child, Child, Preschool, Disease-Free Survival, Female, Hematopoietic Stem Cell Transplantation, Humans, Infant, Leukemia, Male, Recurrence, Survival Rate, Time Factors, Young Adult, relapse acute leukemia second transplant, Clinical Sciences, Immunology, Cardiovascular medicine and haematology
Abstract

Children with acute leukemia who relapse after hematopoietic cell transplantation (HCT) have few therapeutic options. We studied 251 children and young adults with acute myelogenous or lymphoblastic leukemia who underwent a second HCT for relapse after their first HCT. The median age at second HCT was 11 years, and the median interval between first and second HCT was 17 months. Most of the patients (n = 187; 75%) were in remission, received a myeloablative conditioning regimen (n = 157; 63%), and underwent unrelated donor HCT (n = 230; 92%). The 2-year probability of leukemia-free survival (LFS) was 33% after transplantation in patients in remission, compared with 19% after transplantation in patients not in remission (P = .02). The corresponding 8-year probabilities were 24% and 10% (P = .003). A higher rate of relapse contributed to the difference in LFS. The 2-year probability of relapse after transplantation was 42% in patients in remission and 56% in those in relapse (P = .05). The corresponding 8-year probabilities were 49% and 64% (P = .04). These data extend the findings of others showing that patients with a low disease burden are more likely to benefit from a second transplantation. Late relapse led to a 10% decrement in LFS beyond the second year after second HCT. This differs from first HCT, in which most relapses occur within 2 years after HCT.

Published
2019

7. Noninfectious Pulmonary Toxicity after Allogeneic Hematopoietic Cell Transplantation

Author

Patel, Sagar S., Ahn, Kwang Woo, Khanal, Manoj, Bupp, Caitrin, Allbee-Johnson, Mariam, Majhail, Navneet S., Hamilton, Betty K., Rotz, Seth J., Hashem, Hasan, Beitinjaneh, Amer, Lazarus, Hillard M., Krem, Maxwell M., Prestidge, Tim, Bhatt, Neel S., Sharma, Akshay, Gadalla, Shahinaz M., Murthy, Hemant S., Broglie, Larisa, Nishihori, Taiga, Freytes, César O., Hildebrandt, Gerhard C., Gergis, Usama, Seo, Sachiko, Wirk, Baldeep, Pasquini, Marcelo C., Savani, Bipin N., Sorror, Mohamed L., Stadtmauer, Edward A., and Chhabra, Saurabh

Published
2022
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8. Impact of Pretransplantation Renal Dysfunction on Outcomes after Allogeneic Hematopoietic Cell Transplantation

Author

Farhadfar, Nosha, Dias, Ajoy, Wang, Tao, Fretham, Caitrin, Chhabra, Saurabh, Murthy, Hemant S., Broglie, Larisa, D'Souza, Anita, Gadalla, Shahinaz M., Gale, Robert Peter, Hashmi, Shahrukh, Al-Homsi, A. Samer, Hildebrandt, Gerhard C., Hematti, Peiman, Rizzieri, David, Chee, Lynette, Lazarus, Hillard M., Bredeson, Christopher, Jaimes, Edgar A., Beitinjaneh, Amer, Bashey, Asad, Prestidge, Tim, Krem, Maxwell M., Marks, David I., Benoit, Stefanie, Yared, Jean A., Nishihori, Taiga, Olsson, Richard F., Freytes, Cesar O., Stadtmauer, Edward, Savani, Bipin N., Sorror, Mohamed L., Ganguly, Siddhartha, Wingard, John R., and Pasquini, Marcelo

Published
2021
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10. Subsequent neoplasms and late mortality in children undergoing allogeneic transplantation for nonmalignant diseases

Author

Kahn, Justine M., Brazauskas, Ruta, Tecca, Heather R., Bo-Subait, Stephanie, Buchbinder, David, Battiwala, Minoo, Flowers, Mary E.D., Savani, Bipin N., Phelan, Rachel, Broglie, Larisa, Abraham, Allistair A., Keating, Amy K., Daly, Andrew, Wirk, Baldeep, George, Biju, Alter, Blanche P., Ustun, Celalettin, Freytes, Cesar O., Beitinjaneh, Amer M., Duncan, Christine, Copelan, Edward, Hildebrandt, Gerhard C., Murthy, Hemant S., Lazarus, Hillard M., Auletta, Jeffery J., Myers, Kasiani C., Williams, Kirsten M., Page, Kristin M., Vrooman, Lynda M., Norkin, Maxim, Byrne, Michael, Diaz, Miguel Angel, Kamani, Naynesh, Bhatt, Neel S., Rezvani, Andrew, Farhadfar, Nosha, Mehta, Parinda A., Hematti, Peiman, Shaw, Peter J., Kamble, Rammurti T., Schears, Raquel, Olsson, Richard F., Hayashi, Robert J., Gale, Robert Peter, Mayo, Samantha J., Chhabra, Saurabh, Rotz, Seth J., Badawy, Sherif M., Ganguly, Siddhartha, Pavletic, Steven, Nishihori, Taiga, Prestidge, Tim, Agrawal, Vaibhav, Hogan, William J., Inamoto, Yoshihiro, Shaw, Bronwen E., and Satwani, Prakash

Published
2020
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11. Effect of Autograft CD34+ Dose on Outcome in Pediatric Patients Undergoing Autologous Hematopoietic Stem Cell Transplant for Central Nervous System Tumors

Author

Knight, Tristan E., Ahn, Kwang Woo, Hebert, Kyle M., Atshan, Rasha, Wall, Donna A., Chiengthong, Kanhatai, Rotz, Seth J., Fraint, Ellen, Rangarajan, Hemalatha G., Auletta, Jeffery J., Sharma, Akshay, Kitko, Carrie L., Hashem, Hasan, Williams, Kirsten M., Wirk, Baldeep, Dvorak, Christopher C., Myers, Kasiani C., Pulsipher, Michael A., Warwick, Anne B., Lalefar, Nahal Rose, Schultz, Kirk R., Qayed, Muna, Broglie, Larisa, Eapen, Mary, and Yanik, Gregory A.

Published
2023
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12. Real-World Data Showing Trends and Outcomes by Race and Ethnicity in Allogeneic Hematopoietic Cell Transplantation: A Report from the Center for International Blood and Marrow Transplant Research

Author

Auletta, Jeffery J., Kou, Jianqun, Chen, Min, Bolon, Yung-Tsi, Broglie, Larisa, Bupp, Caitrin, Christianson, Debra, Cusatis, Rachel N., Devine, Steven M., Eapen, Mary, Hamadani, Mehdi, Hengen, Mary, Lee, Stephanie J., Moskop, Amy, Page, Kristin M., Pasquini, Marcelo C., Perez, Waleska S., Phelan, Rachel, Riches, Marcie L., Rizzo, J. Douglas, Saber, Wael, Spellman, Stephen R., Stefanski, Heather E., Steinert, Patricia, Tuschl, Eileen, Yusuf, Rafeek, Zhang, Mei-Jie, and Shaw, Bronwen E.

Published
2023
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13. Expanded HCT-CI Definitions Capture Comorbidity Better for Younger Patients of Allogeneic HCT for Nonmalignant Diseases

Author

Broglie, Larisa, Friend, Brian D., Chhabra, Saurabh, Logan, Brent R., Bupp, Caitrin, Schiller, Gary, Savani, Bipin N., Stadtmauer, Edward, Abraham, Allistair A., Aljurf, Mahmoud, Badawy, Sherif M., Perez, Miguel Angel Diaz, Guinan, Eva C., Hashem, Hasan, Krem, Maxwell M., Lazarus, Hillard M., Rotz, Seth J., Wirk, Baldeep, Yared, Jean A., Pasquini, Marcelo, Thakar, Monica S., and Sorror, Mohamed L.

Published
2023
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14. Adapting the HCT-CI Definitions for Children, Adolescents, and Young Adults with Hematologic Malignancies Undergoing Allogeneic Hematopoietic Cell Transplantation

Author

Friend, Brian D., Broglie, Larisa, Logan, Brent R., Chhabra, Saurabh, Bupp, Caitrin, Schiller, Gary, Beitinjaneh, Amer, Perez, Miguel Angel Diaz, Guilcher, Gregory M.T., Hashem, Hasan, Hildebrandt, Gerhard C., Krem, Maxwell M., Lazarus, Hillard M., Nishihori, Taiga, Nusrat, Roomi, Rotz, Seth J., Wirk, Baldeep, Wieduwilt, Matthew, Pasquini, Marcelo, Savani, Bipin N., Stadtmauer, Edward A., Sorror, Mohamed L., and Thakar, Monica S.

Published
2023
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18. Genotype, Oxidase Status, and Preceding Infection or Autoinflammation Do Not Affect Allogeneic HCT Outcomes for CGD

Author

Leiding, Jennifer W, primary, Arnold, Danielle E, additional, Parikh, Suhag H, additional, Logan, Brent R, additional, Marsh, Rebecca A, additional, Griffith, Linda M, additional, Wu, Ruizhe, additional, Kidd, Sharon, additional, Mallhi, Kanwaldeep K, additional, Chellapandian, Deepak, additional, Si Lim, Stephanie, additional, Grunebaum, Eyal, additional, Falcone, Emilia Liana, additional, Murguia-Favela, Luis, additional, Grossman, Debbi, additional, Prasad, Vinod K, additional, Heimall, Jennifer R, additional, Touzot, Fabien, additional, Burroughs, Lauri, additional, Bleesing, Jack, additional, Kapoor, Neena, additional, Dara, Jasmeen, additional, Williams, Olatundun, additional, Kapadia, Malika, additional, Oshrine, Benjamin R, additional, Bednarski, Jeffrey J, additional, Rayes, Ahmad, additional, Chong, Hey, additional, Cuvelier, Geoffrey D.E., additional, Forbes Satter, Lisa R, additional, Martinez, Caridad, additional, Vander Lugt, Mark T, additional, Yu, Lolie C, additional, Chandrakasan, Shanmuganathan, additional, Joshi, Avni, additional, Prockop, Susan E., additional, Dávila Saldaña, Blachy J, additional, Aquino, Victor, additional, Broglie, Larisa, additional, Ebens, Christen L, additional, Madden, Lisa M., additional, DeSantes, Kenneth, additional, Milner, Jordan, additional, Rangarajan, Hemalatha G, additional, Shah, Ami, additional, Gilio, Alfred P, additional, Knutsen, Alan P, additional, Miller, Holly K, additional, Moore, Theodore B, additional, Graham, Pamela, additional, Bauchat, Andrea, additional, Bunin, Nancy J., additional, Teira, Pierre, additional, Petrovic, Aleksandra, additional, Chandra, Sharat, additional, Abdel-Azim, Hisham, additional, Dorsey, Morna, additional, Birbrayer, Olga, additional, Cowan, Morton J, additional, Dvorak, Christopher C, additional, Haddad, Elie, additional, Kohn, Donald B, additional, Notarangelo, Luigi D, additional, Pai, Sung-Yun, additional, Puck, Jennifer M., additional, Pulsipher, Michael A, additional, Torgerson, Troy R., additional, Malech, Harry L, additional, and Kang, Elizabeth M, additional

Published
2023
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19. Updated Trends in Hematopoietic Cell Transplantation in the United States with an Additional Focus on Adolescent and Young Adult Transplantation Activity and Outcomes

Author

Phelan, Rachel, Chen, Min, Bupp, Caitrin, Bolon, Yung-Tsi, Broglie, Larisa, Brunner-Grady, Janet, Burns, Linda J., Chhabra, Saurabh, Christianson, Debra, Cusatis, Rachel, Devine, Steven M., D'Souza, Anita, Eapen, Mary, Hamadani, Mehdi, Hengen, Mary, Lee, Stephanie J., Moskop, Amy, Page, Kristin M., Pasquini, Marcelo, Pérez, Waleska S., Riches, Marcie, Rizzo, Doug, Saber, Wael, Spellman, Stephen R., Stefanski, Heather E., Steinert, Patricia, Weisdorf, Daniel, Horowitz, Mary, Auletta, Jeffery J., Shaw, Bronwen E., and Arora, Mukta

Published
2022
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21. Allogenic Hematopoietic Cell Transplantations Are Effective in Patients with p47phox Chronic Granulomatous Disease: A Primary Immune Deficiency Treatment Consortium Study

Author

Grunebaum, Eyal, primary, Arnold, Danielle E., additional, Logan, Brent, additional, Parikh, Suhag, additional, Marsh, Rebecca A., additional, Griffith, Linda M., additional, Mallhi, Kanwaldeep, additional, Chellapandian, Deepak, additional, Lim, Stephanie Si, additional, Deal, Christin L., additional, Murguía-Favela, Luis, additional, Mousallem, Talal I., additional, Prasad, Prof. Vinod K., additional, Teira, Pierre, additional, Touzot, Fabien, additional, Bunin, Nancy J., additional, Heimall, Jennifer R., additional, Burroughs, Lauri M., additional, Kapadia, Malika, additional, Prockop, Susan, additional, Chandra, Sharat, additional, Chandrakasan, Shanmuganathan, additional, Chaudhury, Sonali, additional, Broglie, Larisa, additional, O’Reilly, Richard J., additional, Dávila Saldaña, Blachy J., additional, Schaefer, Edo, additional, Chong, Hey, additional, Bednarski, Jeffrey J., additional, Rayes, Ahmad, additional, DeSantes, Kenneth, additional, Kohn, Donald B., additional, Notarangelo, Luigi D., additional, Pai, Sung-Yun, additional, Puck, Jennifer, additional, Torgerson, Troy, additional, Cowan, Morton J., additional, Dvorak, Christopher C., additional, Satter, Lisa Forbes, additional, Haddad, Elie, additional, Pulsipher, Michael, additional, Malech, Harry L., additional, Kang, Elizabeth M., additional, and Leiding, Jennifer W., additional

Published
2023
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22. Access: A Multi-Center, Phase II Trial of HLA-Mismatched Unrelated Donor Hematopoietic Cell Transplantation with Post-Transplantation Cyclophosphamide for Patients with Hematologic Malignancies

Author

Al Malki, Monzr M., primary, Shaffer, Brian C., additional, Devine, Steven M., additional, Shaw, Bronwen E., additional, Broglie, Larisa, additional, Qayed, Muna, additional, Choi, Sung Won, additional, Spellman, Stephen, additional, Malmberg, Craig, additional, Ndifon, Eric, additional, Logan, Brent, additional, Auletta, Jeffery James, additional, Stefanski, Heather E., additional, Olson, Janelle, additional, and Jimenez, Antonio Jimenez, additional

Published
2023
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25. The impacts of total body irradiation on umbilical cord blood hematopoietic stem cell transplantation

Author

Wang, Hao, primary, Berger, Kristin N., additional, Miller, Elizabeth L., additional, Fu, Wei, additional, Broglie, Larisa, additional, Goldman, Frederick D., additional, Konig, Heiko, additional, Lim, Su Jin, additional, Berg, Arthur S., additional, Talano, Julie-An, additional, Comito, Melanie A., additional, Farag, Sherif S., additional, and Pu, Jeffrey J., additional

Published
2023
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26. Access: A Multi-Center, Phase II Trial of HLA-Mismatched Unrelated Donor Hematopoietic Cell Transplantation with Post-Transplantation Cyclophosphamide for Patients with Hematologic Malignancies

Author

Al Malki, Monzr M., primary, Devine, Steven M, additional, Shaw, Bronwen E., additional, Broglie, Larisa, additional, Qayed, Muna, additional, Choi, Sung W., additional, Spellman, Stephen R., additional, Malmberg, Craig, additional, Ndifon, Eric, additional, Logan, Brent, additional, Auletta, Jeffery, additional, Stefanski, Heather E, additional, Olson, Janelle A., additional, and Jimenez Jimenez, Antonio, additional

Published
2022
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27. sj-docx-1-tah-10.1177_20406207231170708 – Supplemental material for The impacts of total body irradiation on umbilical cord blood hematopoietic stem cell transplantation

Author

Wang, Hao, Berger, Kristin N., Miller, Elizabeth L., Fu, Wei, Broglie, Larisa, Goldman, Frederick D., Konig, Heiko, Lim, Su Jin, Berg, Arthur S., Talano, Julie-An, Comito, Melanie A., Farag, Sherif S., and Pu, Jeffrey J.

Subjects
FOS: Clinical medicine, Cardiology, 111599 Pharmacology and Pharmaceutical Sciences not elsewhere classified, 111299 Oncology and Carcinogenesis not elsewhere classified
Abstract

Supplemental material, sj-docx-1-tah-10.1177_20406207231170708 for The impacts of total body irradiation on umbilical cord blood hematopoietic stem cell transplantation by Hao Wang, Kristin N. Berger, Elizabeth L. Miller, Wei Fu, Larisa Broglie, Frederick D. Goldman, Heiko Konig, Su Jin Lim, Arthur S. Berg, Julie-An Talano, Melanie A. Comito, Sherif S. Farag and Jeffrey J. Pu in Therapeutic Advances in Hematology

Published
2023
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29. 125 - Hematopoietic Cell Transplantation in 240 Patients with Chronic Granulomatous Disease: A Pidtc Report

Author

Arnold, Danielle E., Parikh, Suhag, Logan, Brent, Marsh, Rebecca, Griffith, Linda M., Wu, Ruizhe, Mallhi, Kanwaldeep, Chellapandian, Deepak, Si, Stephanie, Grunebaum, Eyal, Broglie, Larisa, Prasad, Vinod K., Heimall, Jennifer, Bunin, Nancy J., Teira, Pierre, Touzot, Fabien, Burroughs, Lauri M., Petrovic, Aleksandra, Bleesing, Jack J., Chandra, Sharat, Kapoor, Neena, Dara, Jasmeen, Dorsey, Morna, Williams, Olatundun, Kapadia, Malika, Bednarski, Jeffrey, Rayes, Ahmad, Chen, Karin, Chong, Hey, Cuvelier, Geoff D.E., Forbes, Lisa R., Martinez, Caridad, Vander Lugt, Mark T., Yu, Lolie C., Chandrakasan, Shanmuganathan, Joshi, Avni, Prockop, Susan E., Davila Saldana, Blachy J., Aquino, Victor, Ebens, Christen L., Madden, Lisa, DeSantes, Kenneth, Milner, Jordan, Rangarajan, Hemalatha G., Shah, Ami J, Gillio, Alfred P., Knutsen, Alan P., Miller, Holly K., Moore, Theodore B., Wright, Nicola, Cowan, Morton J., Dvorak, Christopher C., Haddad, Elie, Kohn, Donald B., Notarangelo, Luigi D., Pai, Sung-Yun, Puck, Jennifer, Pulsipher, Mike A., Torgerson, Troy, Malech, Harry L., Kang, Elizabeth M., and Leiding, Jennifer W.

Published
2022
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31. Trends in Late Mortality Amongst Two-Year Survivors of Pediatric and Young Adult Allogeneic Hematopoietic Cell Transplantation for Acute Leukemias: On Behalf of the CIBMTR Late Effects Working Committee

Author

Broglie, Larisa, primary, Bo-Subait, Stephanie, additional, Chen, Yue, additional, Brazauskas, Ruta, additional, Buchbinder, David, additional, Hamilton, Betty K., additional, Schoemans, Helene M., additional, Phelan, Rachel, additional, and Satwani, Prakash, additional

Published
2022
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34. A Primary Immune Deficiency Treatment Consortium (PIDTC) Study of Chronic and Late Onset Medical Complications after Initial Hematopoietic Cell Transplantation (HCT) for Severe Combined Immunodeficiency Disease (SCID)

Author

Eissa, Hesham, primary, Thakar, Monica S., additional, Shah, Ami J, additional, Buckley, Rebecca H., additional, Logan, Brent, additional, Griffith, Linda M., additional, Dong, Huaying, additional, O’Reilly, Richard J., additional, Kapoor, Neena, additional, Satter, Lisa Forbes, additional, Chandra, Sharat, additional, Bleesing, Jack J., additional, Kapadia, Malika, additional, Parrott, Roberta E, additional, Chandrakasan, Shanmuganathan, additional, Bednarski II, Jeffrey J., additional, Jyonouchi, Soma, additional, Madden, Lisa M., additional, Rayes, Ahmad, additional, Ebens, Christen L., additional, Teira, Pierre, additional, Dávila Saldaña, Blachy J, additional, Burroughs, Lauri M., additional, Prockop, Susan E., additional, Williams, Olatundun, additional, Chellapandian, Deepak, additional, Gillio, Alfred P., additional, Goldman, Frederick, additional, Malech, Harry L., additional, DeSantes, Kenneth, additional, Cuvelier, Geoff D.E., additional, Rozmus, Jacob, additional, Quinones, Ralph, additional, Yu, Lolie C., additional, Broglie, Larisa, additional, Aquino, Victor, additional, Shereck, Evan, additional, Moore, Theodore B., additional, Martinez, Caridad, additional, Vander Lugt, Mark T., additional, Leiding, Jennifer W., additional, Torgerson, Troy, additional, Pai, Sung-Yun, additional, Pulsipher, Mike A., additional, Notarangelo, Luigi D., additional, Puck, Jennifer, additional, Dvorak, Christopher C., additional, Haddad, Elie, additional, Cowan, Morton J., additional, and Heimall, Jennifer, additional

Published
2022
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36. Diagnostic work‐up for severe aplastic anemia in children: Consensus of the North American Pediatric Aplastic Anemia Consortium

Author

Shimano, Kristin A., primary, Narla, Anupama, additional, Rose, Melissa J., additional, Gloude, Nicholas J., additional, Allen, Steven W., additional, Bergstrom, Katie, additional, Broglie, Larisa, additional, Carella, Beth A., additional, Castillo, Paul, additional, Jong, Jill L. O., additional, Dror, Yigal, additional, Geddis, Amy E., additional, Huang, James N., additional, Lau, Bonnie W., additional, McGuinn, Catherine, additional, Nakano, Taizo A., additional, Overholt, Kathleen, additional, Rothman, Jennifer A., additional, Sharathkumar, Anjali, additional, Shereck, Evan, additional, Vlachos, Adrianna, additional, Olson, Timothy S., additional, Bertuch, Alison A., additional, Wlodarski, Marcin W., additional, Shimamura, Akiko, additional, and Boklan, Jessica, additional

Published
2021
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37. Delirium in Children Undergoing Hematopoietic Cell Transplantation: A Multi-Institutional Point Prevalence Study

Author

Traube, Chani, primary, Gerber, Linda M., additional, Mauer, Elizabeth A., additional, Small, Keshia, additional, Broglie, Larisa, additional, Chopra, Yogi Raj, additional, Duncan, Christine N., additional, Ebens, Christen L., additional, Fitzgerald, Julie C., additional, Freedman, Jason L., additional, Hudspeth, Michelle P., additional, Hurley, Caitlin, additional, Mahadeo, Kris M., additional, McArthur, Jennifer, additional, Shapiro, Miriam C., additional, Sharron, Matthew P., additional, Wall, Donna A., additional, Zinter, Matt S., additional, Greenwald, Bruce M., additional, Silver, Gabrielle, additional, and Boulad, Farid, additional

Published
2021
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38. Expanded Comorbidity Definitions Improve Applicability of the Hematopoietic Stem Cell Transplantation-Comorbidity Index for Children, Adolescents, and Young Adults with Hematologic Malignancies Undergoing Allogeneic Stem Cell Transplantation

Author

Friend, Brian D, primary, Broglie, Larisa, additional, Logan, Brent, additional, Fretham, Caitrin, additional, Schiller, Gary J., additional, Savani, Bipin N., additional, Stadtmauer, Edward, additional, Chhabra, Saurabh, additional, Pasquini, Marcelo C., additional, Thakar, Monica, additional, and Sorror, Mohamed, additional

Published
2020
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39. Expanded Comorbidity Definitions Improve Application of the Hematopoietic Cell Transplantation Comorbidity Index (HCT-CI) for Children and Young Adults with Non-Malignant Diseases Receiving Allogeneic Hematopoietic Cell Transplantation

Author

Broglie, Larisa, primary, Friend, Brian D, additional, Logan, Brent, additional, Fretham, Caitrin, additional, Schiller, Gary J., additional, Savani, Bipin N., additional, Stadtmauer, Edward A., additional, Chhabra, Saurabh, additional, Pasquini, Marcelo C., additional, Thakar, Monica, additional, and Sorror, Mohamed, additional

Published
2020
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40. 382 - Allogenic Hematopoietic Cell Transplantations Are Effective in Patients with p47phox Chronic Granulomatous Disease: A Primary Immune Deficiency Treatment Consortium Study

Author

Grunebaum, Eyal, Arnold, Danielle E., Logan, Brent, Parikh, Suhag, Marsh, Rebecca A., Griffith, Linda M., Mallhi, Kanwaldeep, Chellapandian, Deepak, Lim, Stephanie Si, Deal, Christin L., Murguía-Favela, Luis, Mousallem, Talal I., Prasad, Prof. Vinod K., Teira, Pierre, Touzot, Fabien, Bunin, Nancy J., Heimall, Jennifer R., Burroughs, Lauri M., Kapadia, Malika, Prockop, Susan, Chandra, Sharat, Chandrakasan, Shanmuganathan, Chaudhury, Sonali, Broglie, Larisa, O’Reilly, Richard J., Dávila Saldaña, Blachy J., Schaefer, Edo, Chong, Hey, Bednarski, Jeffrey J., II, Rayes, Ahmad, DeSantes, Kenneth, Kohn, Donald B., Notarangelo, Luigi D., Pai, Sung-Yun, Puck, Jennifer, Torgerson, Troy, Cowan, Morton J., Dvorak, Christopher C., Satter, Lisa Forbes, Haddad, Elie, Pulsipher, Michael, Malech, Harry L., Kang, Elizabeth M., and Leiding, Jennifer W.

Published
2023
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41. 308 - Access: A Multi-Center, Phase II Trial of HLA-Mismatched Unrelated Donor Hematopoietic Cell Transplantation with Post-Transplantation Cyclophosphamide for Patients with Hematologic Malignancies

Author

Al Malki, Monzr M., Shaffer, Brian C., Devine, Steven M., Shaw, Bronwen E., Broglie, Larisa, Qayed, Muna, Choi, Sung Won, Spellman, Stephen, Malmberg, Craig, Ndifon, Eric, Logan, Brent, Auletta, Jeffery James, Stefanski, Heather E., Olson, Janelle, and Jimenez, Antonio Jimenez

Published
2023
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46. 437 - A Primary Immune Deficiency Treatment Consortium (PIDTC) Study of Chronic and Late Onset Medical Complications after Initial Hematopoietic Cell Transplantation (HCT) for Severe Combined Immunodeficiency Disease (SCID)

Author

Eissa, Hesham, Thakar, Monica S., Shah, Ami J, Buckley, Rebecca H., Logan, Brent, Griffith, Linda M., Dong, Huaying, O’Reilly, Richard J., Kapoor, Neena, Satter, Lisa Forbes, Chandra, Sharat, Bleesing, Jack J., Kapadia, Malika, Parrott, Roberta E, Chandrakasan, Shanmuganathan, Bednarski II, Jeffrey J., Jyonouchi, Soma, Madden, Lisa M., Rayes, Ahmad, Ebens, Christen L., Teira, Pierre, Dávila Saldaña, Blachy J, Burroughs, Lauri M., Prockop, Susan E., Williams, Olatundun, Chellapandian, Deepak, Gillio, Alfred P., Goldman, Frederick, Malech, Harry L., DeSantes, Kenneth, Cuvelier, Geoff D.E., Rozmus, Jacob, Quinones, Ralph, Yu, Lolie C., Broglie, Larisa, Aquino, Victor, Shereck, Evan, Moore, Theodore B., Martinez, Caridad, Vander Lugt, Mark T., Leiding, Jennifer W., Torgerson, Troy, Pai, Sung-Yun, Pulsipher, Mike A., Notarangelo, Luigi D., Puck, Jennifer, Dvorak, Christopher C., Haddad, Elie, Cowan, Morton J., and Heimall, Jennifer

Published
2022
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