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9. Bakeries Go Pro: The integration of protein into baking represents a profound shift that combines nutritional enhancement with the intrinsic pleasure of baked goods

Author

Canonne, Sebastien, Ghotra, Baljit, and Bhatia, Monica

Subjects
Amino acids, Business, Food and beverage industries
Abstract

The evolution that brought the protein fortification trend into baking goes beyond mere recipe adaptation; it represents a deep recognition of protein's multifaceted role in dietary wellness in addition to [...]

Published
2024

10. 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

11. A diagnostic classifier for pediatric chronic graft-versus-host disease: results of the ABLE/PBMTC 1202 study

Author

Cuvelier, Geoffrey D. E., Ng, Bernard, Abdossamadi, Sayeh, Nemecek, Eneida R., Melton, Alexis, Kitko, Carrie L., Lewis, Victor A., Schechter, Tal, Jacobsohn, David A., Harris, Andrew C., Pulsipher, Michael A., Bittencourt, Henrique, Choi, Sung Won, Caywood, Emi H., Kasow, Kimberly A., Bhatia, Monica, Oshrine, Benjamin R., Chaudhury, Sonali, Coulter, Donald, Chewning, Joseph H., Joyce, Michael, Savaşan, Süreyya, Pawlowska, Anna B., Megason, Gail C., Mitchell, David, Cheerva, Alexandra C., Lawitschka, Anita, Ostroumov, Elena, and Schultz, Kirk R.

Published
2023
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13. Hematopoietic Cell Transplantation in Patients With Primary Immune Regulatory Disorders (PIRD): A Primary Immune Deficiency Treatment Consortium (PIDTC) Survey.

Author

Chan, Alice Y, Leiding, Jennifer W, Liu, Xuerong, Logan, Brent R, Burroughs, Lauri M, Allenspach, Eric J, Skoda-Smith, Suzanne, Uzel, Gulbu, Notarangelo, Luigi D, Slatter, Mary, Gennery, Andrew R, Smith, Angela R, Pai, Sung-Yun, Jordan, Michael B, Marsh, Rebecca A, Cowan, Morton J, Dvorak, Christopher C, Craddock, John A, Prockop, Susan E, Chandrakasan, Shanmuganathan, Kapoor, Neena, Buckley, Rebecca H, Parikh, Suhag, Chellapandian, Deepak, Oshrine, Benjamin R, Bednarski, Jeffrey J, Cooper, Megan A, Shenoy, Shalini, Davila Saldana, Blachy J, Forbes, Lisa R, Martinez, Caridad, Haddad, Elie, Shyr, David C, Chen, Karin, Sullivan, Kathleen E, Heimall, Jennifer, Wright, Nicola, Bhatia, Monica, Cuvelier, Geoffrey DE, Goldman, Frederick D, Meyts, Isabelle, Miller, Holly K, Seidel, Markus G, Vander Lugt, Mark T, Bacchetta, Rosa, Weinacht, Katja G, Andolina, Jeffrey R, Caywood, Emi, Chong, Hey, de la Morena, Maria Teresa, Aquino, Victor M, Shereck, Evan, Walter, Jolan E, Dorsey, Morna J, Seroogy, Christine M, Griffith, Linda M, Kohn, Donald B, Puck, Jennifer M, Pulsipher, Michael A, and Torgerson, Troy R

Subjects
Animals, Humans, Treatment Outcome, Hematopoietic Stem Cell Transplantation, Adolescent, Adult, Middle Aged, Child, Child, Preschool, Infant, T-Lymphocytes, Regulatory, Young Adult, Surveys and Questionnaires, Primary Immunodeficiency Diseases, autoimmunity, genetics, hematopoietic cell transplant, immune dysregulation, primary immune deficiencies, Clinical Research, Rare Diseases, Transplantation, 5.2 Cellular and gene therapies, Development of treatments and therapeutic interventions, Inflammatory and immune system, Immunology, Medical Microbiology
Abstract

Primary Immune Regulatory Disorders (PIRD) are an expanding group of diseases caused by gene defects in several different immune pathways, such as regulatory T cell function. Patients with PIRD develop clinical manifestations associated with diminished and exaggerated immune responses. Management of these patients is complicated; oftentimes immunosuppressive therapies are insufficient, and patients may require hematopoietic cell transplant (HCT) for treatment. Analysis of HCT data in PIRD patients have previously focused on a single gene defect. This study surveyed transplanted patients with a phenotypic clinical picture consistent with PIRD treated in 33 Primary Immune Deficiency Treatment Consortium centers and European centers. Our data showed that PIRD patients often had immunodeficient and autoimmune features affecting multiple organ systems. Transplantation resulted in resolution of disease manifestations in more than half of the patients with an overall 5-years survival of 67%. This study, the first to encompass disorders across the PIRD spectrum, highlights the need for further research in PIRD management.

Published
2020

14. Reproductive Health Assessment and Reports of Fertility Counseling in Pediatric and Adolescent Patients with Sickle Cell Disease After Hematopoietic Cell Transplantation

Author

George, Sobenna A., Veludhandi, Anirudh, Xiang, Yijin, Liu, Katie, Stenger, Elizabeth, Arnold, Staci D., Mehta, Akanksha, Schirmer, David A., Spencer, Jessica B., Guilcher, Gregory M.T., Bhatia, Monica, Abraham, Allistair, Gomez-Lobo, Veronica, Krishnamurti, Lakshmanan, and Meacham, Lillian R.

Published
2024
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15. Metabolomic identification of α-ketoglutaric acid elevation in pediatric chronic graft-versus-host disease

Author

Subburaj, Divya, Ng, Bernard, Kariminia, Amina, Abdossamadi, Sayeh, Lauener, Madeline, Nemecek, Eneida R., Rozmus, Jacob, Kharbanda, Sandhya, Kitko, Carrie L., Lewis, Victor A., Schechter-Finklestein, Tal, Jacobsohn, David A., Harris, Andrew C., Pulsipher, Michael A., Bittencourt, Henrique, Choi, Sung Won, Caywood, Emi H., Kasow, Kimberly A., Bhatia, Monica, Oshrine, Benjamin R., Coulter, Donald, Chewning, Joseph H., Joyce, Michael, Pawlowska, Anna B., Megason, Gail C., Lawitschka, Anita, Ostroumov, Elena, Klein Geltink, Ramon, Cuvelier, Geoffrey D.E., and Schultz, Kirk R.

Published
2022
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16. Outcomes of Measurable Residual Disease in Pediatric Acute Myeloid Leukemia before and after Hematopoietic Stem Cell Transplant: Validation of Difference from Normal Flow Cytometry with Chimerism Studies and Wilms Tumor 1 Gene Expression

Author

Jacobsohn, David A, Loken, Michael R, Fei, Mingwei, Adams, Alexia, Brodersen, Lisa Eidenschink, Logan, Brent R, Ahn, Kwang Woo, Shaw, Bronwen E, Kletzel, Morris, Olszewski, Marie, Khan, Sana, Meshinchi, Soheil, Keating, Amy, Harris, Andrew, Teira, Pierre, Duerst, Reggie E, Margossian, Steven P, Martin, Paul L, Petrovic, Aleksandra, Dvorak, Christopher C, Nemecek, Eneida R, Boyer, Michael W, Chen, Allen R, Davis, Jeffrey H, Shenoy, Shalini, Savasan, Sureyya, Hudspeth, Michelle P, Adams, Roberta H, Lewis, Victor A, Kheradpour, Albert, Kasow, Kimberly A, Gillio, Alfred P, Haight, Ann E, Bhatia, Monica, Bambach, Barbara J, Haines, Hilary L, Quigg, Troy C, Greiner, Robert J, Talano, Julie-An M, Delgado, David C, Cheerva, Alexandra, Gowda, Madhu, Ahuja, Sanjay, Ozkaynak, Mehmet, Mitchell, David, Schultz, Kirk R, Fry, Terry J, Loeb, David M, and Pulsipher, Michael A

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Pediatric, Rare Diseases, Stem Cell Research - Nonembryonic - Human, Clinical Research, Transplantation, Pediatric Research Initiative, Hematology, Regenerative Medicine, Genetics, Stem Cell Research, Cancer, Pediatric Cancer, Detection, screening and diagnosis, 4.2 Evaluation of markers and technologies, Inflammatory and immune system, Adolescent, Adult, Allografts, Child, Child, Preschool, Disease-Free Survival, Female, Flow Cytometry, Hematopoietic Stem Cell Transplantation, Humans, Infant, Infant, Newborn, Leukemia, Myeloid, Acute, Male, Neoplasm, Residual, Transplantation Conditioning, Transplantation, Homologous, Unrelated Donors, WT1 Proteins, Cytogenetics and molecular genetics, Measurable residual disease, Stem cell transplantation, Laboratory hematology, Clinical Sciences, Immunology, Cardiovascular medicine and haematology
Abstract

We enrolled 150 patients in a prospective multicenter study of children with acute myeloid leukemia undergoing hematopoietic stem cell transplantation (HSCT) to compare the detection of measurable residual disease (MRD) by a "difference from normal" flow cytometry (ΔN) approach with assessment of Wilms tumor 1 (WT1) gene expression without access to the diagnostic specimen. Prospective analysis of the specimens using this approach showed that 23% of patients screened for HSCT had detectable residual disease by ΔN (.04% to 53%). Of those patients who proceeded to transplant as being in morphologic remission, 10 had detectable disease (.04% to 14%) by ΔN. The disease-free survival of this group was 10% (0 to 35%) compared with 55% (46% to 64%, P < .001) for those without disease. The ΔN assay was validated using the post-HSCT specimen by sorting abnormal or suspicious cells to confirm recipient or donor origin by chimerism studies. All 15 patients who had confirmation of tumor detection relapsed, whereas the 2 patients with suspicious phenotype cells lacking this confirmation did not. The phenotype of the relapse specimen was then used retrospectively to assess the pre-HSCT specimen, allowing identification of additional samples with low levels of MRD involvement that were previously undetected. Quantitative assessment of WT1 gene expression was not predictive of relapse or other outcomes in either pre- or post-transplant specimens. MRD detected by ΔN was highly specific, but did not identify most relapsing patients. The application of the assay was limited by poor quality among one-third of the specimens and lack of a diagnostic phenotype for comparison.

Published
2018

17. Long-term follow-up of IPEX syndrome patients after different therapeutic strategies: An international multicenter retrospective study.

Author

Barzaghi, Federica, Amaya Hernandez, Laura Cristina, Neven, Benedicte, Ricci, Silvia, Kucuk, Zeynep Yesim, Bleesing, Jack J, Nademi, Zohreh, Slatter, Mary Anne, Ulloa, Erlinda Rose, Shcherbina, Anna, Roppelt, Anna, Worth, Austen, Silva, Juliana, Aiuti, Alessandro, Murguia-Favela, Luis, Speckmann, Carsten, Carneiro-Sampaio, Magda, Fernandes, Juliana Folloni, Baris, Safa, Ozen, Ahmet, Karakoc-Aydiner, Elif, Kiykim, Ayca, Schulz, Ansgar, Steinmann, Sandra, Notarangelo, Lucia Dora, Gambineri, Eleonora, Lionetti, Paolo, Shearer, William Thomas, Forbes, Lisa R, Martinez, Caridad, Moshous, Despina, Blanche, Stephane, Fisher, Alain, Ruemmele, Frank M, Tissandier, Come, Ouachee-Chardin, Marie, Rieux-Laucat, Frédéric, Cavazzana, Marina, Qasim, Waseem, Lucarelli, Barbarella, Albert, Michael H, Kobayashi, Ichiro, Alonso, Laura, Diaz De Heredia, Cristina, Kanegane, Hirokazu, Lawitschka, Anita, Seo, Jong Jin, Gonzalez-Vicent, Marta, Diaz, Miguel Angel, Goyal, Rakesh Kumar, Sauer, Martin G, Yesilipek, Akif, Kim, Minsoo, Yilmaz-Demirdag, Yesim, Bhatia, Monica, Khlevner, Julie, Richmond Padilla, Erick J, Martino, Silvana, Montin, Davide, Neth, Olaf, Molinos-Quintana, Agueda, Valverde-Fernandez, Justo, Broides, Arnon, Pinsk, Vered, Ballauf, Antje, Haerynck, Filomeen, Bordon, Victoria, Dhooge, Catharina, Garcia-Lloret, Maria Laura, Bredius, Robbert G, Kałwak, Krzysztof, Haddad, Elie, Seidel, Markus Gerhard, Duckers, Gregor, Pai, Sung-Yun, Dvorak, Christopher C, Ehl, Stephan, Locatelli, Franco, Goldman, Frederick, Gennery, Andrew Richard, Cowan, Mort J, Roncarolo, Maria-Grazia, Bacchetta, Rosa, and Primary Immune Deficiency Treatment Consortium (PIDTC) and the Inborn Errors Working Party (IEWP) of the European Society for Blood and Marrow Transplantation (EBMT)

Subjects
Primary Immune Deficiency Treatment Consortium (PIDTC) and the Inborn Errors Working Party (IEWP) of the European Society for Blood and Marrow Transplantation, Humans, Genetic Diseases, X-Linked, Diabetes Mellitus, Type 1, Immune System Diseases, Diarrhea, Disease-Free Survival, Hematopoietic Stem Cell Transplantation, Survival Rate, Retrospective Studies, Follow-Up Studies, Mutation, Adolescent, Adult, Child, Child, Preschool, Infant, Female, Male, Forkhead Transcription Factors, Allografts, Immunosuppression Therapy, FOXP3, IPEX, Treg cells, enteropathy, genetic autoimmunity, hematopoietic stem cell transplantation, immunosuppression, neonatal diabetes, primary immune deficiency, rapamycin, Clinical Research, Stem Cell Research, Regenerative Medicine, Pediatric, Genetics, Transplantation, Aetiology, 2.1 Biological and endogenous factors, Immunology, Allergy
Abstract

BackgroundImmunodysregulation polyendocrinopathy enteropathy x-linked (IPEX) syndrome is a monogenic autoimmune disease caused by FOXP3 mutations. Because it is a rare disease, the natural history and response to treatments, including allogeneic hematopoietic stem cell transplantation (HSCT) and immunosuppression (IS), have not been thoroughly examined.ObjectiveThis analysis sought to evaluate disease onset, progression, and long-term outcome of the 2 main treatments in long-term IPEX survivors.MethodsClinical histories of 96 patients with a genetically proven IPEX syndrome were collected from 38 institutions worldwide and retrospectively analyzed. To investigate possible factors suitable to predict the outcome, an organ involvement (OI) scoring system was developed.ResultsWe confirm neonatal onset with enteropathy, type 1 diabetes, and eczema. In addition, we found less common manifestations in delayed onset patients or during disease evolution. There is no correlation between the site of mutation and the disease course or outcome, and the same genotype can present with variable phenotypes. HSCT patients (n = 58) had a median follow-up of 2.7 years (range, 1 week-15 years). Patients receiving chronic IS (n = 34) had a median follow-up of 4 years (range, 2 months-25 years). The overall survival after HSCT was 73.2% (95% CI, 59.4-83.0) and after IS was 65.1% (95% CI, 62.8-95.8). The pretreatment OI score was the only significant predictor of overall survival after transplant (P = .035) but not under IS.ConclusionsPatients receiving chronic IS were hampered by disease recurrence or complications, impacting long-term disease-free survival. When performed in patients with a low OI score, HSCT resulted in disease resolution with better quality of life, independent of age, donor source, or conditioning regimen.

Published
2018

18. Exagamglogene Autotemcel for Severe Sickle Cell Disease

Author

Frangoul, Haydar, primary, Locatelli, Franco, additional, Sharma, Akshay, additional, Bhatia, Monica, additional, Mapara, Markus, additional, Molinari, Lyndsay, additional, Wall, Donna, additional, Liem, Robert I., additional, Telfer, Paul, additional, Shah, Ami J., additional, Cavazzana, Marina, additional, Corbacioglu, Selim, additional, Rondelli, Damiano, additional, Meisel, Roland, additional, Dedeken, Laurence, additional, Lobitz, Stephan, additional, de Montalembert, Mariane, additional, Steinberg, Martin H., additional, Walters, Mark C., additional, Eckrich, Michael J., additional, Imren, Suzan, additional, Bower, Laura, additional, Simard, Christopher, additional, Zhou, Weiyu, additional, Xuan, Fengjuan, additional, Morrow, Phuong Khanh, additional, Hobbs, William E., additional, and Grupp, Stephan A., additional

Published
2024
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19. Preservation of Pulmonary Function Following Hematopoietic Cell Transplant for Sickle Cell Disease: A STAR Study

Author

Horan, Fiona, primary, Bendiak, Glenda, additional, Abraham, Allistair, additional, Liu, Katie, additional, Gillespie, Scott, additional, Chellapandian, Deepak, additional, Shah, Rikin K, additional, Bhatia, Monica, additional, Chaudhury, Sonali, additional, Eckrich, Michael, additional, Jaroscak, Dr. Jennifer Joi, additional, Kasow, Kimberly A., additional, Krajewski, Jennifer A., additional, Ngwube, Alexander, additional, Horan, John, additional, Krishnamurti, Lakshmanan, additional, Shenoy, Shalini, additional, Guilcher, Gregory MT, additional, and Stenger, Elizabeth O., additional

Published
2024
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20. Elimination of Vaso-Occlusive Crises after Exagamglogene Autotemcel in Patients with Severe Sickle Cell Disease

Author

Frangoul, Haydar, primary, Locatelli, Franco, additional, Sharma, Akshay, additional, Bhatia, Monica, additional, Mapara, Markus Y., additional, Molinari, Lyndsay, additional, Wall, Donna A., additional, Liem, Robert, additional, Telfer, Paul, additional, Shah, Ami J., additional, Cavazzana, Marina, additional, Corbacioglu, Selim, additional, Rondelli, Damiano, additional, Meisel, Roland, additional, Dedeken, Laurence, additional, Lobitz, Stephan, additional, de Montalembert, Mariane, additional, Steinberg, Martin H., additional, Walters, Mark, additional, Bower, Laura, additional, Imren, Suzan, additional, Shi, Daoyuan, additional, Simard, Christopher, additional, Zhang, Lanju, additional, Morrow, Phuong Khanh, additional, Hobbs, William, additional, and Grupp, Stephan A., additional

Published
2024
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21. Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation.

Author

Gluckman, Eliane, Cappelli, Barbara, Bernaudin, Francoise, Labopin, Myriam, Volt, Fernanda, Carreras, Jeanette, Pinto Simões, Belinda, Ferster, Alina, Dupont, Sophie, de la Fuente, Josu, Dalle, Jean-Hugues, Zecca, Marco, Walters, Mark C, Krishnamurti, Lakshmanan, Bhatia, Monica, Leung, Kathryn, Yanik, Gregory, Kurtzberg, Joanne, Dhedin, Nathalie, Kuentz, Mathieu, Michel, Gerard, Apperley, Jane, Lutz, Patrick, Neven, Bénédicte, Bertrand, Yves, Vannier, Jean Pierre, Ayas, Mouhab, Cavazzana, Marina, Matthes-Martin, Susanne, Rocha, Vanderson, Elayoubi, Hanadi, Kenzey, Chantal, Bader, Peter, Locatelli, Franco, Ruggeri, Annalisa, and Eapen, Mary

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Immunology, Hematology, Prevention, Sickle Cell Disease, Stem Cell Research, Rare Diseases, Clinical Research, Stem Cell Research - Nonembryonic - Human, Regenerative Medicine, Transplantation, Adolescent, Anemia, Sickle Cell, Child, Child, Preschool, Disease-Free Survival, Female, Graft Survival, HLA Antigens, Hematopoietic Stem Cell Transplantation, Histocompatibility, Humans, Infant, Male, Siblings, Surveys and Questionnaires, Survival Rate, Transplantation Conditioning, Treatment Outcome, Eurocord, the Pediatric Working Party of the European Society for Blood and Marrow Transplantation, and the Center for International Blood and Marrow Transplant Research, Cardiorespiratory Medicine and Haematology, Clinical Sciences, Paediatrics and Reproductive Medicine, Biochemistry and cell biology, Cardiovascular medicine and haematology, Paediatrics
Abstract

Despite advances in supportive therapy to prevent complications of sickle cell disease (SCD), access to care is not universal. Hematopoietic cell transplantation is, to date, the only curative therapy for SCD, but its application is limited by availability of a suitable HLA-matched donor and lack of awareness of the benefits of transplant. Included in this study are 1000 recipients of HLA-identical sibling transplants performed between 1986 and 2013 and reported to the European Society for Blood and Marrow Transplantation, Eurocord, and the Center for International Blood and Marrow Transplant Research. The primary endpoint was event-free survival, defined as being alive without graft failure; risk factors were studied using a Cox regression models. The median age at transplantation was 9 years, and the median follow-up was longer than 5 years. Most patients received a myeloablative conditioning regimen (n = 873; 87%); the remainder received reduced-intensity conditioning regimens (n = 125; 13%). Bone marrow was the predominant stem cell source (n = 839; 84%); peripheral blood and cord blood progenitors were used in 73 (7%) and 88 (9%) patients, respectively. The 5-year event-free survival and overall survival were 91.4% (95% confidence interval, 89.6%-93.3%) and 92.9% (95% confidence interval, 91.1%-94.6%), respectively. Event-free survival was lower with increasing age at transplantation (hazard ratio [HR], 1.09; P < .001) and higher for transplantations performed after 2006 (HR, 0.95; P = .013). Twenty-three patients experienced graft failure, and 70 patients (7%) died, with the most common cause of death being infection. The excellent outcome of a cohort transplanted over the course of 3 decades confirms the role of HLA-identical sibling transplantation for children and adults with SCD.

Published
2017

23. Identification of unstable regulatory and autoreactive effector T cells that are expanded in patients with FOXP3 mutations

Author

Borna, Šimon, primary, Lee, Esmond, additional, Nideffer, Jason, additional, Ramachandran, Akshaya, additional, Wang, Bing, additional, Baker, Jeanette, additional, Mavers, Melissa, additional, Lakshmanan, Uma, additional, Narula, Mansi, additional, Garrett, Amy Kang-hee, additional, Schulze, Janika, additional, Olek, Sven, additional, Marois, Louis, additional, Gernez, Yael, additional, Bhatia, Monica, additional, Chong, Hey Jin, additional, Walter, Jolan, additional, Kitcharoensakkul, Maleewan, additional, Lang, Abigail, additional, Cooper, Megan A., additional, Bertaina, Alice, additional, Roncarolo, Maria Grazia, additional, Meffre, Eric, additional, and Bacchetta, Rosa, additional

Published
2023
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27. 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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29. Long-Term Organ Function After HCT for SCD: A Report From the Sickle Cell Transplant Advocacy and Research Alliance

Author

Stenger, Elizabeth, Xiang, Yijin, Wetzel, Martha, Gillespie, Scott, Chellapandian, Deepak, Shah, Rikin, Arnold, Staci D., Bhatia, Monica, Chaudhury, Sonali, Eckrich, Michael J., Kanter, Julie, Kasow, Kimberly A., Krajewski, Jennifer, Nickel, Robert S., Ngwube, Alexander I., Olson, Tim S., Rangarajan, Hemalatha G., Wobma, Holly, Guilcher, Gregory M.T., Horan, John T., Krishnamurti, Lakshmanan, Shenoy, Shalini, and Abraham, Allistair

Published
2023
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30. Benefits and challenges with diagnosing chronic and late acute GVHD in children using the NIH consensus criteria

Author

Cuvelier, Geoffrey D.E., Nemecek, Eneida R., Wahlstrom, Justin T., Kitko, Carrie L., Lewis, Victor A., Schechter, Tal, Jacobsohn, David A., Harris, Andrew C., Pulsipher, Michael A., Bittencourt, Henrique, Choi, Sung Won, Caywood, Emi H., Kasow, Kimberly A., Bhatia, Monica, Oshrine, Benjamin R., Flower, Allyson, Chaudhury, Sonali, Coulter, Donald, Chewning, Joseph H., Joyce, Michael, Savaşan, Süreyya, Pawlowska, Anna B., Megason, Gail C., Mitchell, David, Cheerva, Alexandra C., Lawitschka, Anita, West, Lori J., Pan, Bo, Al Hamarneh, Yazid N., Halevy, Anat, and Schultz, Kirk R.

Published
2019
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32. Hematopoietic stem cell transplantation for CD40 ligand deficiency: Results from an EBMT/ESID-IEWP-SCETIDE-PIDTC study

Author

Ferrua, Francesca, Galimberti, Stefania, Courteille, Virginie, Slatter, Mary Anne, Booth, Claire, Moshous, Despina, Neven, Benedicte, Blanche, Stephane, Cavazzana, Marina, Laberko, Alexandra, Shcherbina, Anna, Balashov, Dmitry, Soncini, Elena, Porta, Fulvio, Al-Mousa, Hamoud, Al-Saud, Bandar, Al-Dhekri, Hasan, Arnaout, Rand, Formankova, Renata, Bertrand, Yves, Lange, Andrzej, Smart, Joanne, Wolska-Kusnierz, Beata, Aquino, Victor M., Dvorak, Christopher C., Fasth, Anders, Fouyssac, Fanny, Heilmann, Carsten, Hoenig, Manfred, Schuetz, Catharina, Kelečić, Jadranka, Bredius, Robbert G.M., Lankester, Arjan C., Lindemans, Caroline A., Suarez, Felipe, Sullivan, Kathleen E., Albert, Michael H., Kałwak, Krzysztof, Barlogis, Vincent, Bhatia, Monica, Bordon, Victoria, Czogala, Wojciech, Alonso, Laura, Dogu, Figen, Gozdzik, Jolanta, Ikinciogullari, Aydan, Kriván, Gergely, Ljungman, Per, Meyts, Isabelle, Mustillo, Peter, Smith, Angela R., Speckmann, Carsten, Sundin, Mikael, Keogh, Steven John, Shaw, Peter John, Boelens, Jaap Jan, Schulz, Ansgar S., Sedlacek, Petr, Veys, Paul, Mahlaoui, Nizar, Janda, Ales, Davies, E. Graham, Fischer, Alain, Cowan, Morton J., and Gennery, Andrew Richard

Published
2019
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33. Long‐term outcomes after unrelated donor transplantation for severe sickle cell disease on the BMT CTN 0601 trial.

Author

Eapen, Mary, Kou, Jianqun, Andreansky, Martin, Bhatia, Monica, Brochstein, Joel, Chaudhury, Sonali, Haight, Ann E., Haines, Hilary, Jacobsohn, David, Jaroscak, Jennifer, Kasow, Kimberly A., Krishnamurti, Lakshmanan, Levine, John E., Leung, Kathryn, Margolis, David, Yu, Lolie C., Horowitz, Mary M., Kamani, Naynesh, Walters, Mark C., and Shenoy, Shalini

Published
2024
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34. Late Effects Screening Guidelines after Hematopoietic Cell Transplantation (HCT) for Hemoglobinopathy: Consensus Statement from the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects after Pediatric HCT

Author

Shenoy, Shalini, Gaziev, Javid, Angelucci, Emanuele, King, Allison, Bhatia, Monica, Smith, Angela, Bresters, Dorine, Haight, Anne E., Duncan, Christine N., de la Fuente, Josu, Dietz, Andrew C., Baker, K. Scott, Pulsipher, Michael A., and Walters, Mark C.

Published
2018
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38. Current Results and Future Research Priorities in Late Effects after Hematopoietic Stem Cell Transplantation for Children with Sickle Cell Disease and Thalassemia: A Consensus Statement from the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects after Pediatric Hematopoietic Stem Cell Transplantation

Author

Shenoy, Shalini, Angelucci, Emanuele, Arnold, Staci D., Baker, K. Scott, Bhatia, Monica, Bresters, Dorine, Dietz, Andrew C., De La Fuente, Josu, Duncan, Christine, Gaziev, Javid, King, Allison A., Pulsipher, Michael A., Smith, Angela R., and Walters, Mark C.

Published
2017
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41. CHAPTER 4: WORK AND SUSTAINABILITY AT TWIN OAKS INTENTIONAL COMMUNITY.

Author

Bhatia, Monica

Subjects
COMMUNAL living, SUSTAINABILITY, COOPERATIVE housing, SOCIAL reproduction, ENERGY consumption, DECISION making, SOCIAL services
Abstract

A growing body of literature suggests that creating a just sustainable society will require reorganizing economic arrangements and, in particular, rethinking work. Previous studies have recognized alternative organizations, such as cooperatives and intentional communities, as sites for building more democratic, sustainable models of work. This study contributes a description and analysis of work and sustainability at Twin Oaks Intentional Community, an 80-person income- and resource-sharing commune in Louisa, Virginia. Some measures show that Twin Oaks members live more sustainably in terms of energy consumption than the average US resident. In this article, I investigate the relationship between sustainability and work at Twin Oaks. I find that sustainable work is linked to the following key principles: broadening definitions of work, prioritizing community well-being, and democratizing decision making. In doing so, I contribute to previous literature on work, sustainability, and alternative organizations by suggesting that (1) sustainability in intentional communities is deeply intertwined with systems of work; (2) broadening definitions of work to include social reproductive labour contributes to sustainability; and (3) the democratization of work can further goals of sustainability. [ABSTRACT FROM AUTHOR]

Published
2023
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44. Bacterial bloodstream infections in pediatric allogeneic hematopoietic stem cell recipients before and after implementation of a central line-associated bloodstream infection protocol: A single-center experience

Author

Chang, Alicia K., Foca, Marc D., Jin, Zhezhen, Vasudev, Rahul, Laird, Mary, Schwartz, Sharon, Qureshi, Mahvish, Kolb, Michelle, Levinson, Anya, Bhatia, Monica, Kung, Andrew, Garvin, James, George, Diane, Della-Latta, Phyllis, Whittier, Susan, Saiman, Lisa, and Satwani, Prakash

Published
2016
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45. A trial of unrelated donor marrow transplantation for children with severe sickle cell disease

Author

Shenoy, Shalini, Eapen, Mary, Panepinto, Julie A., Logan, Brent R., Wu, Juan, Abraham, Allistair, Brochstein, Joel, Chaudhury, Sonali, Godder, Kamar, Haight, Ann E., Kasow, Kimberly A., Leung, Kathryn, Andreansky, Martin, Bhatia, Monica, Dalal, Jignesh, Haines, Hilary, Jaroscak, Jennifer, Lazarus, Hillard M., Levine, John E., Krishnamurti, Lakshmanan, Margolis, David, Megason, Gail C., Yu, Lolie C., Pulsipher, Michael A., Gersten, Iris, DiFronzo, Nancy, Horowitz, Mary M., Walters, Mark C., and Kamani, Naynesh

Published
2016
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47. Clinical Outcomes and Long-Term Follow-up of Children with Mixed Myeloid Chimerism Following Hematopoietic Cell Transplantation (HCT) for Sickle Cell Disease (SCD)

Author

Guilcher, Gregory MT, primary, Dalal, Stuti, additional, Suresh, Tara, additional, Yelamali, Aditya R., additional, Aljayyousi, Haneen, additional, Bhatia, Monica, additional, Haight, Ann E, additional, Khandelwal, Pooja, additional, Khoury, Ruby, additional, Lafay, Quitterie, additional, Mandava, Mamatha, additional, Nickel, Robert Sheppard, additional, Ngwube, Alexander, additional, Rangarajan, Hemalatha G., additional, Shah, Niketa C., additional, Stenger, Elizabeth O., additional, Yelamanchili, Nitya, additional, and Shenoy, Shalini, additional

Published
2023
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