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2. Granulocyte Transfusions in Patients with Chronic Granulomatous Disease Undergoing Hematopoietic Cell Transplantation or Gene Therapy

Author

Arnold, Danielle E, Chellapandian, Deepak, Parikh, Suhag, Mallhi, Kanwaldeep, Marsh, Rebecca A, Heimall, Jennifer R, Grossman, Debra, Chitty-Lopez, Maria, Murguia-Favela, Luis, Gennery, Andrew R, Boulad, Farid, Arbuckle, Erin, Cowan, Morton J, Dvorak, Christopher C, Griffith, Linda M, Haddad, Elie, Kohn, Donald B, Notarangelo, Luigi D, Pai, Sung-Yun, Puck, Jennifer M, Pulsipher, Michael A, Torgerson, Troy, Kang, Elizabeth M, Malech, Harry L, and Leiding, Jennifer W

Subjects
Biomedical and Clinical Sciences, Immunology, Transplantation, Clinical Research, Hematology, Inflammatory and immune system, Genetic Therapy, Graft vs Host Disease, Granulocytes, Granulomatous Disease, Chronic, Hematopoietic Stem Cell Transplantation, Humans, Retrospective Studies, Transplantation Conditioning, Granulocyte transfusions, Chronic granulomatous disease, Alloimmunization, Hematopoietic cell transplantation, Graft failure
Abstract

Granulocyte transfusions are sometimes used as adjunctive therapy for the treatment of infection in patients with chronic granulomatous disease (CGD). However, granulocyte transfusions can be associated with a high rate of alloimmunization, and their role in CGD patients undergoing hematopoietic cell transplantation (HCT) or gene therapy (GT) is unknown. We identified 27 patients with CGD who received granulocyte transfusions pre- (within 6 months) and/or post-HCT or GT in a retrospective survey. Twelve patients received granulocyte transfusions as a bridge to cellular therapy. Six (50%) of these patients had a complete or partial response. However, six of 10 (60%) patients for whom testing was performed developed anti-HLA antibodies, and three of the patients also had severe immune-mediated cytopenia within the first 100 days post-HCT or GT. Fifteen patients received granulocyte transfusions post-HCT only. HLA antibodies were not checked for any of these 15 patients, but there were no cases of early immune-mediated cytopenia. Out of 25 patients who underwent HCT, there were 5 (20%) cases of primary graft failure. Three of the patients with primary graft failure had received granulocyte transfusions pre-HCT and were subsequently found to have anti-HLA antibodies. In this small cohort of patients with CGD, granulocyte transfusions pre-HCT or GT were associated with high rates of alloimmunization, primary graft failure, and early severe immune-mediated cytopenia post-HCT or GT. Granulocyte transfusions post-HCT do not appear to confer an increased risk of graft failure.

Published
2022

3. Impact of rabbit anti-thymocyte globulin (ATG) exposure on outcomes after ex vivo T-cell–depleted hematopoietic cell transplantation in pediatric and young adult patients

Author

Lakkaraja, Madhavi, Mauguen, Audrey, Boulad, Farid, Cancio, Maria I., Curran, Kevin J., Harris, Andrew C., Kernan, Nancy A., Klein, Elizabeth, Kung, Andrew L., Oved, Joseph, Prockop, Susan, Scaradavou, Andromachi, Spitzer, Barbara, O'Reilly, Richard J., and Boelens, Jaap Jan

Published
2024
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4. Association between busulfan exposure and survival in patients undergoing a CD34+ selected stem cell transplantation

Author

Tamari, Roni, Scordo, Michael, Kunvarjee, Binni M., Proli, Anthony, Lin, Andrew, Flynn, Jessica, Cho, Christina, Devlin, Sean, Klein, Elizabeth, Boulad, Farid, Cancio, Maria I., Curran, Kevin J., Jakubowski, Ann A., Kernan, Nancy A., Kung, Andrew L., O’Reilly, Richard J., Papadopoulos, Esperanza B., Prockop, Susan, Scaradavou, Andromachi, Shaffer, Brian C., Shah, Gunjan, Spitzer, Barbara, Gyurkocza, Boglarka, Giralt, Sergio A., Perales, Miguel-Angel, and Boelens, Jaap Jan

Published
2023
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5. Genomic signature of Fanconi anaemia DNA repair pathway deficiency in cancer

Author

Webster, Andrew L. H., Sanders, Mathijs A., Patel, Krupa, Dietrich, Ralf, Noonan, Raymond J., Lach, Francis P., White, Ryan R., Goldfarb, Audrey, Hadi, Kevin, Edwards, Matthew M., Donovan, Frank X., Hoogenboezem, Remco M., Jung, Moonjung, Sridhar, Sunandini, Wiley, Tom F., Fedrigo, Olivier, Tian, Huasong, Rosiene, Joel, Heineman, Thomas, Kennedy, Jennifer A., Bean, Lorenzo, Rosti, Rasim O., Tryon, Rebecca, Gonzalez, Ashlyn-Maree, Rosenberg, Allana, Luo, Ji-Dung, Carroll, Thomas S., Shroff, Sanjana, Beaumont, Michael, Velleuer, Eunike, Rastatter, Jeff C., Wells, Susanne I., Surrallés, Jordi, Bagby, Grover, MacMillan, Margaret L., Wagner, John E., Cancio, Maria, Boulad, Farid, Scognamiglio, Theresa, Vaughan, Roger, Beaumont, Kristin G., Koren, Amnon, Imielinski, Marcin, Chandrasekharappa, Settara C., Auerbach, Arleen D., Singh, Bhuvanesh, Kutler, David I., Campbell, Peter J., and Smogorzewska, Agata

Published
2022
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6. Correction: Chronic Granulomatous Disease-Associated IBD Resolves and Does Not Adversely Impact Survival Following Allogeneic HCT.

Author

Marsh, Rebecca, Leiding, Jennifer, Logan, Brent, Griffith, Linda, Arnold, Danielle, Haddad, Elie, Falcone, E, Yin, Ziyan, Patel, Kadam, Arbuckle, Erin, Bleesing, Jack, Sullivan, Kathleen, Heimall, Jennifer, Burroughs, Lauri, Skoda-Smith, Suzanne, Chandrakasan, Shanmuganathan, Yu, Lolie, Oshrine, Benjamin, Cuvelier, Geoffrey, Thakar, Monica, Chen, Karin, Teira, Pierre, Shenoy, Shalini, Phelan, Rachel, Forbes, Lisa, Martinez, Caridad, Chellapandian, Deepak, Dávila Saldaña, Blachy, Shah, Ami, Weinacht, Katja, Joshi, Avni, Boulad, Farid, Quigg, Troy, Grossman, Debi, Torgerson, Troy, Graham, Pamela, Prasad, Vinod, Knutsen, Alan, Chong, Hey, Miller, Holly, de la Morena, M, DeSantes, Kenneth, Stenger, Elizabeth, Pai, Sung-Yun, Routes, John, Kapoor, Neena, Pulsipher, Michael, Malech, Harry, Parikh, Suhag, Kang, Elizabeth, Notarangelo, Luigi, Dvorak, Christopher, Puck, Jennifer, Cowan, Morton, and Kohn, Donald

Abstract

The original version of this article unfortunately contained the missing author, Caridad Martinez. The authors would like to correct the list. We apologize for any inconvenience that this may have caused. The correct author list is shown above.

Published
2020

7. Low toxicity and favorable overall survival in relapsed/refractory B-ALL following CAR T cells and CD34-selected T-cell depleted allogeneic hematopoietic cell transplant

Author

Fabrizio, Vanessa A, Kernan, Nancy A, Boulad, Farid, Cancio, Maria, Allen, Jennifer, Higman, Meghan, Margossian, Steven P, Mauguen, Audrey, Prockop, Susan, Scaradavou, Andromachi, Shah, Niketa, Spitzer, Barbara, Stieglitz, Elliot, Yeager, Nicholas, O’Reilly, Richard J, Brentjens, Renier J, Jan Boelens, Jaap, and Curran, Kevin J

Subjects
Stem Cell Research - Nonembryonic - Human, Pediatric, Stem Cell Research, Clinical Research, Transplantation, Cancer, Clinical Trials and Supportive Activities, Regenerative Medicine, Evaluation of treatments and therapeutic interventions, 6.2 Cellular and gene therapies, Child, Graft vs Host Disease, Hematopoietic Stem Cell Transplantation, Humans, Immunotherapy, Adoptive, Infant, Neoplasm Recurrence, Local, Retrospective Studies, T-Lymphocytes, Young Adult, Clinical Sciences, Oncology and Carcinogenesis, Immunology
Abstract

To define the tolerability and outcome of allogeneic hematopoietic stem cell transplant (allo-HSCT) following CAR T-cell therapy, we retrospectively reviewed pediatric/young adult patients with relapsed/refractory B-ALL who underwent this treatment. Fifteen patients (median age 13 years; range 1-20 years) with a median potential follow-up of 39 months demonstrated 24-month cumulative incidence of relapse, cumulative incidence of TRM, and OS of 16% (95% CI: 0-37%), 20% (95% CI: 0-40%), and 80% (95% CI: 60-100%), respectively. Severe toxicity following CAR T cells did not impact OS (p = 0.27), while greater time from CAR T cells to allo-HSCT (>80 days) was associated with a decrease in OS. In comparing CD34-selected T-cell depleted (TCD; n = 9) vs unmodified (n = 6) allo-HSCT, the cumulative incidence of relapse, TRM, and OS at 24 months was 22% (95% CI: 0-49%) vs 0% (p = 0.14), 0% vs 50% [95% CI: 10-90%] (p = 0.02) and 100% vs 50% [95% CI: 10-90%] (p = 0.02). In this small cohort of patients, CAR T cells followed by a CD34-selected TCD allo-HSCT appears to result in less TRM and favorable OS when compared with unmodified allo-HSCT. There was no evidence that disease control was impacted by the type of consolidative allo-HSCT, which demonstrates the feasibility of this approach.

Published
2020

8. Chronic Granulomatous Disease-Associated IBD Resolves and Does Not Adversely Impact Survival Following Allogeneic HCT.

Author

Marsh, Rebecca A, Leiding, Jennifer W, Logan, Brent R, Griffith, Linda M, Arnold, Danielle E, Haddad, Elie, Falcone, E Liana, Yin, Ziyan, Patel, Kadam, Arbuckle, Erin, Bleesing, Jack J, Sullivan, Kathleen E, Heimall, Jennifer, Burroughs, Lauri M, Skoda-Smith, Suzanne, Chandrakasan, Shanmuganathan, Yu, Lolie C, Oshrine, Benjamin R, Cuvelier, Geoffrey DE, Thakar, Monica S, Chen, Karin, Teira, Pierre, Shenoy, Shalini, Phelan, Rachel, Forbes, Lisa R, Chellapandian, Deepak, Dávila Saldaña, Blachy J, Shah, Ami J, Weinacht, Katja G, Joshi, Avni, Boulad, Farid, Quigg, Troy C, Dvorak, Christopher C, Grossman, Debi, Torgerson, Troy, Graham, Pamela, Prasad, Vinod, Knutsen, Alan, Chong, Hey, Miller, Holly, de la Morena, M Teresa, DeSantes, Kenneth, Cowan, Morton J, Notarangelo, Luigi D, Kohn, Donald B, Stenger, Elizabeth, Pai, Sung-Yun, Routes, John M, Puck, Jennifer M, Kapoor, Neena, Pulsipher, Michael A, Malech, Harry L, Parikh, Suhag, Kang, Elizabeth M, and submitted on behalf of the Primary Immune Deficiency Treatment Consortium

Subjects
submitted on behalf of the Primary Immune Deficiency Treatment Consortium, Neutrophils, Transplantation Chimera, Humans, Inflammatory Bowel Diseases, Granulomatous Disease, Chronic, Graft vs Host Disease, Leukocyte Count, Prognosis, Treatment Outcome, Hematopoietic Stem Cell Transplantation, Transplantation, Homologous, Severity of Illness Index, Incidence, Retrospective Studies, Adolescent, Adult, Child, Child, Preschool, Infant, Female, Male, Young Adult, Allogeneic hematopoietic cell transplantation, allogeneic bone marrow transplantation, allogeneic hematopoietic stem cell transplantation, chronic granulomatous disease, inflammatory bowel disease, primary immunodeficiency, Autoimmune Disease, Inflammatory Bowel Disease, Digestive Diseases, Rare Diseases, Clinical Research, Transplantation, Oral and gastrointestinal, Immunology
Abstract

IntroductionInflammatory bowel disease (IBD) affects approximately 1/3 of patients with chronic granulomatous disease (CGD). Comprehensive investigation of the effect of allogeneic hematopoietic cell transplantation (HCT) on CGD IBD and the impact of IBD on transplant outcomes is lacking.MethodsWe collected data retrospectively from 145 patients with CGD who had received allogeneic HCT at 26 Primary Immune Deficiency Treatment Consortium (PIDTC) centers between January 1, 2005 and June 30, 2016.ResultsForty-nine CGD patients with IBD and 96 patients without IBD underwent allogeneic HCT. Eighty-nine percent of patients with IBD and 93% of patients without IBD engrafted (p = 0.476). Upper gastrointestinal acute GVHD occurred in 8.5% of patients with IBD and 3.5% of patients without IBD (p = 0.246). Lower gastrointestinal acute GVHD occurred in 10.6% of patients with IBD and 11.8% of patients without IBD (p = 0.845). The cumulative incidence of acute GVHD grades II-IV was 30% (CI 17-43%) in patients with IBD and 20% (CI 12-29%) in patients without IBD (p = 0.09). Five-year overall survival was equivalent for patients with and without IBD: 80% [CI 66-89%] and 83% [CI 72-90%], respectively (p = 0.689). All 33 surviving evaluable patients with a history of IBD experienced resolution of IBD by 2 years following allogeneic HCT.ConclusionsIn this cohort, allogeneic HCT was curative for CGD-associated IBD. IBD should not contraindicate HCT, as it does not lead to an increased risk of mortality. This study is registered at clinicaltrials.gov NCT02082353.

Published
2019

9. Metformin for treatment of cytopenias in children and young adults with Fanconi anemia

Author

Pollard, Jessica A., Furutani, Elissa, Liu, Shanshan, Esrick, Erica, Cohen, Laurie E., Bledsoe, Jacob, Liu, Chih-Wei, Lu, Kun, de Haro, Maria Jose Ramirez, Surrallés, Jordi, Malsch, Maggie, Kuniholm, Ashley, Galvin, Ashley, Armant, Myriam, Kim, Annette S., Ballotti, Kaitlyn, Moreau, Lisa, Zhou, Yu, Babushok, Daria, Boulad, Farid, Carroll, Clint, Hartung, Helge, Hont, Amy, Nakano, Taizo, Olson, Tim, Sze, Sei-Gyung, Thompson, Alexis A., Wlodarski, Marcin W., Gu, Xuesong, Libermann, Towia A., D'Andrea, Alan, Grompe, Markus, Weller, Edie, and Shimamura, Akiko

Published
2022
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10. Antithymocyte globulin exposure in CD34+ T-cell–depleted allogeneic hematopoietic cell transplantation

Author

Lakkaraja, Madhavi, Scordo, Michael, Mauguen, Audrey, Cho, Christina, Devlin, Sean, Ruiz, Josel D., Klein, Elizabeth, Avecilla, Scott T., Boulad, Farid, Cancio, Maria I., Curran, Kevin J., Jakubowski, Ann A., Kernan, Nancy A., Kung, Andrew L., O'Reilly, Richard J., Papadopoulos, Esperanza B., Prockop, Susan, van Roessel, Ichelle, Scaradavou, Andromachi, Shaffer, Brian C., Shah, Gunjan, Spitzer, Barbara, Tamari, Roni, Giralt, Sergio A., Perales, Miguel-Angel, and Boelens, Jaap Jan

Published
2022
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11. Lentiviral globin gene therapy with reduced-intensity conditioning in adults with β-thalassemia: a phase 1 trial

Author

Boulad, Farid, Maggio, Aurelio, Wang, Xiuyan, Moi, Paolo, Acuto, Santina, Kogel, Friederike, Takpradit, Chayamon, Prockop, Susan, Mansilla-Soto, Jorge, Cabriolu, Annalisa, Odak, Ashlesha, Qu, Jinrong, Thummar, Keyur, Du, Fang, Shen, Lingbo, Raso, Simona, Barone, Rita, Di Maggio, Rosario, Pitrolo, Lorella, Giambona, Antonino, Mingoia, Maura, Everett, John K., Hokama, Pascha, Roche, Aoife M., Cantu, Vito Adrian, Adhikari, Hriju, Reddy, Shantan, Bouhassira, Eric, Mohandas, Narla, Bushman, Frederic D., Rivière, Isabelle, and Sadelain, Michel

Published
2022
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12. Third-party cytomegalovirus-specific T cells improved survival in refractory cytomegalovirus viremia after hematopoietic transplant

Author

Prockop, Susan E., Hasan, Aisha, Doubrovina, Ekaterina, Dahi, Parastoo B., Rodriguez-Sanchez, Irene, Curry, Michael, Mauguen, Audrey, Papanicolaou, Genovefa A., Su, Yiqi, Yao, JinJuan, Arcila, Maria, Boulad, Farid, Castro- Malaspina, Hugo, Cho, Christina, Curran, Kevin J., Giralt, Sergio, Kernan, Nancy A., Koehne, Guenther, Jakubowski, Ann, Papadopoulos, Esperanza, Perales, Miguel-Angel, Politikos, Ioannis, Price, Keith, Selvakumar, Annamalai, Sauter, Craig S., Tamari, Roni, Vizconde, Teresa, Young, James W., and O'Reilly, Richard J.

Subjects
T cells -- Health aspects, Cellular therapy -- Testing, Viremia -- Care and treatment, Cytomegalovirus infections -- Care and treatment, Hematopoietic stem cells -- Transplantation, Health care industry
Abstract

BACKGROUND. Refractory CMV viremia and disease are associated with significant morbidity and mortality in recipients of hematopoietic stem cell transplant (HCT). METHODS. In phase I/II trials, we treated 67 subjects for CMV viremia or disease arising after HCT with adoptive transfer of banked, third-party, CMVpp65-sensitized T cells (CMVpp65-VSTs). All were evaluable for toxicity and 59 for response. Evaluable subjects had CMV disease or persisting viremia that had failed at least 2 weeks of induction therapy with a median of 3 antiviral drugs; 84.7% had more than 3 of 11 high-risk features. CMVpp65-VSTs were specific for 1 to 3 CMVpp65 epitopes, presented by a limited set of HLA class I or II alleles, and were selected based on high-resolution HLA matching at 2 of 10 HLA alleles and matching for subject and subject's HCT donor for 1 or more alleles through which the CMVpp65- VSTs were restricted. RESULTS. T cell infusions were well tolerated. Of 59 subjects evaluable for response, 38 (64%) achieved complete or durable partial responses. CONCLUSIONS. Recipients responding to CMVpp65VSTs experienced an improved overall survival. Of the risk factors evaluated, transplant type, recipient [CD4.sup.+] and [CD8.sup.+] T cell levels prior to adoptive therapy, and the HLA restriction of CMVpp65-VSTs infused each significantly affected responses. In addition, CMVpp65-specific T cells of HCT donor or recipient origin contributed to the durability of both complete and partial responses. TRIAL REGISTRATION. NCT00674648; NCT01646645; NCT02136797 (NIH). FUNDING. NIH (P01 CA23766, R21 CA162002 and P30 CA008748); Aubrey Fund; Claire Tow Foundation; Major Family Foundation; 'Rick' Eisemann Pediatric Research Fund; Banbury Foundation; Edith Robertson Foundation; Larry Smead Foundation., Introduction Cytomegalovirus (CMV) infections remain a major cause of morbidity and mortality following allogeneic hematopoietic stem cell transplants (HCTs) (1-5). Although prophylactic or preemptive treatment with ganciclovir and/or foscarnet and, [...]

Published
2023
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13. Hematopoietic Stem Cell Transplantation Activity in Pediatric Cancer between 2008 and 2014 in the United States: A Center for International Blood and Marrow Transplant Research Report

Author

Khandelwal, Pooja, Millard, Heather R, Thiel, Elizabeth, Abdel-Azim, Hisham, Abraham, Allistair A, Auletta, Jeffery J, Boulad, Farid, Brown, Valerie I, Camitta, Bruce M, Chan, Ka Wah, Chaudhury, Sonali, Cowan, Morton J, Angel-Diaz, Miguel, Gadalla, Shahinaz M, Gale, Robert Peter, Hale, Gregory, Kasow, Kimberly A, Keating, Amy K, Kitko, Carrie L, MacMillan, Margaret L, Olsson, Richard F, Page, Kristin M, Seber, Adriana, Smith, Angela R, Warwick, Anne B, Wirk, Baldeep, and Mehta, Parinda A

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Immunology, Pediatric Cancer, Orphan Drug, Regenerative Medicine, Pediatric Research Initiative, Pediatric, Transplantation, Rare Diseases, Hematology, Cancer, Stem Cell Research - Nonembryonic - Human, Childhood Leukemia, Stem Cell Research, Clinical Research, Adolescent, Allografts, Autografts, Brain Neoplasms, Calcineurin Inhibitors, Child, Child, Preschool, Female, Hematopoietic Stem Cell Transplantation, Humans, Infant, Male, Methotrexate, Neuroblastoma, Retrospective Studies, Transplantation Conditioning, Hematopoietic stem cell transplantation, Pediatric cancers, Clinical Sciences, Cardiovascular medicine and haematology
Abstract

This Center for International Blood and Marrow Transplant Research report describes the use of hematopoietic stem cell transplantation (HSCT) in pediatric patients with cancer, 4408 undergoing allogeneic (allo) and3076 undergoing autologous (auto) HSCT in the United States between 2008 and 2014. In both settings, there was a greater proportion of boys (n = 4327; 57%), children < 10 years of age (n = 4412; 59%), whites (n = 5787; 77%), and children with a performance score ≥ 90% at HSCT (n = 6187; 83%). Leukemia was the most common indication for an allo-transplant (n = 4170; 94%), and among these, acute lymphoblastic leukemia in second complete remission (n = 829; 20%) and acute myeloid leukemia in first complete remission (n = 800; 19%) werethe most common. The most frequently used donor relation, stem cell sources, and HLA match were unrelated donor (n = 2933; 67%), bone marrow (n = 2378; 54%), and matched at 8/8 HLA antigens (n = 1098; 37%) respectively. Most allo-transplants used myeloablative conditioning (n = 4070; 92%) and calcineurin inhibitors and methotrexate (n = 2245; 51%) for acute graft-versus-host disease prophylaxis. Neuroblastoma was the most common primary neoplasm for an auto-transplant (n = 1338; 44%). Tandem auto-transplants for neuroblastoma declined after 2012 (40% in 2011, 25% in 2012, and 8% in 2014), whereas tandem auto-transplants increased for brain tumors (57% in 2008 and 77% in 2014). Allo-transplants from relatives other than HLA-identical siblings doubled between 2008 and 2014 (3% in 2008 and 6% in 2014). These trends will be monitored in future reports of transplant practices in the United States.

Published
2017

14. CD4+ T-cell reconstitution predicts survival outcomes after acute graft-versus-host-disease: a dual-center validation

Author

de Koning, Coco, Prockop, Susan, van Roessel, Ichelle, Kernan, Nancy, Klein, Elizabeth, Langenhorst, Jurgen, Szanto, Celina, Belderbos, Mirjam, Bierings, Marc, Boulad, Farid, Bresters, Dorine, Cancio, Maria, Curran, Kevin, Kollen, Wouter, O'Reilly, Richard, Scaradavou, Andromachi, Spitzer, Barbara, Versluijs, Birgitta, Huitema, Alwin, Lindemans, Caroline, Nierkens, Stefan, and Boelens, Jaap Jan

Published
2021
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15. Management of a major varicella zoster exposure in a pediatric oncology population.

Author

Rossetto, Carol, Sepkowitz, Kent, Ackerman, Jill, Corke, Rachel, Pickett, Nina J., Cudjoe, Wini, Eagan, Janet, Kamboj, Mini, O'Reilly, Richard J., and Boulad, Farid

Subjects
CHICKENPOX vaccines, CANCER patients, PEDIATRIC oncology, VACCINATION status, CHILDHOOD cancer
Abstract

Management of the exposure of pediatric oncology patients to varicella zoster virus (VZV) is controversial. We report the exposure of 56 patients to a single child with chicken pox at a pediatric cancer housing facility and describe our strategic approach for their management. We reviewed the immune and clinical status of 56 children with cancer receiving ongoing treatment at Memorial Sloan Kettering Cancer Center (MSK) who, while living at a pediatric cancer housing facility, were exposed to the index patient. The management of patients exposed included: (1) determination of immune status, (2) availability of vaccination history or VZV disease prophylaxis, (3) exposure status and subsequent isolation during the period of incubation, and (4) VZV disease prophylaxis. In addition to the 56 patients exposed to the index case, eight children with cancer treated at other facilities and 11 healthy siblings living in the facility were exposed. Of the 56 MSK patients, 21 were classified as immunosuppressed and received varicella zoster immune globulin (human), intravenous standard immune globulin, or acyclovir based on serostatus and immune function. The cohort was followed for 4 weeks after the exposure and no secondary infections were diagnosed. We performed a risk assessment and created a management plan to control and prevent further exposure and development of disease. No secondary cases developed. This strategic approach could serve as a model for the management of VZV exposure for other pediatric oncology centers. [ABSTRACT FROM AUTHOR]

Published
2024
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17. Clinical features and outcomes of patients with Shwachman-Diamond syndrome and myelodysplastic syndrome or acute myeloid leukaemia: a multicentre, retrospective, cohort study

Author

Myers, Kasiani C, Furutani, Elissa, Weller, Edie, Siegele, Bradford, Galvin, Ashley, Arsenault, Valerie, Alter, Blanche P, Boulad, Farid, Bueso-Ramos, Carlos, Burroughs, Lauri, Castillo, Paul, Connelly, James, Davies, Stella M, DiNardo, Courtney D, Hanif, Iftikhar, Ho, Richard H, Karras, Nicole, Manalang, Michelle, McReynolds, Lisa J, Nakano, Taizo A, Nalepa, Grzegorz, Norkin, Maxim, Oberley, Matthew J, Orgel, Etan, Pastore, Yves D, Rosenthal, Joseph, Walkovich, Kelly, Larson, Jordan, Malsch, Maggie, Elghetany, M Tarek, Fleming, Mark D, and Shimamura, Akiko

Published
2020
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18. Toxicity and response after CD19-specific CAR T-cell therapy in pediatric/young adult relapsed/refractory B-ALL

Author

Curran, Kevin J., Margossian, Steven P., Kernan, Nancy A., Silverman, Lewis B., Williams, David A., Shukla, Neerav, Kobos, Rachel, Forlenza, Christopher J., Steinherz, Peter, Prockop, Susan, Boulad, Farid, Spitzer, Barbara, Cancio, Maria I., Boelens, Jaap Jan, Kung, Andrew L., Khakoo, Yasmin, Szenes, Victoria, Park, Jae H., Sauter, Craig S., Heller, Glenn, Wang, Xiuyan, Senechal, Brigitte, O'Reilly, Richard J., Riviere, Isabelle, Sadelain, Michel, and Brentjens, Renier J.

Published
2019
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20. Off-the-shelf EBV-specific T cell immunotherapy for rituximab-refractory EBV-associated lymphoma following transplantation

Author

Prockop, Susan, Doubrovina, Ekaterina, Suser, Stephanie, Heller, Glenn, Barker, Juliet, Dahi, Parastoo, Perales, Miguel A., Papadopoulos, Esperanza, Sauter, Craig, Castro-Malaspina, Hugo, Boulad, Farid, Curran, Kevin J., Giralt, Sergio, Gyurkocza, Boglarka, Hsu, Katharine C., Jakubowski, Ann, Hanash, Alan M., Kernan, Nancy A., Kobos, Rachel, Koehne, Guenther, Landau, Heather, Ponce, Doris, Spitzer, Barbara, Young, James W., Behr, Gerald, Dunphy, Mark, Haque, Sofia, Teruya-Feldstein, Julie, Arcila, Maria, Moung, Christine, Hsu, Susan, Hasan, Aisha, and O'Reilly, Richard J.

Subjects
T cells, Banks (Finance), Antineoplastic agents, Non-Hodgkin's lymphomas -- Development and progression, Immunotherapy, Drug approval, Antimitotic agents, Health care industry, Memorial Sloan-Kettering Cancer Center
Abstract

BACKGROUND. Adoptive transfer of donor-derived EBV-specific cytotoxic T-lymphocytes (EBV-CTLs) can eradicate EBV- associated lymphomas (EBV-PTLD) after transplantation of hematopoietic cell (HCT) or solid organ (SOT) but is unavailable for most patients. METHODS. We developed a third-party, allogeneic, off-the-shelf bank of 330 GMP-grade EBV-CTL lines from specifically consented healthy HCT donors. We treated 46 recipients of HCT (n = 33) or SOT (n = 13) with established EBV-PTLD, who had failed rituximab therapy, with third-party EBV-CTLs. Treatment cycles consisted of 3 weekly infusions of EBV-CTLs and 3 weeks of observation. RESULTS. EBV-CTLs did not induce significant toxicities. One patient developed grade I skin graft-versus-host disease. Complete remission (CR) or sustained partial remission (PR) was achieved in 68% of HCT recipients and 54% of SOT recipients. For patients who achieved CR/PR or stable disease after cycle 1, one year overall survival was 88.9% and 81.8%, respectively. In addition, 3 of 5 recipients with POD after a first cycle who received EBV-CTLs from a different donor achieved CR or durable PR (60%) and survived longer than 1 year. Maximal responses were achieved after a median of 2 cycles. CONCLUSION. Third-party EBV-CTLs of defined HLA restriction provide safe, immediately accessible treatment for EBV- PTLD. Secondary treatment with EBV-CTLs restricted by a different HLA allele (switch therapy) can also induce remissions if initial EBV-CTLs are ineffective. These results suggest a promising potential therapy for patients with rituximab-refractory EBV-associated lymphoma after transplantation. TRIAL REGISTRATION. Phase II protocols (NCT01498484 and NCT00002663) were approved by the Institutional Review Board at Memorial Sloan Kettering Cancer Center, the FDA, and the National Marrow Donor Program. FUNDING. This work was supported by NIH grants CA23766 and R21CA162002, the Aubrey Fund, the Claire Tow Foundation, the Major Family Foundation, the Max Cure Foundation, the Richard 'Rick' J. Eisemann Pediatric Research Fund, the Banbury Foundation, the Edith Robertson Foundation, and the Larry Smead Foundation. Atara Biotherapeutics licensed the bank of third-party EBV-CTLs from Memorial Sloan Kettering Cancer Center in June 2015., Introduction EBV-induced lymphomas are a significant cause of morbidity and mortality for recipients of allogeneic hematopoietic cell transplants (HCTs) and solid organ transplants (SOTs) (1-5). While decreasing immunosuppression can induce [...]

Published
2020
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21. Combined umbilical cord blood and bone marrow from HLA‐identical sibling donors for hematopoietic stem cell transplantation in children with hemoglobinopathies

Author

Soni, Sandeep, Boulad, Farid, Cowan, Morton J, Scaradavou, Andromachi, Dahake, Jueeli, Edwards, Sandie, and Walters, Mark C

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Immunology, Stem Cell Research - Umbilical Cord Blood/ Placenta, Stem Cell Research - Nonembryonic - Human, Clinical Research, Transplantation, Hematology, Stem Cell Research, Regenerative Medicine, Stem Cell Research - Nonembryonic - Non-Human, Pediatric, Stem Cell Research - Umbilical Cord Blood/ Placenta - Human, 5.2 Cellular and gene therapies, Development of treatments and therapeutic interventions, Good Health and Well Being, Adolescent, Bone Marrow Transplantation, Child, Child, Preschool, Cord Blood Stem Cell Transplantation, Feasibility Studies, Female, Follow-Up Studies, Graft Survival, HLA Antigens, Hematopoietic Stem Cell Transplantation, Hemoglobinopathies, Histocompatibility Testing, Humans, Infant, Male, Prognosis, Survival Rate, bone marrow transplant, cord blood transplant, hemoglobinopathy, sickle cell disease, thalassemia, Clinical Sciences, Oncology and Carcinogenesis, Paediatrics and Reproductive Medicine, Oncology & Carcinogenesis, Oncology and carcinogenesis, Paediatrics
Abstract

BackgroundIt is well established that umbilical cord blood and bone marrow are biologically different stem cell sources.Patients and methodsWe analyzed the feasibility and outcome of hematopoietic stem cell transplantation (HSCT) in 13 children (median age 5.9 years) with hemoglobinopathies after the co- infusion of cord blood (CB) and bone marrow (BM) from the same human leucocyte antigen (HLA) identical sibling donor. We also compared outcomes of children with co-transplantation to outcomes in children with hemoglobinopathies who had received a BM (n = 21) or CB (n = 22) transplant alone.ResultsCompared to CB transplant (CBT) recipients, the co-transplant group had more rapid neutrophil (17 vs. 25 days, P = 0.013) and platelet (29 vs. 48 days, P = 0.009) recovery and less transplant related mortality. Patients who received a co-transplant had a lower incidence of ≥ grade II acute (0% vs. 26.3%) and chronic (0% vs. 21%) graft versus host disease (GVHD) compared to BM transplant (BMT) recipients (P = 0.055 and 0.045, respectively). With a median follow-up of >60 months in each treatment group, the 5-year probability of event free survival (EFS) was 100% in the co-transplant group, 90% after BMT and 86% after CBT (P = 0.42).ConclusionCo-transplantation of CB and BM from HLA-identical sibling donors appears to be a feasible and effective strategy to further optimize outcomes of HSCT for hemoglobinopathies.

Published
2014

22. T-cell depleted allogeneic hematopoietic stem cell transplant for the treatment of Fanconi anemia and MDS/AML

Author

Satty, Alexandra M., primary, Klein, Elizabeth, additional, Mauguen, Audrey, additional, Kunvarjee, Binni, additional, Boelens, Jaap Jan, additional, Cancio, Maria, additional, Curran, Kevin J., additional, Kernan, Nancy A., additional, Prockop, Susan E., additional, Scaradavou, Andromachi, additional, Spitzer, Barbara, additional, Tamari, Roni, additional, Ruggiero, Julianne, additional, Torok-Castanza, Joanne, additional, Mehta, Parinda A., additional, O’Reilly, Richard J., additional, and Boulad, Farid, additional

Published
2023
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24. Understanding the evolving phenotype of vascular complications in telomere biology disorders

Author

Higgs, Cecilia, Crow, Yanick J., Adams, Denise M., Chang, Emmanuel, Hayes, Jr., Don, Herbig, Utz, Huang, James N., Himes, Ryan, Jajoo, Kunal, Johnson, F. Brad, Reynolds, Susan D., Yonekawa, Yoshihiro, Armanios, Mary, Boulad, Farid, DiNardo, Courtney D., Dufour, Carlo, Goldman, Frederick D., Khan, Shakila, Kratz, Christian, Myers, Kasiani C., Raghu, Ganesh, Alter, Blanche P., Aubert, Geraldine, Bhala, Sonia, Cowen, Edward W., Dror, Yigal, El-Youssef, Mounif, Friedman, Bruce, Giri, Neelam, Helms Guba, Lisa, Khincha, Payal P., Lin, Tiffany F., Longhurst, Hilary, McReynolds, Lisa J., Nelson, Adam, Olson, Tim, Pariser, Anne, Perona, Rosario, Sasa, Ghadir, Schratz, Kristen, Simonetto, Douglas A., Townsley, Danielle, Walsh, Michael, Stevens, Katherine, Agarwal, Suneet, Bertuch, Alison A., Savage, Sharon A., and the Clinical Care Consortium for Telomere-associated Ailments (CCCTAA)

Published
2019
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25. A Chemotherapy-Only Regimen of Busulfan, Melphalan, and Fludarabine, and Rabbit Antithymocyte Globulin Followed by Allogeneic T-Cell Depleted Hematopoietic Stem Cell Transplantations for the Treatment of Myeloid Malignancies

Author

Spitzer, Barbara, Jakubowski, Ann A., Papadopoulos, Esperanza B., Fuller, Kirsten, Hilden, Patrick D., Young, James W., Barker, Juliet N., Koehne, Guenther, Perales, Miguel-Angel, Hsu, Katharine C., van den Brink, Marcel R., Kernan, Nancy A., Prockop, Susan E., Scaradavou, Andromachi, Castro-Malaspina, Hugo, O'Reilly, Richard J., and Boulad, Farid

Published
2017
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27. T Cell Depletion as an Alternative Approach for Patients 55 Years or Older Undergoing Allogeneic Stem Cell Transplantation as Curative Therapy for Hematologic Malignancies

Author

Jakubowski, Ann A., Petrlik, Erica, Maloy, Molly, Hilden, Patrick, Papadopoulos, Esperanza, Young, James W., Boulad, Farid, Castro-Malaspina, Hugo, Tamari, Roni, Dahi, Parastoo B., Goldberg, Jenna, Koehne, Guenther, Perales, Miguel-Angel, Sauter, Craig S., O'Reilly, Richard J., and Giralt, Sergio

Published
2017
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28. Late Effects Screening Guidelines after Hematopoietic Cell Transplantation for Inherited Bone Marrow Failure Syndromes: Consensus Statement From the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects After Pediatric HCT

Author

Dietz, Andrew C., Savage, Sharon A., Vlachos, Adrianna, Mehta, Parinda A., Bresters, Dorine, Tolar, Jakub, Bonfim, Carmem, Dalle, Jean Hugues, de la Fuente, Josu, Skinner, Roderick, Boulad, Farid, Duncan, Christine N., Baker, K. Scott, Pulsipher, Michael A., Lipton, Jeffrey M., Wagner, John E., and Alter, Blanche P.

Published
2017
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29. Current Knowledge and Priorities for Future Research in Late Effects after Hematopoietic Cell Transplantation for Inherited Bone Marrow Failure Syndromes: Consensus Statement from the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects after Pediatric Hematopoietic Cell Transplantation

Author

Dietz, Andrew C., Mehta, Parinda A., Vlachos, Adrianna, Savage, Sharon A., Bresters, Dorine, Tolar, Jakub, Boulad, Farid, Dalle, Jean Hugues, Bonfim, Carmem, de la Fuente, Josu, Duncan, Christine N., Baker, K. Scott, Pulsipher, Michael A., Lipton, Jeffrey M., Wagner, John E., and Alter, Blanche P.

Published
2017
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33. Third party CMV viral specific T-cells for refractory CMV viremia and disease after hematopoietic transplant

Author

Prockop, Susan E., primary, Hasan, Aisha N., additional, Doubrovina, Ekaterina, additional, Dahi, Parastoo B., additional, Rodriguez-Sanchez, M. Irene, additional, Curry, Michael, additional, Mauguen, Audrey, additional, Papanicolaou, Genovefa A., additional, Su, Yiqi, additional, Yao, JinJuan, additional, Arcila, Maria E., additional, Boulad, Farid, additional, Castro-Malaspina, Hugo, additional, Cho, Christina, additional, Curran, Kevin J., additional, Giralt, Sergio, additional, Kernan, Nancy A, additional, Koehne, Guenther, additional, Jakubowski, Ann, additional, Papadopoulos, Esperanza, additional, Perales, Miguel-Angel, additional, Politikos, Ioannis, additional, Price, Keith J., additional, Selvakumar, Annamalai, additional, Sauter, Craig S., additional, Tamari, Roni, additional, Vizconde, Teresa, additional, Young, James W., additional, and O'Reilly, Richard J., additional

Published
2023
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36. Impact of Rabbit Anti-Thymocyte Globulin (rATG) Exposure in Ex Vivo T-Cell Depleted Hematopoietic Cell Transplantation in Children and Young Adults

Author

Lakkaraja, Madhavi, primary, Mauguen, Audrey, additional, Boulad, Farid, additional, Cancio, Maria I., additional, Curran, Kevin J., additional, Harris, Andrew C., additional, Kernan, Nancy A., additional, Klein, Elizabeth, additional, Kung, Andrew L., additional, Oved, Joseph H., additional, Prockop, Susan E., additional, Scaradavou, Andromachi, additional, Spitzer, Barbara, additional, O’Reilly, Richard J., additional, and Boelens, Jaap-Jan, additional

Published
2023
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37. Excellent Progress in Overall Transplant Outcomes for Patients with Fanconi Anemia Including Adult FA Patients and Those with Myelodysplastic Syndrome Using Risk Adjusted Cytoreduction without Radiation

Author

Mehta, Parinda A., primary, Wilhelm, Jamie, additional, Leemhuis, Thomas, additional, Baker, K. Scott, additional, Boulad, Farid, additional, Myers, Kasiani, additional, Mallhi, Kanwaldeep, additional, Harris, Michelle, additional, Brooks, Kaitlin, additional, Mueller, Robin, additional, Goodridge, Erica, additional, Hunter, Melissa, additional, Lane, Adam, additional, and Davies, Stella M., additional

Published
2023
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39. Hematopoietic stem cell transplantation for infantile osteopetrosis

Author

Orchard, Paul J., Fasth, Anders L., Le Rademacher, Jennifer, He, Wensheng, Boelens, Jaap Jan, Horwitz, Edwin M., Al-Seraihy, Amal, Ayas, Mouhab, Bonfim, Carmem M., Boulad, Farid, Lund, Troy, Buchbinder, David K., Kapoor, Neena, O'Brien, Tracey A., Perez, Miguel A. Diaz, Veys, Paul A., and Eapen, Mary

Published
2015
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41. Transplant results in adults with Fanconi anaemia

Author

Bierings, Marc, Bonfim, Carmem, Peffault De Latour, Regis, Aljurf, Mahmoud, Mehta, Parinda A., Knol, Cora, Boulad, Farid, Tbakhi, Abdelghani, Esquirol, Albert, McQuaker, Grant, Sucak, Gulsan A., Othman, Tarek B., Halkes, Constantijn J. M., Carpenter, Ben, Niederwieser, Dietger, Zecca, Marco, Kröger, Nicolaus, Michallet, Mauricette, Risitano, Antonio M., Ehninger, Gerhard, Porcher, Raphael, Dufour, Carlo, Bonfirm, Carmem, Socié, Gerard, Gurman, Gunham, Ghavamzadeh, Ardesir, Hamladji, Rise‐Marie, van Lint, M.T., Stepensky, Polina, Koh, Mickey, Ozkurt, Zubeyde Nur, Veelken, Joan Hendrik, Bunjes, Donald, Beelen, Dietrich, Campos, Antonio, Robinson, Stephen, Alessandrino, E. Paolo, Unal, Ali, Fernandez Navarro, José Maria, Mufti, G.J., Velardi, Andrea, Passweg, Jakob, Apperley, Jane, Sengeloev, Henrik, Ljungman, Per, Foá, Roberto, Alegre, Adrián, Espiga, Carlos Richard, Cornelissen, J.J., Di Bartolomeo, Paolo, Cordonnier, Catherine, Browne, Paul, Jubert, Charlotte, Gastl, Günther, Pierelli, Luca, Johansson, Jan‐Erik, Fagioli, Franca, Moraleda, José, Zuckerman, Tsila, Bazarbachi, Ali, Sedlacek, Petr, Rössig, Claudia, Wynn, R.F., Hallek, Michael, Toren, Amos, Zudaire, Teresa, Clausen, Joahannes, Spencer, Andrew, Grazon Lopez, Sebastian, Schots, Rik, Komarnicki, M., González Muniz, Soledad, Vitek, Antonin, Rambaldi, Alessandro, Merli, Francesco, Rubio, Marie Thérese, Rossig, Claudia, Cabrera Marín, José Rafael, Porto, Fulvio, Kerre, Tessa, Metzner, Bernd, Stein, Jerry, Bertrand, Yves, Ciceri, Fabio, Chybicka, Alicja, Diez‐Martin, J.L., Bayoumy, Mohamed, de la Fuente, Josu, and Fegueux, Nathalie

Published
2018
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42. Abstract 6196: Fanconi anemia pathway deficiency drives copy number variation in squamous cell carcinoma

Author

Webster, Andrew L., primary, Sanders, Mathijs A., additional, Patel, Krupa, additional, Dietrich, Ralf, additional, Noonan, Raymond J., additional, Lach, Francis P., additional, White, Ryan R., additional, Goldfarb, Audrey M., additional, Hadi, Kevin, additional, Edwards, Matthew M., additional, Donovan, Frank X., additional, Jung, Moonjung, additional, Sridhar, Sunandini, additional, Fedrigo, Olivier, additional, Tian, Huasong, additional, Rosiene, Joel, additional, Heineman, Thomas, additional, Kennedy, Jennifer, additional, Bean, Lorenzo, additional, Rosti, Rasim O., additional, Tryon, Rebecca, additional, Gonzalez, Ashlyn-Maree, additional, Rosenberg, Allana, additional, Luo, Ji-Dung, additional, Carrol, Thomas, additional, Velleuer, Eunike, additional, Rastatter, Jeff C., additional, Wells, Susanne I., additional, Surrallés, Jordi, additional, Bagby, Grover, additional, MacMillan, Margaret L., additional, Wagner, John E., additional, Cancio, Maria, additional, Boulad, Farid, additional, Scognamiglio, Theresa, additional, Vaughan, Roger, additional, Koren, Amnon, additional, Imielinski, Marcin, additional, Chandrasekharappa, Settara, additional, Auerbach, Arleen D., additional, Singh, Bhuvanesh, additional, Kutler, David, additional, Campbell, Peter J., additional, and Smogorzewska, Agata, additional

Published
2022
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44. T Cell–Depleted Stem Cell Transplantation for Adults with High-Risk Acute Lymphoblastic Leukemia: Long-Term Survival for Patients in First Complete Remission with a Decreased Risk of Graft-versus-Host Disease

Author

Goldberg, Jenna D., Linker, Alex, Kuk, Deborah, Ratan, Ravin, Jurcic, Joseph, Barker, Juliet N., Castro-Malaspina, Hugo, Giralt, Sergio, Hsu, Katharine, Jakubowski, Ann A., Jenq, Robert, Koehne, Guenther, Papadopoulos, Esperanza B., van den Brink, Marcel R.M., Young, James W., Boulad, Farid, Kernan, Nancy A., O’Reilly, Richard J., Prockop, Susan E., Yahalom, Joachim, Heller, Glenn, and Perales, Miguel-Angel

Published
2013
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45. Adoptive immunotherapy with unselected or EBV-specific T cells for biopsy-proven EBV+ lymphomas after allogeneic hematopoietic cell transplantation

Author

Doubrovina, Ekaterina, Oflaz-Sozmen, Banu, Prockop, Susan E., Kernan, Nancy A., Abramson, Sara, Teruya-Feldstein, Julie, Hedvat, Cyrus, Chou, Joanne F., Heller, Glenn, Barker, Juliet N., Boulad, Farid, Castro-Malaspina, Hugo, George, Diane, Jakubowski, Ann, Koehne, Guenther, Papadopoulos, Esperanza B., Scaradavou, Andromachi, Small, Trudy N., Khalaf, Ramzi, Young, James W., and O'Reilly, Richard J.

Published
2012
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46. Sequence and Structural Convergence of Broad and Potent HIV Antibodies That Mimic CD4 Binding

Author

Scheid, Johannes F., Mouquet, Hugo, Ueberheide, Beatrix, Diskin, Ron, Klein, Florian, Oliveira, Thiago Y. K., Pietzsch, John, Fenyo, David, Abadir, Alexander, Velinzon, Klara, Hurley, Arlene, Myung, Sunnie, Boulad, Farid, Poignard, Pascal, Burton, Dennis R., Pereyra, Florencia, Ho, David D., Walker, Bruce D., Seaman, Michael S., Bjorkman, Pamela J., Chait, Brian T., and Nussenzweig, Michel C.

Published
2011
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47. Reduced Late Mortality Risk Contributes to Similar Survival after Double-Unit Cord Blood Transplantation Compared with Related and Unrelated Donor Hematopoietic Stem Cell Transplantation

Author

Ponce, Doris M., Zheng, Junting, Gonzales, Anne Marie, Lubin, Marissa, Heller, Glenn, Castro-Malaspina, Hugo, Giralt, Sergio, Hsu, Katharine, Jakubowski, Ann A., Jenq, Robert R., Koehne, Guenther, Papadopoulos, Esperanza B., Perales, Miguel A., van den Brink, Marcel R., Young, James W., Boulad, Farid, Kernan, Nancy A., Kobos, Rachel, Prockop, Susan, Scaradavou, Andromachi, Small, Trudy, O’Reilly, Richard J., and Barker, Juliet N.

Published
2011
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