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1. Effect of rabbit ATG PK on outcomes after TCR-αβ/CD19-depleted pediatric haploidentical HCT for hematologic malignancy.

Author

Dvorak, Christopher, Long-Boyle, Janel, Holbrook-Brown, Lucia, Abdel-Azim, Hisham, Bertaina, Alice, Vatsayan, Anant, Talano, Julie-An, Bunin, Nancy, Anderson, Eric, Flower, Allyson, Lalefar, Nahal, Higham, Christine, Kapoor, Neena, Klein, Orly, Odinakachukwu, Maryanne, Cho, Soohee, Jacobsohn, David, Collier, Willem, and Pulsipher, Michael

Subjects
Adolescent, Adult, Animals, Child, Child, Preschool, Female, Humans, Infant, Male, Rabbits, Young Adult, Antigens, CD19, Antilymphocyte Serum, Hematologic Neoplasms, Hematopoietic Stem Cell Transplantation, Receptors, Antigen, T-Cell, alpha-beta, Transplantation, Haploidentical, Treatment Outcome
Abstract

We hypothesized that the inferior disease-free survival (DFS) seen in older patients who underwent αβ-T-cell/CD19-depleted (AB-TCD) haploidentical hematopoietic cell transplantation (HCT) for hematologic malignancies is caused by excessive exposure to rabbit antithymocyte globulin (rATG; Thymoglobulin). Between 2015 and 2023, 163 patients with a median age of 13 years (range, 0.4-27.4) underwent AB-TCD haploidentical HCT for the treatment of acute lymphoblastic leukemia (n = 98), acute myeloid leukemia/myelodysplastic syndrome (n = 49), or other malignancies (n = 16) at 9 centers in 2 prospective trials. Exposures to rATG before and after HCT were predicted using a validated pharmacokinetic model. Receiver operating characteristic curves were used to identify the optimal target windows for rATG exposure in terms of outcomes. We identified 4 quadrants of rATG exposure, namely quadrant 1 (n = 52) with a high pre-HCT area under curve (AUC; ≥50 arbitrary units [AU] per day per milliliter) and a low post-HCT AUC (

Published
2024

3. Genotype, oxidase status, and preceding infection or autoinflammation do not affect allogeneic HCT outcomes for CGD

Author

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

Published
2023
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4. Infections in Infants with SCID: Isolation, Infection Screening, and Prophylaxis in PIDTC Centers.

Author

Dorsey, Morna J, Wright, Nicola AM, Chaimowitz, Natalia S, Dávila Saldaña, Blachy J, Miller, Holly, Keller, Michael D, Thakar, Monica S, Shah, Ami J, Abu-Arja, Rolla, Andolina, Jeffrey, Aquino, Victor, Barnum, JL, Bednarski, Jeffrey J, Bhatia, Monica, Bonilla, Francisco A, Butte, Manish J, Bunin, Nancy J, Chandra, Sharat, Chaudhury, Sonali, Chen, Karin, Chong, Hey, Cuvelier, Geoffrey DE, Dalal, Jignesh, DeFelice, Magee L, DeSantes, Kenneth B, Forbes, Lisa R, Gillio, Alfred, Goldman, Fred, Joshi, Avni Y, Kapoor, Neena, Knutsen, Alan P, Kobrynski, Lisa, Lieberman, Jay A, Leiding, Jennifer W, Oshrine, Benjamin, Patel, Kiran P, Prockop, Susan, Quigg, Troy C, Quinones, Ralph, Schultz, Kirk R, Seroogy, Christine, Shyr, David, Siegel, Subhadra, Smith, Angela R, Torgerson, Troy R, Vander Lugt, Mark T, Yu, Lolie C, Cowan, Morton J, Buckley, Rebecca H, Dvorak, Christopher C, Griffith, Linda M, Haddad, Elie, Kohn, Donald B, Logan, Brent, Notarangelo, Luigi D, Pai, Sung-Yun, Puck, Jennifer, Pulsipher, Michael A, and Heimall, Jennifer

Subjects
Humans, Severe Combined Immunodeficiency, Disease Susceptibility, Neonatal Screening, Prognosis, Antibiotic Prophylaxis, Hematopoietic Stem Cell Transplantation, Age of Onset, Infection Control, Infant, Infant, Newborn, Disease Management, Female, Male, Public Health Surveillance, Time-to-Treatment, Surveys and Questionnaires, Clinical Decision-Making, Infections, hematopoietic stem cell transplant, newborn screening, primary immunodeficiency, prophylaxis, severe combined immunodeficiency, Stem Cell Research, Perinatal Period - Conditions Originating in Perinatal Period, Pediatric, Prevention, Regenerative Medicine, Transplantation, Infant Mortality, Health Services, Clinical Research, Infection, Immunology
Abstract

PurposeThe Primary Immune Deficiency Treatment Consortium (PIDTC) enrolled children with severe combined immunodeficiency (SCID) in a prospective natural history study of hematopoietic stem cell transplant (HSCT) outcomes over the last decade. Despite newborn screening (NBS) for SCID, infections occurred prior to HSCT. This study's objectives were to define the types and timing of infection prior to HSCT in patients diagnosed via NBS or by family history (FH) and to understand the breadth of strategies employed at PIDTC centers for infection prevention.MethodsWe analyzed retrospective data on infections and pre-transplant management in patients with SCID diagnosed by NBS and/or FH and treated with HSCT between 2010 and 2014. PIDTC centers were surveyed in 2018 to understand their practices and protocols for pre-HSCT management.ResultsInfections were more common in patients diagnosed via NBS (55%) versus those diagnosed via FH (19%) (p = 0.012). Outpatient versus inpatient management did not impact infections (47% vs 35%, respectively; p = 0.423). There was no consensus among PIDTC survey respondents as to the best setting (inpatient vs outpatient) for pre-HSCT management. While isolation practices varied, immunoglobulin replacement and antimicrobial prophylaxis were more uniformly implemented.ConclusionInfants with SCID diagnosed due to FH had lower rates of infection and proceeded to HSCT more quickly than did those diagnosed via NBS. Pre-HSCT management practices were highly variable between centers, although uses of prophylaxis and immunoglobulin support were more consistent. This study demonstrates a critical need for development of evidence-based guidelines for the pre-HSCT management of infants with SCID following an abnormal NBS.Trial registrationNCT01186913.

Published
2021

5. Monogenic early-onset lymphoproliferation and autoimmunity: Natural history of STAT3 gain-of-function syndrome

Author

Aleshkevich, Svetlana, Allende, Luis M., Atkinson, T. Prescott, Atschekzei, Faranaz, Aydemir, Sezin, Aygunes, Utku, Barlogis, Vincent, Baumann, Ulrich, Belko, John, Bezrodnik, Liliana, Biebl, Ariane, Broderick, Lori, Bunin, Nancy J., Caldirola, Maria Soledad, Castelle, Martin, Celmeli, Fatih, Charbonnier, Louis-Marie, Chatila, Talal A., Chellapandian, Deepak, Cokugras, Haluk, Conlon, Niall, Cox, Fionnuala, Crickx, Etienne, Dalgic, Buket, ASH Dalm, Virgil, Danielian, Silvia, Dominguez-Pinilla, Nerea, Dujovny, Tal, Ebbo, Mikael, Eken, Ahmet, Esty, Brittany, Fabre, Alexandre, Fischer, Alain, Hannibal, Mark, Huppert, Laura, Ikeda, Marc D., Jolles, Stephen, Jolly, Kent W., Jones, Neil, Kanariou, Maria, Karakoc-Aydiner, Elif, Karamantziani, Theoni, Kelaidi, Charikleia, Keogan, Mary, Pac Kisaarslan, Ayşenur, Kiykim, Ayca, Klocperk, Adam, Kotsonis, Kosmas, Kuzmenko, Natalia, Leroy, Sylvie, Lianou, Dimitra, Longhurst, Hilary, Lorenz, Myriam Ricarda, Maffucci, Patrick, Manson, Ania, Marchal, Sarah, Malphettes, Marion, Marega, Lia Furlaneto, Mauracher, Andrea A., Meesilpavikai, Kornvalee, Miller, Holly, Mombourquette, Joy, Morgan, Noel G., Mukhina, Anna, Nathalie, Aladjidi, Nelken, Brigitte, Nolan, David, Norlin, Anna-Carin, Oleastro, Matias, Ozcan, Alper, Pasquet, Marlene, Pegler, José Roberto, Picard, Capucine, Polychronopoulou, Sophia, Quartier, Pierre, Quesada, Juan Francisco, Ramakers, Jan, Randall, Katrina L., Rao, V. Koneti, Remiker, Allison, Resin, Geraldine, Richmond, Peter, Rieux-Laucat, Frederic, Rodina, Yulia, Rohrlich, Pierre, Sachs, Johnathan, Sakovich, Inga, Santarlas, Christopher, Sari, Sinan, Sawicki, Gregory, Schauer, Uwe, Scheffler Mendoza, Selma C., Schvetz, Oksana, Schmidt, Reinhold Ernst, Schwarz, Klaus, Sediva, Anna, Sinclair, Kyle, Slatter, Mary, Sleasman, John, Stergiou, Katerina, Suratannon, Narissara, Tanita, Kay, Thompson, Grace, Travis, Stephen, Trojan, Timothy, Tsinti, Maria, Unal, Ekrem, Urdinez, Luciano, Vazquez-Gomez, Felisa, Villa, Mariana, Weinrich, Michael, Weiss, Mitchell J., Wright, Benjamin, Yilmaz, Ebru, Zachova, Radana, Zhang, Yu, Leiding, Jennifer W., Vogel, Tiphanie P., Santarlas, Valentine G.J., Mhaskar, Rahul, Smith, Madison R., Carisey, Alexandre, Vargas-Hernández, Alexander, Silva-Carmona, Manuel, Heeg, Maximilian, Rensing-Ehl, Anne, Neven, Bénédicte, Hadjadj, Jérôme, Hambleton, Sophie, Ronan Leahy, Timothy, Cunningham-Rundles, Charlotte, Dutmer, Cullen M., Sharapova, Svetlana O., Taskinen, Mervi, Chua, Ignatius, Hague, Rosie, Klemann, Christian, Kostyuchenko, Larysa, Morio, Tomohiro, Thatayatikom, Akaluck, Ozen, Ahmet, Scherbina, Anna, Bauer, Cindy S., Flanagan, Sarah E., Gambineri, Eleonora, Giovannini-Chami, Lisa, Heimall, Jennifer, Sullivan, Kathleen E., Allenspach, Eric, Romberg, Neil, Deane, Sean G., Prince, Benjamin T., Rose, Melissa J., Bohnsack, John, Mousallem, Talal, Jesudas, Rohith, Santos Vilela, Maria Marluce Dos, O’Sullivan, Michael, Pachlopnik Schmid, Jana, Průhová, Štěpánka, Rees, Matthew, Su, Helen, Bahna, Sami, Baris, Safa, Bartnikas, Lisa M., Chang Berger, Amy, Briggs, Tracy A., Brothers, Shannon, Bundy, Vanessa, Chan, Alice Y., Chandrakasan, Shanmuganathan, Christiansen, Mette, Cole, Theresa, Cook, Matthew C., Desai, Mukesh M., Fischer, Ute, Fulcher, David A., Gallo, Silvanna, Gauthier, Amelie, Gennery, Andrew R., Gonçalo Marques, José, Gottrand, Frédéric, Grimbacher, Bodo, Grunebaum, Eyal, Haapaniemi, Emma, Hämäläinen, Sari, Heiskanen, Kaarina, Heiskanen-Kosma, Tarja, Hoffman, Hal M., Gonzalez-Granado, Luis Ignacio, Guerrerio, Anthony L., Kainulainen, Leena, Kumar, Ashish, Lawrence, Monica G., Levin, Carina, Martelius, Timi, Neth, Olaf, Olbrich, Peter, Palma, Alejandro, Patel, Niraj C., Pozos, Tamara, Preece, Kahn, Lugo Reyes, Saúl Oswaldo, Russell, Mark A., Schejter, Yael, Seroogy, Christine, Sinclair, Jan, Skevofilax, Effie, Suan, Daniel, Suez, Daniel, Szabolcs, Paul, Velasco, Helena, Warnatz, Klaus, Walkovich, Kelly, Worth, Austen, Seppänen, Mikko R.J., Torgerson, Troy R., Sogkas, Georgios, Ehl, Stephan, Tangye, Stuart G., Cooper, Megan A., Milner, Joshua D., and Forbes Satter, Lisa R.

Published
2023
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6. Higher Risks of Toxicity and Incomplete Recovery in 13- to 17-Year-Old Females after Marrow Donation: RDSafe Peds Results.

Author

Pulsipher, Michael A, Logan, Brent R, Kiefer, Deidre M, Chitphakdithai, Pintip, Riches, Marcie L, Rizzo, J Douglas, Anderlini, Paolo, Leitman, Susan F, Varni, James W, Kobusingye, Hati, Besser, RaeAnne M, Miller, John P, Drexler, Rebecca J, Abdel-Mageed, Aly, Ahmed, Ibrahim A, Ball, Edward D, Bolwell, Brian J, Bunin, Nancy J, Cheerva, Alexandra, Delgado, David C, Dvorak, Christopher C, Gillio, Alfred P, Hahn, Theresa E, Hale, Gregory A, Haight, Ann E, Hayes-Lattin, Brandon M, Kasow, Kimberly A, Linenberger, Michael, Magalhaes-Silverman, Margarida, Mori, Shahram, Prasad, Vinod K, Quigg, Troy C, Sahdev, Indira, Schriber, Jeffrey R, Shenoy, Shalini, Tse, William T, Yanik, Gregory A, Navarro, Willis H, Horowitz, Mary M, Confer, Dennis L, Shaw, Bronwen E, and Switzer, Galen E

Subjects
Humans, Pain, Tissue and Organ Harvesting, Bone Marrow Transplantation, Transplantation, Homologous, Age Factors, Sex Factors, Time Factors, Adolescent, Tissue Donors, Female, Male, BM collection toxicities, Donor safety, PBSC collection toxicities, Stem cell transplantation, Transplantation, Chronic Pain, Neurosciences, Pediatric, Clinical Research, Stem Cell Research, Pain Research, Clinical Trials and Supportive Activities, Prevention, Immunology, Clinical Sciences
Abstract

Although donation of bone marrow (BM) or peripheral blood stem cells (PBSCs) from children to family members undergoing allogeneic transplantation are well-established procedures, studies detailing levels of pain, symptoms, and long-term recovery are lacking. To address this lack, we prospectively enrolled 294 donors age 20% of females and males age 13 to 17 years do not return to baseline pain levels by 1 year after BM donation. Studies aimed at decreasing symptoms and improving recovery in older children are warranted.

Published
2019

7. Effect of Aging and Predonation Comorbidities on the Related Peripheral Blood Stem Cell Donor Experience: Report from the Related Donor Safety Study.

Author

Pulsipher, Michael A, Logan, Brent R, Chitphakdithai, Pintip, Kiefer, Deidre M, Riches, Marcie L, Rizzo, J Douglas, Anderlini, Paolo, Leitman, Susan F, Varni, James W, Kobusingye, Hati, Besser, RaeAnne M, Miller, John P, Drexler, Rebecca J, Abdel-Mageed, Aly, Ahmed, Ibrahim A, Akard, Luke P, Artz, Andrew S, Ball, Edward D, Bayer, Ruthee-Lu, Bigelow, Carolyn, Bolwell, Brian J, Broun, E Randolph, Bunin, Nancy J, Delgado, David C, Duckworth, Katharine, Dvorak, Christopher C, Hahn, Theresa E, Haight, Ann E, Hari, Parameswaran N, Hayes-Lattin, Brandon M, Jacobsohn, David A, Jakubowski, Ann A, Kasow, Kimberly A, Lazarus, Hillard M, Liesveld, Jane L, Linenberger, Michael, Litzow, Mark R, Longo, Walter, Magalhaes-Silverman, Margarida, McCarty, John M, McGuirk, Joseph P, Mori, Shahram, Prasad, Vinod K, Rowley, Scott D, Rybka, Witold B, Sahdev, Indira, Schriber, Jeffrey R, Selby, George B, Shaughnessy, Paul J, Shenoy, Shalini, Spitzer, Thomas, Tse, William T, Uberti, Joseph P, Vusirikala, Madhuri, Waller, Edmund K, Weisdorf, Daniel J, Yanik, Gregory A, Navarro, Willis H, Horowitz, Mary M, Switzer, Galen E, Shaw, Bronwen E, and Confer, Dennis L

Subjects
Humans, Peripheral Blood Stem Cell Transplantation, Comorbidity, Adolescent, Adult, Aged, Middle Aged, Blood Donors, Female, Male, Young Adult, Peripheral Blood Stem Cells, BM collection toxicities, Donor safety, PBSC collection toxicities, Stem cell transplantation, Transplantation, Pain Research, Stem Cell Research - Nonembryonic - Human, Clinical Research, Chronic Pain, Stem Cell Research, Aging, Clinical Sciences, Immunology
Abstract

The development of reduced-intensity approaches for allogeneic hematopoietic cell transplantation has resulted in growing numbers of older related donors (RDs) of peripheral blood stem cells (PBSCs). The effects of age on donation efficacy, toxicity, and long-term recovery in RDs are poorly understood. To address this we analyzed hematologic variables, pain, donation-related symptoms, and recovery in 1211 PBSC RDs aged 18 to 79 enrolled in the Related Donor Safety Study. RDs aged > 60 had a lower median CD34+ level before apheresis compared with younger RDs (age > 60, 59 × 106/L; age 41 to 60, 81 × 106/L; age 18 to 40, 121 × 106/L; P < .001). This resulted in older donors undergoing more apheresis procedures (49% versus 30% ≥ 2 collections, P < .001) and higher collection volumes (52% versus 32% > 24 L, P < .001), leading to high percentages of donors aged > 60 with postcollection thrombocytopenia

Published
2019

9. Variables affecting outcomes after allogeneic hematopoietic stem cell transplant for cerebral adrenoleukodystrophy

Author

Chiesa, Robert, Boelens, Jaap Jan, Duncan, Christine N., Kühl, Jörn-Sven, Sevin, Caroline, Kapoor, Neena, Prasad, Vinod K., Lindemans, Caroline A., Jones, Simon A., Amartino, Hernan M., Algeri, Mattia, Bunin, Nancy, Diaz-de-Heredia, Cristina, Loes, Daniel J., Shamir, Esther, Timm, Alison, McNeil, Elizabeth, Dietz, Andrew C., and Orchard, Paul J.

Published
2022
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12. 106 Outcomes Following Hematopoietic Cell Transplant for CD3δ Severe Combined Immune Deficiency: a PIDTC Natural History Study

Author

Kalashnikova, Tatiana, primary, Cuvelier, Geoff, additional, Logan, Brent, additional, Grunebaum, Eyal, additional, Guilcher, Gregory, additional, Lewis, Victor, additional, McAuley, Grace, additional, Marwaha, Ashish, additional, Murguia-Favela, Luis, additional, Prince, Bradley, additional, Romero, Zulema, additional, Rubin, Tamar, additional, Suresh, Sneha, additional, Buckley, Rebecca, additional, Dvorak, Christopher, additional, Satter, Lisa, additional, Aquino, Victor, additional, Bunin, Nancy, additional, Joshi, Avni, additional, Prokop, Susan, additional, Heimall, Jennifer, additional, Haddad, Elie, additional, Leiding, Jennifer, additional, Puck, Jennifer, additional, Burroughs, Lauri, additional, Griffith, Linda, additional, Notarangelo, Luigi, additional, Pulsipher, Michael, additional, Cowan, Morton, additional, Marsh, Rebecca, additional, Pai, Sung-Yun, additional, Torgerson, Troy, additional, Kohn, Donald B., additional, and Wright, Nicola, additional

Published
2024
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13. Reduced-Intensity/Reduced-Toxicity Conditioning Approaches Are Tolerated in XIAP Deficiency but Patients Fare Poorly with Acute GVHD

Author

Arnold, Danielle E., Nofal, Rofida, Wakefield, Connor, Lehmberg, Kai, Wustrau, Katharina, Albert, Michael H., Morris, Emma C., Heimall, Jennifer R., Bunin, Nancy J., Kumar, Ashish, Jordan, Michael B., Cole, Theresa, Choo, Sharon, Brettig, Tim, Speckmann, Carsten, Ehl, Stephan, Salamonowicz, Malgorzata, Wahlstrom, Justin, Rao, Kanchan, Booth, Claire, Worth, Austen, and Marsh, Rebecca A.

Published
2022
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14. Immune Dysregulation in Human ITCH Deficiency Successfully Treated with Hematopoietic Cell Transplantation

Author

Patel, Trusha, Henrickson, Sarah E., Moser, Emily K., Field, Natania S., Maurer, Kelly, Dawany, Noor, Conrad, Maire, Bunin, Nancy, Freedman, Jason L., Heimall, Jennifer, Arnold, Danielle E., Wang, Jing, Markowitz, Jonathan E., Payne-Poff, Sarah Beth, Williams, Kelli W., Russo, Pierre A., Wherry, E. John, Devoto, Marcella, Oliver, Paula, Sullivan, Kathleen E., and Kelsen, Judith R.

Published
2021
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15. Long-term outcomes of 176 patients with X-linked hyper-IgM syndrome treated with or without hematopoietic cell transplantation.

Author

de la Morena, M Teresa, Leonard, David, Torgerson, Troy R, Cabral-Marques, Otavio, Slatter, Mary, Aghamohammadi, Asghar, Chandra, Sharat, Murguia-Favela, Luis, Bonilla, Francisco A, Kanariou, Maria, Damrongwatanasuk, Rongras, Kuo, Caroline Y, Dvorak, Christopher C, Meyts, Isabelle, Chen, Karin, Kobrynski, Lisa, Kapoor, Neena, Richter, Darko, DiGiovanni, Daniela, Dhalla, Fatima, Farmaki, Evangelia, Speckmann, Carsten, Español, Teresa, Shcherbina, Anna, Hanson, Imelda Celine, Litzman, Jiri, Routes, John M, Wong, Melanie, Fuleihan, Ramsay, Seneviratne, Suranjith L, Small, Trudy N, Janda, Ales, Bezrodnik, Liliana, Seger, Reinhard, Raccio, Andrea Gomez, Edgar, J David M, Chou, Janet, Abbott, Jordan K, van Montfrans, Joris, González-Granado, Luis Ignacio, Bunin, Nancy, Kutukculer, Necil, Gray, Paul, Seminario, Gisela, Pasic, Srdjan, Aquino, Victor, Wysocki, Christian, Abolhassani, Hassan, Dorsey, Morna, Cunningham-Rundles, Charlotte, Knutsen, Alan P, Sleasman, John, Costa Carvalho, Beatriz Tavares, Condino-Neto, Antonio, Grunebaum, Eyal, Chapel, Helen, Ochs, Hans D, Filipovich, Alexandra, Cowan, Mort, Gennery, Andrew, Cant, Andrew, Notarangelo, Luigi D, and Roifman, Chaim M

Subjects
Humans, Hematopoietic Stem Cell Transplantation, Proportional Hazards Models, Retrospective Studies, Cohort Studies, Follow-Up Studies, Time, Adolescent, Adult, Middle Aged, Child, Child, Preschool, Infant, Female, Male, Hyper-IgM Immunodeficiency Syndrome, Young Adult, Kaplan-Meier Estimate, CD40 ligand, Karnofsky/Lansky scores, X-linked hyper-IgM syndrome, defects in class-switch recombination, hematopoietic cell transplantation, long-term outcomes, primary immunodeficiency, Pediatric, Clinical Research, Transplantation, Inflammatory and immune system, Good Health and Well Being, Immunology, Allergy
Abstract

BackgroundX-linked hyper-IgM syndrome (XHIGM) is a primary immunodeficiency with high morbidity and mortality compared with those seen in healthy subjects. Hematopoietic cell transplantation (HCT) has been considered a curative therapy, but the procedure has inherent complications and might not be available for all patients.ObjectivesWe sought to collect data on the clinical presentation, treatment, and follow-up of a large sample of patients with XHIGM to (1) compare long-term overall survival and general well-being of patients treated with or without HCT along with clinical factors associated with mortality and (2) summarize clinical practice and risk factors in the subgroup of patients treated with HCT.MethodsPhysicians caring for patients with primary immunodeficiency diseases were identified through the Jeffrey Modell Foundation, United States Immunodeficiency Network, Latin American Society for Immunodeficiency, and Primary Immune Deficiency Treatment Consortium. Data were collected with a Research Electronic Data Capture Web application. Survival from time of diagnosis or transplantation was estimated by using the Kaplan-Meier method compared with log-rank tests and modeled by using proportional hazards regression.ResultsTwenty-eight clinical sites provided data on 189 patients given a diagnosis of XHIGM between 1964 and 2013; 176 had valid follow-up and vital status information. Sixty-seven (38%) patients received HCT. The average follow-up time was 8.5 ± 7.2 years (range, 0.1-36.2 years). No difference in overall survival was observed between patients treated with or without HCT (P = .671). However, risk associated with HCT decreased for diagnosis years 1987-1995; the hazard ratio was significantly less than 1 for diagnosis years 1995-1999. Liver disease was a significant predictor of overall survival (hazard ratio, 4.9; 95% confidence limits, 2.2-10.8; P

Published
2017

17. Partially CD3+-Depleted Unrelated and Haploidentical Donor Peripheral Stem Cell Transplantation Has Favorable Graft-versus-Host Disease and Survival Rates in Pediatric Hematologic Malignancy

Author

Seif, Alix E., Li, Yimei, Monos, Dimitri S., Heidemann, Stephanie C., Aplenc, Richard, Barrett, David M., Casper, James T., Freedman, Jason L., Grupp, Stephan A., Margolis, David A., Olson, Timothy S., Teachey, David T., Keever-Taylor, Carolyn A., Wang, Yongping, Talano, Julie-An M., and Bunin, Nancy J.

Published
2020
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18. Tabelecleucel for EBV+ PTLD following allogeneic HCT or SOT in a multicenter expanded access protocol

Author

Nikiforow, Sarah, primary, Whangbo, Jennifer S, additional, Reshef, Ran, additional, Tsai, Donald E, additional, Bunin, Nancy J., additional, Abu-Arja, Rolla F, additional, Mahadeo, Kris Michael, additional, Weng, Wen-Kai, additional, Van Besien, Koen, additional, Loeb, David, additional, Nasta, Sunita D., additional, Nemecek, Eneida R., additional, Zhao, Weizhi, additional, Sun, Yan, additional, Galderisi, Faith C., additional, Wahlstrom, Justin, additional, Mehta, Aditi, additional, Gamelin, Laurence Isabelle, additional, Dinavahi, Rajani, additional, and Prockop, Susan E., additional

Published
2024
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19. Pilot Study of Unrelated Donor (URD) Peripheral Stem Cell Transplant (PSCT) with CD45RA-Depleted Addback Following TCRab/CD19 Depletion in Pediatric Patients with Hematologic Malignancies

Author

Elgarten, Caitlin W., primary, Heidemann, Stephanie, additional, Freedman, Jason L., additional, Grupp, Stephan A., additional, Hankins, Patricia, additional, Kadauke, Stephan, additional, Li, Yimei, additional, McGlynn, Barbara, additional, Olson, Timothy S., additional, Wang, Yongping, additional, Wray, Lisa, additional, Wang, Lei, additional, Seif, Alix E., additional, and Bunin, Nancy J., additional

Published
2024
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20. Outcomes Following Matched Sibling Donor Transplant for Severe Combined Immunodeficiency: A Report from the Pidtc

Author

Rayes, Ahmad, primary, Logan, Brent, additional, Liu, Xuerong, additional, Dara, Jasmeen, additional, Buckley, Rebecca H., additional, Oved, Joseph H., additional, Kapoor, Neena, additional, Kapadia, Malika, additional, Chandra, Sharat, additional, Martinez, Caridad, additional, Bunin, Nancy J., additional, Chandrakasan, Shanmuganathan, additional, Burroughs, Lauri M., additional, Bednarski, Jeffrey J, additional, Haines, Hilary, additional, Cuvelier, Geoff D.E., additional, Eissa, Hesham, additional, Talano, Julie A, additional, Saldana, Blachy J, additional, Ebens, Christen L., additional, Chaudhury, Sonali, additional, Shereck, Evan, additional, Aquino, Victor, additional, Knutsen, Alan P., additional, Alexander, Jessie L, additional, Gillio, Alfred P., additional, Chellapandian, Deepak, additional, Shah, Ami J., additional, Miller, Holly K., additional, Lugt, Mark T. Vander, additional, DeSantes, Kenneth, additional, Dorsey, Morna, additional, Parrott, Roberta E, additional, O'Reilly, Richard J., additional, Aguayo-Hiraldo, Paibel, additional, Torgerson, Troy, additional, Leiding, Jennifer W., additional, Marsh, Rebecca A., additional, Griffith, Linda M., additional, Pulsipher, Mike A., additional, Kohn, Donald B., additional, Notarangelo, Luigi D., additional, Cowan, Morton J., additional, Puck, Jennifer, additional, Heimall, Jennifer R., additional, Haddad, Elie, additional, Pai, Sung-Yun, additional, and Dvorak, Christopher C., additional

Published
2024
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21. Optimal Rabbit Antithymocyte Pharmacokinetics (PK) Is Associated with Improved Disease-Free Survival (DFS) in TCR-Αβ/CD19-Depleted Pediatric Haploidentical Hematopoietic Cell Transplantation for Hematologic Malignancies

Author

Dvorak, Christopher C., primary, Holbrook-Brown, Lucia, additional, Long-Boyle, Janel Renee, additional, Abdel-Azim, Hisham, additional, Bertaina, Alice, additional, Vatsayan, Anant, additional, Talano, Julie A, additional, Bunin, Nancy J., additional, Anderson, Eric J, additional, Flower, Allyson, additional, Lalefar, Nahal, additional, Higham, Christine S., additional, Kapoor, Neena, additional, Odinakachukwu, Maryanne A, additional, Jacobsohn, David A, additional, and Pulsipher, Mike A., additional

Published
2024
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22. Outcomes of Unrelated Partial T Cell Depleted Stem Cell Transplantation for Patients with Hemoglobinopathies

Author

Gibson, Nora M., primary, Elgarten, Caitlin W., additional, Oved, Joseph H., additional, Wray, Lisa, additional, Freedman, Jason L., additional, Wang, Yongping, additional, Kadauke, Stephan, additional, Hankins, Patricia, additional, Nicholas, Peter, additional, Worster, Elizabeth, additional, Kwiatkowski, Janet L., additional, Grupp, Stephan A., additional, Bunin, Nancy J., additional, and Olson, Timothy S., additional

Published
2024
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23. Unrelated and Haploidentical Peripheral Stem Cell Transplant with Tcrαβ/CD19 Depletion for the Treatment of Pediatric Patients with Inborn Errors of Immunity

Author

McClory, Susan E., primary, Elgarten, Caitlin W., additional, Quijano, Kaitlyn N, additional, Wohlschlaeger, Anne, additional, Hankins, Mrs. Patricia, additional, Wang, Lei, additional, Wang, Yongping, additional, Kadauke, Stephan, additional, Monos, Dimitri, additional, Olson, Timothy S., additional, Grupp, Stephan A., additional, Ochfeld, Elisa, additional, Heimall, Jennifer R., additional, and Bunin, Nancy J., additional

Published
2024
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24. Adenoviral Cellular Therapy with Adenovirus (ADV)-Specific Cytotoxic T-Lymphocytes (CTLs) for Resistant Infections in Children, Adolescent, and Young Adult (CAYA) Patients Following Allogeneic Hematopoietic Stem Cell Transplantation (AlloHSCT): A Promising Approach

Author

Schaefer, Edo, primary, Vu, Christine, additional, Flower, Allyson, additional, Ayello, Janet, additional, Chu, Yaya, additional, van de Ven, Carmella, additional, Mintzer, Elizabeth, additional, Harrison, Lauren, additional, Morris, Erin, additional, Wang, Yongping, additional, Shenoy, Shalini, additional, Lee, Dean Anthony, additional, Abu-Arja, Rolla, additional, Johnson, Bryon D, additional, Verneris, Michael R., additional, Dvorak, Christopher C., additional, Chu, Julia, additional, Cooke, Kenneth R., additional, Talano, Julie A, additional, Bunin, Nancy J., additional, and Cairo, Mitchell S., additional

Published
2024
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27. Phase II/III trial of a pre‐transplant farnesyl transferase inhibitor in juvenile myelomonocytic leukemia: A report from the Children's Oncology Group

Author

Stieglitz, Elliot, Ward, Ashley F, Gerbing, Robert B, Alonzo, Todd A, Arceci, Robert J, Liu, Y Lucy, Emanuel, Peter D, Widemann, Brigitte C, Cheng, Jennifer W, Jayaprakash, Nalini, Balis, Frank M, Castleberry, Robert P, Bunin, Nancy J, Loh, Mignon L, and Cooper, Todd M

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Oncology and Carcinogenesis, Clinical Trials and Supportive Activities, Rare Diseases, Pediatric, Pediatric Cancer, Transplantation, Cancer, Hematology, Clinical Research, Childhood Leukemia, Stem Cell Research, Evaluation of treatments and therapeutic interventions, 6.1 Pharmaceuticals, 6.2 Cellular and gene therapies, Adult, Aged, Aged, 80 and over, Antineoplastic Combined Chemotherapy Protocols, Child, Child, Preschool, Cytarabine, Disease-Free Survival, Enzyme Inhibitors, Farnesyl-Diphosphate Farnesyltransferase, Female, Hematopoietic Stem Cell Transplantation, Humans, Infant, Isotretinoin, Leukemia, Myelomonocytic, Juvenile, Male, Middle Aged, Quinolones, Survival Rate, Vidarabine, 13-cis retinoic acid, farnesyl transferase inhibitor, hematopoietic stem cell transplant, juvenile myelomonocytic leukemia, tipifarnib, Clinical Sciences, Paediatrics and Reproductive Medicine, Oncology & Carcinogenesis, Oncology and carcinogenesis, Paediatrics
Abstract

BackgroundJuvenile myelomonocytic leukemia (JMML) is not durably responsive to chemotherapy, and approximately 50% of patients relapse after hematopoietic stem cell transplant (HSCT). Here we report the activity and acute toxicity of the farnesyl transferase inhibitor tipifarnib, the response rate to 13-cis retinoic acid (CRA) in combination with cytoreductive chemotherapy, and survival following HSCT in children with JMML.ProcedureEighty-five patients with newly diagnosed JMML were enrolled on AAML0122 between 2001 and 2006. Forty-seven consented to receive tipifarnib in a phase II window before proceeding to a phase III trial of CRA in combination with fludarabine and cytarabine followed by HSCT and maintenance CRA. Thirty-eight patients enrolled only in the phase III trial.ResultsOverall response rate was 51% after tipifarnib and 68% after fludarabine/cytarabine/CRA. Tipifarnib did not increase pre-transplant toxicities. Forty-six percent of the 44 patients who received protocol compliant HSCT relapsed. Five-year overall survival was 55 ± 11% and event-free survival was 41 ± 11%, with no significant difference between patients who did or did not receive tipifarnib.ConclusionsAdministration of tipifarnib in the window setting followed by HSCT in patients with newly diagnosed JMML was safe and yielded a 51% initial response rate as a single agent, but failed to reduce relapse rates or improve long-term overall survival.

Published
2015

30. Correction to: Infections in Infants with SCID: Isolation, Infection Screening and Prophylaxis in PIDTC Centers

Author

Dorsey, Morna, Wright, Nicola A. M., Chaimowitz, Natalia S., Dávila Saldaña, Blachy J., Miller, Holly, Keller, Michael D., Thakar, Monica S., Shah, Ami J., Abu-Arja, Rolla, Andolina, Jeffrey, Aquino, Victor, Barnum, J. L., Bednarski, Jeffrey J., Bhatia, Monica, Bonilla, Francisco A., Butte, Manish J., Bunin, Nancy J., Burroughs, Lauri M., Chandra, Sharat, Chaudhury, Sonali, Chen, Karin, Chong, Hey, Cuvelier, Geoff, Dalal, Jignesh, DeFelice, Magee L., DeSantes, Kenneth B., Forbes, Lisa R., Gillio, Alfred, Goldman, Fred, Joshi, Avni Y., Kapoor, Neena, Knutsen, Alan P., Kobrynski, Lisa, Lieberman, Jay A., Leiding, Jennifer W., Oshrine, Benjamin, Patel, Kiran P., Prockop, Susan, Quigg, Troy C., Quinones, Ralph, Schultz, Kirk R., Seroogy, Christine, Shyr, David, Siegel, Subhadra, Smith, Angela R., Torgerson, Troy R., Vander Lugt, Mark T., Yu, Lolie C., Cowan, Morton J., Buckley, Rebecca H., Dvorak, Christopher C., Griffith, Linda M., Haddad, Elie, Kohn, Donald B., Logan, Brent, Notarangelo, Luigi D., Pai, Sung-Yun, Puck, Jennifer, Pulsipher, Michael A., and Heimall, Jennifer

Published
2021
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31. One-Unit versus Two-Unit Cord-Blood Transplantation for Hematologic Cancers

Author

Wagner, John E, Eapen, Mary, Carter, Shelly, Wang, Yanli, Schultz, Kirk R, Wall, Donna A, Bunin, Nancy, Delaney, Colleen, Haut, Paul, Margolis, David, Peres, Edward, Verneris, Michael R, Walters, Mark, Horowitz, Mary M, and Kurtzberg, Joanne

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Immunology, Hematology, Stem Cell Research - Nonembryonic - Human, Cancer, Stem Cell Research, Prevention, Clinical Research, Regenerative Medicine, Transplantation, Stem Cell Research - Umbilical Cord Blood/ Placenta, Clinical Trials and Supportive Activities, Rare Diseases, Adolescent, Child, Child, Preschool, Cord Blood Stem Cell Transplantation, Disease-Free Survival, Female, Graft vs Host Disease, Hematologic Neoplasms, Histocompatibility Testing, Humans, Immunotherapy, Infant, Male, Survival Rate, Transplantation Conditioning, Young Adult, Blood and Marrow Transplant Clinical Trials Network, Medical and Health Sciences, General & Internal Medicine, Biomedical and clinical sciences, Health sciences
Abstract

BackgroundUmbilical-cord blood has been used as the source of hematopoietic stem cells in an estimated 30,000 transplants. The limited number of hematopoietic cells in a single cord-blood unit prevents its use in recipients with larger body mass and results in delayed hematopoietic recovery and higher mortality. Therefore, we hypothesized that the greater numbers of hematopoietic cells in two units of cord blood would be associated with improved outcomes after transplantation.MethodsBetween December 1, 2006, and February 24, 2012, a total of 224 patients 1 to 21 years of age with hematologic cancer were randomly assigned to undergo double-unit (111 patients) or single-unit (113 patients) cord-blood transplantation after a uniform myeloablative conditioning regimen and immunoprophylaxis for graft-versus-host disease (GVHD). The primary end point was 1-year overall survival.ResultsTreatment groups were matched for age, sex, self-reported race (white vs. nonwhite), performance status, degree of donor-recipient HLA matching, and disease type and status at transplantation. The 1-year overall survival rate was 65% (95% confidence interval [CI], 56 to 74) and 73% (95% CI, 63 to 80) among recipients of double and single cord-blood units, respectively (P=0.17). Similar outcomes in the two groups were also observed with respect to the rates of disease-free survival, neutrophil recovery, transplantation-related death, relapse, infections, immunologic reconstitution, and grade II-IV acute GVHD. However, improved platelet recovery and lower incidences of grade III and IV acute and extensive chronic GVHD were observed among recipients of a single cord-blood unit.ConclusionsWe found that among children and adolescents with hematologic cancer, survival rates were similar after single-unit and double-unit cord-blood transplantation; however, a single-unit cord-blood transplant was associated with better platelet recovery and a lower risk of GVHD. (Funded by the National Heart, Lung, and Blood Institute and the National Cancer Institute; ClinicalTrials.gov number, NCT00412360.).

Published
2014

32. Comparison of outcomes of hematopoietic stem cell transplantation without chemotherapy conditioning by using matched sibling and unrelated donors for treatment of severe combined immunodeficiency

Author

Dvorak, Christopher C, Hassan, Amel, Slatter, Mary A, Hönig, Manfred, Lankester, Arjan C, Buckley, Rebecca H, Pulsipher, Michael A, Davis, Jeffrey H, Güngör, Tayfun, Gabriel, Melissa, Bleesing, Jacob H, Bunin, Nancy, Sedlacek, Petr, Connelly, James A, Crawford, David F, Notarangelo, Luigi D, Pai, Sung-Yun, Hassid, Jake, Veys, Paul, Gennery, Andrew R, and Cowan, Morton J

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Transplantation, Cancer, Regenerative Medicine, Hematology, Clinical Research, Stem Cell Research - Nonembryonic - Human, Stem Cell Research, Adult, Australia, B-Lymphocytes, Child, Chimerism, Europe, Female, Graft vs Host Disease, Hematopoietic Stem Cell Transplantation, Histocompatibility, Histocompatibility Testing, Humans, Infant, Infant, Newborn, Male, North America, Retrospective Studies, Severe Combined Immunodeficiency, Siblings, Survival Analysis, T-Lymphocytes, Transplantation Conditioning, Treatment Outcome, Unrelated Donors, Volunteers, Severe combined immunodeficiency, hematopoietic cell transplantation, sibling donors, unrelated donors, umbilical cord blood, conditioning, serotherapy, Conditioning, Hematopoietic cell transplantation, Serotherapy, Sibling donors, Umbilical cord blood, Unrelated donors, Immunology, Allergy
Abstract

BackgroundPatients with severe combined immunodeficiency disease who have matched sibling donors (MSDs) can proceed to hematopoietic cell transplantation (HCT) without conditioning chemotherapy.ObjectiveWe sought to determine whether the results of HCT without chemotherapy-based conditioning from matched unrelated donors (URDs), either from volunteer adults or umbilical cord blood, are comparable with those from MSDs.MethodsWe performed a multicenter survey of severe combined immunodeficiency transplantation centers in North America, Europe, and Australia to compile retrospective data on patients who have undergone unconditioned HCT from either URDs (n = 37) or MSDs (n = 66).ResultsMost patients undergoing URD HCT (92%) achieved donor T-cell engraftment compared with 97% for those with MSDs; however, estimated 5-year overall and event-free survival were worse for URD recipients (71% and 60%, respectively) compared with MSD recipients (92% and 89%, respectively; P < .01 for both). URD recipients who received pre-HCT serotherapy had similar 5-year overall survival (100%) to MSD recipients. The incidences of grade II to IV acute and chronic graft-versus-host disease were higher in URD (50% and 39%, respectively) compared with MSD (22% and 5%, respectively) recipients (P < .01 for both). In the surviving patients there was no difference in T-cell reconstitution at the last follow-up between the URD and MSD recipients; however, MSD recipients were more likely to achieve B-cell reconstitution (72% vs 17%, P < .001).ConclusionUnconditioned URD HCT achieves excellent rates of donor T-cell engraftment similar to that seen in MSD recipients, and reconstitution rates are adequate. However, only a minority will have myeloid and B-cell reconstitution, and attention must be paid to graft-versus-host disease prophylaxis. This approach might be safer in children ineligible for intense regimens to spare the potential complications of chemotherapy.

Published
2014

34. Neurocognitive Dysfunction in Hematopoietic Cell Transplant Recipients: Expert Review from the Late Effects and Quality of Life Working Committee of the Center for International Blood and Marrow Transplant Research and Complications and Quality of Life Working Party of the European Society for Blood and Marrow Transplantation

Author

Kelly, Debra Lynch, Buchbinder, David, Duarte, Rafael F., Auletta, Jeffrey J., Bhatt, Neel, Byrne, Michael, DeFilipp, Zachariah, Gabriel, Melissa, Mahindra, Anuj, Norkin, Maxim, Schoemans, Helene, Shah, Ami J., Ahmed, Ibrahim, Atsuta, Yoshiko, Basak, Grzegorz W., Beattie, Sara, Bhella, Sita, Bredeson, Christopher, Bunin, Nancy, Dalal, Jignesh, Daly, Andrew, Gajewski, James, Gale, Robert Peter, Galvin, John, Hamadani, Mehdi, Hayashi, Robert J., Adekola, Kehinde, Law, Jason, Lee, Catherine J., Liesveld, Jane, Malone, Adriana K., Nagler, Arnon, Naik, Seema, Nishihori, Taiga, Parsons, Susan K., Scherwath, Angela, Schofield, Hannah-Lise, Soiffer, Robert, Szer, Jeff, Twist, Ida, Warwick, Anne, Wirk, Baldeep M., Yi, Jean, Battiwalla, Minoo, Flowers, Mary E., Savani, Bipin, and Shaw, Bronwen E.

Published
2018
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35. Hematopoietic stem cell transplantation in patients with gain-of-function signal transducer and activator of transcription 1 mutations

Author

Leiding, Jennifer W., Okada, Satoshi, Hagin, David, Abinun, Mario, Shcherbina, Anna, Balashov, Dmitry N., Kim, Vy H.D., Ovadia, Adi, Guthery, Stephen L., Pulsipher, Michael, Lilic, Desa, Devlin, Lisa A., Christie, Sharon, Depner, Mark, Fuchs, Sebastian, van Royen-Kerkhof, Annet, Lindemans, Caroline, Petrovic, Aleksandra, Sullivan, Kathleen E., Bunin, Nancy, Kilic, Sara Sebnem, Arpaci, Fikret, Calle-Martin, Oscar de la, Martinez-Martinez, Laura, Aldave, Juan Carlos, Kobayashi, Masao, Ohkawa, Teppei, Imai, Kohsuke, Iguchi, Akihiro, Roifman, Chaim M., Gennery, Andrew R., Slatter, Mary, Ochs, Hans D., Morio, Tomohiro, and Torgerson, Troy R.

Published
2018
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36. Germline CBL mutations cause developmental abnormalities and predispose to juvenile myelomonocytic leukemia.

Author

Niemeyer, Charlotte M, Kang, Michelle W, Shin, Danielle H, Furlan, Ingrid, Erlacher, Miriam, Bunin, Nancy J, Bunda, Severa, Finklestein, Jerry Z, Gorr, Thomas A, Mehta, Parinda, Schmid, Irene, Kropshofer, Gabriele, Corbacioglu, Selim, Lang, Peter J, Klein, Christoph, Schlegel, Paul-Gerhard, Heinzmann, Andrea, Schneider, Michaela, Starý, Jan, van den Heuvel-Eibrink, Marry M, Hasle, Henrik, Locatelli, Franco, Sakai, Debbie, Archambeault, Sophie, Chen, Leslie, Russell, Ryan C, Sybingco, Stephanie S, Ohh, Michael, Braun, Benjamin S, Flotho, Christian, and Loh, Mignon L

Subjects
Humans, Cryptorchidism, Genetic Predisposition to Disease, Pedigree, DNA Mutational Analysis, Developmental Disabilities, Germ-Line Mutation, Child, Preschool, Infant, Infant, Newborn, Female, Male, Proto-Oncogene Proteins c-cbl, Leukemia, Myelomonocytic, Juvenile, Child, Preschool, Newborn, Leukemia, Myelomonocytic, Juvenile, Developmental Biology, Biological Sciences, Medical and Health Sciences
Abstract

CBL encodes a member of the Cbl family of proteins, which functions as an E3 ubiquitin ligase. We describe a dominant developmental disorder resulting from germline missense CBL mutations, which is characterized by impaired growth, developmental delay, cryptorchidism and a predisposition to juvenile myelomonocytic leukemia (JMML). Some individuals experienced spontaneous regression of their JMML but developed vasculitis later in life. Importantly, JMML specimens from affected children show loss of the normal CBL allele through acquired isodisomy. Consistent with these genetic data, the common p.371Y>H altered Cbl protein induces cytokine-independent growth and constitutive phosphorylation of ERK, AKT and S6 only in hematopoietic cells in which normal Cbl expression is reduced by RNA interference. We conclude that germline CBL mutations have developmental, tumorigenic and functional consequences that resemble disorders that are caused by hyperactive Ras/Raf/MEK/ERK signaling and include neurofibromatosis type 1, Noonan syndrome, Costello syndrome, cardiofaciocutaneous syndrome and Legius syndrome.

Published
2010

38. Tabelecleucel for EBV+PTLD after allogeneic HCT or SOT in a multicenter expanded access protocol

Author

Nikiforow, Sarah, Whangbo, Jennifer S., Reshef, Ran, Tsai, Donald E., Bunin, Nancy, Abu-Arja, Rolla, Mahadeo, Kris Michael, Weng, Wen-Kai, Van Besien, Koen, Loeb, David, Nasta, Sunita Dwivedy, Nemecek, Eneida R., Zhao, Weizhi, Sun, Yan, Galderisi, Faith, Wahlstrom, Justin, Mehta, Aditi, Gamelin, Laurence, Dinavahi, Rajani, and Prockop, Susan

Abstract

•In this R/R population with high unmet need, patients responding to tabelecleucel demonstrated a clinically meaningful survival benefit.•Tabelecleucel has a favorable safety profile and shows durable clinical benefit in R/R EBV+PTLD after HCT or SOT.

Published
2024
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40. Neurocognitive dysfunction in hematopoietic cell transplant recipients: expert review from the late effects and Quality of Life Working Committee of the CIBMTR and complications and Quality of Life Working Party of the EBMT

Author

Buchbinder, David, Kelly, Debra Lynch, Duarte, Rafael F., Auletta, Jeffery J., Bhatt, Neel, Byrne, Michael, DeFilipp, Zachariah, Gabriel, Melissa, Mahindra, Anuj, Norkin, Maxim, Schoemans, Helene, Shah, Ami J., Ahmed, Ibrahim, Atsuta, Yoshiko, Basak, Grzegorz W., Beattie, Sara, Bhella, Sita, Bredeson, Christopher, Bunin, Nancy, Dalal, Jignesh, Daly, Andrew, Gajewski, James, Gale, Robert Peter, Galvin, John, Hamadani, Mehdi, Hayashi, Robert J., Adekola, Kehinde, Law, Jason, Lee, Catherine J., Liesveld, Jane, Malone, Adriana K., Nagler, Arnon, Naik, Seema, Nishihori, Taiga, Parsons, Susan K., Scherwath, Angela, Schofield, Hannah-Lise, Soiffer, Robert, Szer, Jeff, Twist, Ida, Warwick, Anne B., Wirk, Baldeep M., Yi, Jean, Battiwalla, Minoo, Flowers, Mary D. E., Savani, Bipin, and Shaw, Bronwen E.

Published
2018
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43. Adoptive immunotherapy for primary immunodeficiency disorders with virus-specific T lymphocytes

Author

Naik, Swati, Nicholas, Sarah K., Martinez, Caridad A., Leen, Ann M., Hanley, Patrick J., Gottschalk, Steven M., Rooney, Cliona M., Hanson, I. Celine, Krance, Robert A., Shpall, Elizabeth J., Cruz, Conrad R., Amrolia, Persis, Lucchini, Giovanna, Bunin, Nancy, Heimall, Jennifer, Klein, Orly R., Gennery, Andrew R., Slatter, Mary A., Vickers, Mark A., Orange, Jordan S., Heslop, Helen E., Bollard, Catherine M., and Keller, Michael D.

Published
2016
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45. Monogenic early-onset lymphoproliferation and autoimmunity: Natural history of STAT3 gain-of-function syndrome

Author

Leiding, Jennifer W., primary, Vogel, Tiphanie P., additional, Santarlas, Valentine G.J., additional, Mhaskar, Rahul, additional, Smith, Madison R., additional, Carisey, Alexandre, additional, Vargas-Hernández, Alexander, additional, Silva-Carmona, Manuel, additional, Heeg, Maximilian, additional, Rensing-Ehl, Anne, additional, Neven, Bénédicte, additional, Hadjadj, Jérôme, additional, Hambleton, Sophie, additional, Ronan Leahy, Timothy, additional, Meesilpavikai, Kornvalee, additional, Cunningham-Rundles, Charlotte, additional, Dutmer, Cullen M., additional, Sharapova, Svetlana O., additional, Taskinen, Mervi, additional, Chua, Ignatius, additional, Hague, Rosie, additional, Klemann, Christian, additional, Kostyuchenko, Larysa, additional, Morio, Tomohiro, additional, Thatayatikom, Akaluck, additional, Ozen, Ahmet, additional, Scherbina, Anna, additional, Bauer, Cindy S., additional, Flanagan, Sarah E., additional, Gambineri, Eleonora, additional, Giovannini-Chami, Lisa, additional, Heimall, Jennifer, additional, Sullivan, Kathleen E., additional, Allenspach, Eric, additional, Romberg, Neil, additional, Deane, Sean G., additional, Prince, Benjamin T., additional, Rose, Melissa J., additional, Bohnsack, John, additional, Mousallem, Talal, additional, Jesudas, Rohith, additional, Santos Vilela, Maria Marluce Dos, additional, O’Sullivan, Michael, additional, Pachlopnik Schmid, Jana, additional, Průhová, Štěpánka, additional, Klocperk, Adam, additional, Rees, Matthew, additional, Su, Helen, additional, Bahna, Sami, additional, Baris, Safa, additional, Bartnikas, Lisa M., additional, Chang Berger, Amy, additional, Briggs, Tracy A., additional, Brothers, Shannon, additional, Bundy, Vanessa, additional, Chan, Alice Y., additional, Chandrakasan, Shanmuganathan, additional, Christiansen, Mette, additional, Cole, Theresa, additional, Cook, Matthew C., additional, Desai, Mukesh M., additional, Fischer, Ute, additional, Fulcher, David A., additional, Gallo, Silvanna, additional, Gauthier, Amelie, additional, Gennery, Andrew R., additional, Gonçalo Marques, José, additional, Gottrand, Frédéric, additional, Grimbacher, Bodo, additional, Grunebaum, Eyal, additional, Haapaniemi, Emma, additional, Hämäläinen, Sari, additional, Heiskanen, Kaarina, additional, Heiskanen-Kosma, Tarja, additional, Hoffman, Hal M., additional, Gonzalez-Granado, Luis Ignacio, additional, Guerrerio, Anthony L., additional, Kainulainen, Leena, additional, Kumar, Ashish, additional, Lawrence, Monica G., additional, Levin, Carina, additional, Martelius, Timi, additional, Neth, Olaf, additional, Olbrich, Peter, additional, Palma, Alejandro, additional, Patel, Niraj C., additional, Pozos, Tamara, additional, Preece, Kahn, additional, Lugo Reyes, Saúl Oswaldo, additional, Russell, Mark A., additional, Schejter, Yael, additional, Seroogy, Christine, additional, Sinclair, Jan, additional, Skevofilax, Effie, additional, Suan, Daniel, additional, Suez, Daniel, additional, Szabolcs, Paul, additional, Velasco, Helena, additional, Warnatz, Klaus, additional, Walkovich, Kelly, additional, Worth, Austen, additional, Seppänen, Mikko R.J., additional, Torgerson, Troy R., additional, Sogkas, Georgios, additional, Ehl, Stephan, additional, Tangye, Stuart G., additional, Cooper, Megan A., additional, Milner, Joshua D., additional, Forbes Satter, Lisa R., additional, Aleshkevich, Svetlana, additional, Allende, Luis M., additional, Atkinson, T. Prescott, additional, Atschekzei, Faranaz, additional, Aydemir, Sezin, additional, Aygunes, Utku, additional, Barlogis, Vincent, additional, Baumann, Ulrich, additional, Belko, John, additional, Bezrodnik, Liliana, additional, Biebl, Ariane, additional, Broderick, Lori, additional, Bunin, Nancy J., additional, Caldirola, Maria Soledad, additional, Castelle, Martin, additional, Celmeli, Fatih, additional, Charbonnier, Louis-Marie, additional, Chatila, Talal A., additional, Chellapandian, Deepak, additional, Cokugras, Haluk, additional, Conlon, Niall, additional, Cox, Fionnuala, additional, Crickx, Etienne, additional, Dalgic, Buket, additional, ASH Dalm, Virgil, additional, Danielian, Silvia, additional, Dominguez-Pinilla, Nerea, additional, Dujovny, Tal, additional, Ebbo, Mikael, additional, Eken, Ahmet, additional, Esty, Brittany, additional, Fabre, Alexandre, additional, Fischer, Alain, additional, Hannibal, Mark, additional, Huppert, Laura, additional, Ikeda, Marc D., additional, Jolles, Stephen, additional, Jolly, Kent W., additional, Jones, Neil, additional, Kanariou, Maria, additional, Karakoc-Aydiner, Elif, additional, Karamantziani, Theoni, additional, Kelaidi, Charikleia, additional, Keogan, Mary, additional, Pac Kisaarslan, Ayşenur, additional, Kiykim, Ayca, additional, Kotsonis, Kosmas, additional, Kuzmenko, Natalia, additional, Leroy, Sylvie, additional, Lianou, Dimitra, additional, Longhurst, Hilary, additional, Lorenz, Myriam Ricarda, additional, Maffucci, Patrick, additional, Manson, Ania, additional, Marchal, Sarah, additional, Malphettes, Marion, additional, Marega, Lia Furlaneto, additional, Mauracher, Andrea A., additional, Miller, Holly, additional, Mombourquette, Joy, additional, Morgan, Noel G., additional, Mukhina, Anna, additional, Nathalie, Aladjidi, additional, Nelken, Brigitte, additional, Nolan, David, additional, Norlin, Anna-Carin, additional, Oleastro, Matias, additional, Ozcan, Alper, additional, Pasquet, Marlene, additional, Pegler, José Roberto, additional, Picard, Capucine, additional, Polychronopoulou, Sophia, additional, Quartier, Pierre, additional, Quesada, Juan Francisco, additional, Ramakers, Jan, additional, Randall, Katrina L., additional, Rao, V. Koneti, additional, Remiker, Allison, additional, Resin, Geraldine, additional, Richmond, Peter, additional, Rieux-Laucat, Frederic, additional, Rodina, Yulia, additional, Rohrlich, Pierre, additional, Sachs, Johnathan, additional, Sakovich, Inga, additional, Santarlas, Christopher, additional, Sari, Sinan, additional, Sawicki, Gregory, additional, Schauer, Uwe, additional, Scheffler Mendoza, Selma C., additional, Schvetz, Oksana, additional, Schmidt, Reinhold Ernst, additional, Schwarz, Klaus, additional, Sediva, Anna, additional, Sinclair, Kyle, additional, Slatter, Mary, additional, Sleasman, John, additional, Stergiou, Katerina, additional, Suratannon, Narissara, additional, Tanita, Kay, additional, Thompson, Grace, additional, Travis, Stephen, additional, Trojan, Timothy, additional, Tsinti, Maria, additional, Unal, Ekrem, additional, Urdinez, Luciano, additional, Vazquez-Gomez, Felisa, additional, Villa, Mariana, additional, Weinrich, Michael, additional, Weiss, Mitchell J., additional, Wright, Benjamin, additional, Yilmaz, Ebru, additional, Zachova, Radana, additional, and Zhang, Yu, additional

Published
2023
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47. Safety and Efficacy of Virus-Specific Cytotoxic T-Lymphocytes Manufactured By the IFN-γ Cytokine Capture System for the Treatment of Refractory Adenovirus, Cytomegalovirus, Epstein Barr Virus, and BK Virus Infections in Children, Adolescents and Young Adults after Allogeneic Hematopoietic Stem Cell Transplantation, Solid Organ Transplantation, or with Primary Immunodeficiency (IND# 17449)

Author

Flower, Allyson, primary, Talano, Julie, additional, Ayello, Janet, additional, Rigot, Olivia, additional, Harrison, Lauren, additional, Morris, Erin, additional, Mintzer, Elizabeth, additional, Maryamchick, Elena, additional, Wang, Yongping, additional, O’Donnell, Lynn C., additional, Abu-Arja, Rolla, additional, Lee, Dean A., additional, Johnson, Bryon D, additional, Chu, Julia, additional, Dvorak, Christopher C., additional, Shenoy, Shalini, additional, Bunin, Nancy J., additional, and Cairo, Mitchell S., additional

Published
2023
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48. 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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49. Manufacture, Immunological Characterization and Clinical Response of GMP Grade Sars-COV-2 Cytotoxic T Lymphocytes (CTLs) Utilizing the Clinimacs® Cytokine Capture System

Author

Chu, Yaya, primary, Lamb, Margaret, additional, Maryamchik, Elena, additional, Rigot, Olivia, additional, Ayello, Janet, additional, Harrison, Lauren, additional, Mintzer, Elizabeth, additional, Shaw, Rosemarie, additional, Behbehani, Gregory, additional, Mardis, Elaine, additional, Miller, Katherine, additional, Rao Venkata, Lakshmi Prakruthi, additional, Chang, Hsiaochi, additional, Lee, Dean A., additional, Rosenthal, Elana Y., additional, Kadauke, Stephan, additional, Bunin, Nancy J., additional, Talano, Julie, additional, Johnson, Bryon D, additional, Wang, Yongping, additional, and Cairo, Mitchell S., additional

Published
2023
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