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1. 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

2. Allogeneic hematopoietic cell transplantation is effective for p47phox chronic granulomatous disease: A Primary Immune Deficiency Treatment Consortium study

Author

Grunebaum, Eyal, Arnold, Danielle E., Logan, Brent, Parikh, Suhag, Marsh, Rebecca A., Griffith, Linda M., Mallhi, Kanwaldeep, Chellapandian, Deepak, Lim, Stephanie Si, Deal, Christin L., Kapoor, Neena, Murguía-Favela, Luis, Falcone, Emilia Liana, Prasad, Vinod K., Touzot, Fabien, Bleesing, Jack J., Chandrakasan, Shanmuganathan, Heimall, Jennifer R., Bednarski, Jeffrey J., Broglie, Larisa A., Chong, Hey Jin, Kapadia, Malika, Prockop, Susan, Dávila Saldaña, Blachy J., Schaefer, Edo, Bauchat, Andrea L., Teira, Pierre, Chandra, Sharat, Parta, Mark, 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., Kang, Elizabeth M., and Leiding, Jennifer W.

Published
2024
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3. Relevance of lymphocyte proliferation to PHA in severe combined immunodeficiency (SCID) and T cell lymphopenia

Author

Abraham, Roshini S., Basu, Amrita, Heimall, Jennifer R., Dunn, Elizabeth, Yip, Alison, Kapadia, Malika, Kapoor, Neena, Satter, Lisa Forbes, Buckley, Rebecca, O'Reilly, Richard, Cuvelier, Geoffrey D.E., Chandra, Sharat, Bednarski, Jeffrey, Chaudhury, Sonali, Moore, Theodore B., Haines, Hilary, Dávila Saldaña, Blachy J., Chellapandian, Deepakbabu, Rayes, Ahmad, Chen, Karin, Caywood, Emi, Chandrakasan, Shanmuganathan, Lugt, Mark Thomas Vander, Ebens, Christen, Teira, Pierre, Shereck, Evan, Miller, Holly, Aquino, Victor, Eissa, Hesham, Yu, Lolie C., Gillio, Alfred, Madden, Lisa, Knutsen, Alan, Shah, Ami J., DeSantes, Kenneth, Barnum, Jessie, Broglie, Larisa, Joshi, Avni Y., Kleiner, Gary, Dara, Jasmeen, Prockop, Susan, Martinez, Caridad, Mousallem, Talal, Oved, Joseph, Burroughs, Lauri, Marsh, Rebecca, Torgerson, Troy R., Leiding, Jennifer W., Pai, Sung Yun, Kohn, Donald B., Pulsipher, Michael A., Griffith, Linda M., Notarangelo, Luigi D., Cowan, Morton J., Puck, Jennifer, Dvorak, Christopher C., and Haddad, Elie

Published
2024
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4. Poor T-cell receptor β repertoire diversity early posttransplant for severe combined immunodeficiency predicts failure of immune reconstitution

Author

Delmonte, Ottavia M, Castagnoli, Riccardo, Yu, Jason, Dvorak, Christopher C, Cowan, Morton J, Saldaña, Blachy J Dávila, De Ravin, Suk See, Mamcarz, Ewelina, Chang, Catherine K, Daley, Stephen R, Griffith, Linda M, Notarangelo, Luigi D, and Puck, Jennifer M

Subjects
Biomedical and Clinical Sciences, Immunology, Hematology, Regenerative Medicine, Transplantation, Genetics, Complementarity Determining Regions, Hematopoietic Stem Cell Transplantation, Humans, Immune Reconstitution, Infant, Receptors, Antigen, T-Cell, Receptors, Antigen, T-Cell, alpha-beta, Severe Combined Immunodeficiency, Complementarity determining region 3, hematopoietic cell transplantation, high-throughput sequencing, immune reconstitution, severe combined immunodeficiency, T-cell receptor, T-cell receptor beta (TRB) diversity, T-cell receptor beta (TRB) repertoire, T-cell receptor β (TRB) diversity, T-cell receptor β (TRB) repertoire, Allergy
Abstract

BackgroundDevelopment of a diverse T-cell receptor β (TRB) repertoire is associated with immune recovery following hematopoietic cell transplantation (HCT) for severe combined immunodeficiency (SCID). High-throughput sequencing of the TRB repertoire allows evaluation of clonotype dynamics during immune reconstitution.ObjectivesWe investigated whether longitudinal analysis of the TRB repertoire would accurately describe T-cell receptor diversity and illustrate the quality of T-cell reconstitution following HCT or gene therapy for SCID.MethodsWe used high-throughput sequencing to study composition and diversity of the TRB repertoire in 27 infants with SCID at 3, 6, and 12 months and yearly posttreatment(s). Total RNA from peripheral blood was used as template to amplify TRB rearrangements.ResultsTRB sequence analysis showed poor diversity at 3 months, followed by significant improvement by 6 months after cellular therapies. Kinetics of development of TRB diversity were similar in patients with a range of underlying gene defects. However, in patients with RAG and DCLRE1C defects, HCT with no conditioning or immune suppression only resulted in lower diversity than did HCT with conditioning. HCT from a matched donor correlated with higher diversity than did HCT from a mismatched donor. Naive CD4+ T-cell count at 6 months post-HCT correlated with higher TRB diversity. A Shannon index of diversity of 5.2 or lower 3 months after HCT predicted a need for a second intervention.ConclusionsTRB repertoire after hematopoietic cell therapies for SCID provides a quantitative and qualitative measure of diversity of T-cell reconstitution and permits early identification of patients who may require a second intervention.

Published
2022

5. Posttransplantation late complications increase over time for patients with SCID: A Primary Immune Deficiency Treatment Consortium (PIDTC) landmark study

Author

Eissa, Hesham, Thakar, Monica S., Shah, Ami J., Logan, Brent R., Griffith, Linda M., Dong, Huaying, Parrott, Roberta E., O’Reilly, Richard J., Dara, Jasmeen, Kapoor, Neena, Forbes Satter, Lisa, Chandra, Sharat, Kapadia, Malika, Chandrakasan, Shanmuganathan, Knutsen, Alan, Jyonouchi, Soma C., Molinari, Lyndsay, Rayes, Ahmad, Ebens, Christen L., Teira, Pierre, Dávila Saldaña, Blachy J., Burroughs, Lauri M., Chaudhury, Sonali, Chellapandian, Deepak, Gillio, Alfred P., Goldman, Fredrick, Malech, Harry L., DeSantes, Kenneth, Cuvelier, Geoff D.E., Rozmus, Jacob, Quinones, Ralph, Yu, Lolie C., Broglie, Larisa, Aquino, Victor, Shereck, Evan, Moore, Theodore B., Vander Lugt, Mark T., Mousallem, Talal I., Oved, Joeseph H., Dorsey, Morna, Abdel-Azim, Hisham, Martinez, Caridad, Bleesing, Jacob H., Prockop, Susan, Kohn, Donald B., Bednarski, Jeffrey J., Leiding, Jennifer, Marsh, Rebecca A., Torgerson, Troy, Notarangelo, Luigi D., Pai, Sung-Yun, Pulsipher, Michael A., Puck, Jennifer M., Dvorak, Christopher C., Haddad, Elie, Buckley, Rebecca H., Cowan, Morton J., and Heimall, Jennifer

Published
2024
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6. 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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7. Intestinal microbiome and metabolome signatures in patients with chronic granulomatous disease

Author

Chandrasekaran, Prabha, Han, Yu, Zerbe, Christa S., Heller, Theo, DeRavin, Suk See, Kreuzberg, Samantha A., Marciano, Beatriz E., Siu, Yik, Jones, Drew R., Abraham, Roshini S., Stephens, Michael C., Tsou, Amy M., Snapper, Scott, Conlan, Sean, Subramanian, Poorani, Quinones, Mariam, Grou, Caroline, Calderon, Virginie, Deming, Clayton, Leiding, Jennifer W., Arnold, Danielle E., Logan, Brent R., Griffith, Linda M., Petrovic, Aleksandra, Mousallem, Talal I., Kapoor, Neena, Heimall, Jennifer R., Barnum, Jessie L., Kapadia, Malika, Wright, Nicola, Rayes, Ahmad, Chandra, Sharat, Broglie, Larisa A., Chellapandian, Deepak, Deal, Christin L., Grunebaum, Eyal, Lim, Stephanie Si, Mallhi, Kanwaldeep, Marsh, Rebecca A., Murguia-Favela, Luis, Parikh, Suhag, Touzot, Fabien, 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., Kang, Elizabeth M., Malech, Harry L., Segre, Julia A., Bryant, Clare E., Holland, Steven M., and Falcone, Emilia Liana

Published
2023
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8. 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

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

Author

Thakar, Monica S, Logan, Brent R, Puck, Jennifer M, Dunn, Elizabeth A, Buckley, Rebecca H, Cowan, Morton J, O'Reilly, Richard J, Kapoor, Neena, Satter, Lisa Forbes, Pai, Sung-Yun, Heimall, Jennifer, Chandra, Sharat, Ebens, Christen L, Chellapandian, Deepak, Williams, Olatundun, Burroughs, Lauri M, Saldana, Blachy Davila, Rayes, Ahmad, Madden, Lisa M, Chandrakasan, Shanmuganathan, Bednarski, Jeffrey J, II, DeSantes, Kenneth B, Cuvelier, Geoffrey D E, Teira, Pierre, Gillio, Alfred P, Eissa, Hesham, Knutsen, Alan P, Goldman, Frederick D, Aquino, Victor M, Shereck, Evan B, Moore, Theodore B, Caywood, Emi H, Lugt, Mark T Vander, Rozmus, Jacob, Broglie, Larisa, Yu, Lolie C, Shah, Ami J, Andolina, Jeffrey R, Liu, Xuerong, Parrott, Roberta E, Dara, Jasmeen, Prockop, Susan, Martinez, Caridad A, Kapadia, Malika, Jyonouchi, Soma C, Sullivan, Kathleen E, Bleesing, Jack J, Chaudhury, Sonali, Petrovic, Aleksandra, Keller, Michael D, Quigg, Troy C, Parikh, Suhag, Shenoy, Shalini, Seroogy, Christine, Rubin, Tamar, Decaluwe, Hélène, Routes, John M, Torgerson, Troy R, Leiding, Jennifer W, Pulsipher, Michael A, Kohn, Donald B, Griffith, Linda M, Haddad, Elie, Dvorak, Christopher C, and Notarangelo, Luigi D

Published
2023
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10. 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

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

Author

Dvorak, Christopher C., Haddad, Elie, Heimall, Jennifer, Dunn, Elizabeth, Cowan, Morton J., Pai, Sung-Yun, Kapoor, Neena, Satter, Lisa Forbes, Buckley, Rebecca H., O’Reilly, Richard J., Chandra, Sharat, Bednarski, Jeffrey J., Williams, Olatundun, Rayes, Ahmad, Moore, Theodore B., Ebens, Christen L., Davila Saldana, Blachy J., Petrovic, Aleksandra, Chellapandian, Deepak, Cuvelier, Geoffrey D.E., Vander Lugt, Mark T., Caywood, Emi H., Chandrakasan, Shanmuganathan, Eissa, Hesham, Goldman, Frederick D., Shereck, Evan, Aquino, Victor M., Desantes, Kenneth B., Madden, Lisa M., Miller, Holly K., Yu, Lolie, Broglie, Larisa, Gillio, Alfred, Shah, Ami J., Knutsen, Alan P., Andolina, Jeffrey P., Joshi, Avni Y., Szabolcs, Paul, Kapadia, Malika, Martinez, Caridad A., Parrot, Roberta E., Sullivan, Kathleen E., Prockop, Susan E., Abraham, Roshini S., Thakar, Monica S., Leiding, Jennifer W., Kohn, Donald B., Pulsipher, Michael A., Griffith, Linda M., Notarangelo, Luigi D., and Puck, Jennifer M.

Published
2023
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12. The diagnosis of severe combined immunodeficiency (SCID): The Primary Immune Deficiency Treatment Consortium (PIDTC) 2022 Definitions

Author

Dvorak, Christopher C., Haddad, Elie, Heimall, Jennifer, Dunn, Elizabeth, Buckley, Rebecca H., Kohn, Donald B., Cowan, Morton J., Pai, Sung-Yun, Griffith, Linda M., Cuvelier, Geoffrey D.E., Eissa, Hesham, Shah, Ami J., O’Reilly, Richard J., Pulsipher, Michael A., Wright, Nicola A.M., Abraham, Roshini S., Satter, Lisa Forbes, Notarangelo, Luigi D., and Puck, Jennifer M.

Published
2023
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13. Aberrant T-cell exhaustion in severe combined immunodeficiency survivors with poor T-cell reconstitution after transplantation

Author

Labrosse, Roxane, Boufaied, Ines, Bourdin, Benoîte, Gona, Saideep, Randolph, Haley E., Logan, Brent R., Bourbonnais, Sara, Berthe, Chloé, Chan, Wendy, Buckley, Rebecca H., Parrott, Roberta E., Cuvelier, Geoffrey D.E., Kapoor, Neena, Chandra, Sharat, Dávila Saldaña, Blachy J., Eissa, Hesham, Goldman, Fred D., Heimall, Jennifer, O’Reilly, Richard, Chaudhury, Sonali, Kolb, Edward A., Shenoy, Shalini, Griffith, Linda M., Pulsipher, Michael, Kohn, Donald B., Notarangelo, Luigi D., Pai, Sung-Yun, Cowan, Morton J., Dvorak, Christopher C., Haddad, Élie, Puck, Jennifer M., Barreiro, Luis B., and Decaluwe, Hélène

Published
2023
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14. 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

15. The genetic landscape of severe combined immunodeficiency in the United States and Canada in the current era (2010-2018).

Author

Dvorak, Christopher C, Haddad, Elie, Buckley, Rebecca H, Cowan, Morton J, Logan, Brent, Griffith, Linda M, Kohn, Donald B, Pai, Sung-Yun, Notarangelo, Luigi, Shearer, William, Prockop, Susan, Kapoor, Neena, Heimall, Jennifer, Chaudhury, Sonali, Shyr, David, Chandra, Sharat, Cuvelier, Geoff, Moore, Theodore, Shenoy, Shalini, Goldman, Fred, Smith, Angela R, Sunkersett, Gauri, Vander Lugt, Mark, Caywood, Emi, Quigg, Troy, Torgerson, Troy, Chandrakasan, Shanmuganathan, Craddock, John, Dávila Saldaña, Blachy J, Gillio, Alfred, Shereck, Evan, Aquino, Victor, DeSantes, Kenneth, Knutsen, Alan, Thakar, Monica, Yu, Lolie, and Puck, Jennifer M

Subjects
Humans, Severe Combined Immunodeficiency, Child, Child, Preschool, Infant, Infant, Newborn, Canada, United States, Female, Male, Good Health and Well Being, Immunology, Allergy
Abstract

In a 250 patient cohort from the US and Canada in the current era (2010–2018), we show that over 90% of patients with severe combined immunodeficiency (SCID) can be genetically-characterized.

Published
2019

16. Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC

Author

Cuvelier, Geoffrey D.E., Logan, Brent R., Prockop, Susan E., Buckley, Rebecca H., Kuo, Caroline Y., Griffith, Linda M., Liu, Xuerong, Yip, Alison, Hershfield, Michael S., Ayoub, Paul G., Moore, Theodore B., Dorsey, Morna J., O'Reilly, Richard J., Kapoor, Neena, Pai, Sung-Yun, Kapadia, Malika, Ebens, Christen L., Forbes Satter, Lisa R., Burroughs, Lauri M., Petrovic, Aleksandra, Chellapandian, Deepak, Heimall, Jennifer, Shyr, David C., Rayes, Ahmad, Bednarski, Jeffrey J., Chandra, Sharat, Chandrakasan, Shanmuganathan, Gillio, Alfred P., Madden, Lisa, Quigg, Troy C., Caywood, Emi H., Dávila Saldaña, Blachy J., DeSantes, Kenneth, Eissa, Hesham, Goldman, Frederick D., Rozmus, Jacob, Shah, Ami J., Vander Lugt, Mark T., Thakar, Monica S., Parrott, Roberta E., Martinez, Caridad, Leiding, Jennifer W., Torgerson, Troy R., Pulsipher, Michael A., Notarangelo, Luigi D., Cowan, Morton J., Dvorak, Christopher C., Haddad, Elie, Puck, Jennifer M., and Kohn, Donald B.

Published
2022
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17. Toward a Better Understanding of the Atypical Features of Chronic Graft-Versus-Host Disease: A Report from the 2020 National Institutes of Health Consensus Project Task Force

Author

Cuvelier, Geoffrey D.E., Schoettler, Michelle, Buxbaum, Nataliya P., Pinal-Fernandez, Iago, Schmalzing, Marc, Distler, Jörg H.W., Penack, Olaf, Santomasso, Bianca D., Zeiser, Robert, Angstwurm, Klemens, MacDonald, Kelli P.A., Kimberly, W. Taylor, Taylor, Naomi, Bilic, Ervina, Banas, Bernhard, Buettner-Herold, Maike, Sinha, Namita, Greinix, Hildegard T., Pidala, Joseph, Schultz, Kirk R., Williams, Kirsten M., Inamoto, Yoshihiro, Cutler, Corey, Griffith, Linda M., Lee, Stephanie J., Sarantopoulos, Stefanie, Pavletic, Steven Z., and Wolff, Daniel

Published
2022
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18. Myeloablative Autologous Stem-Cell Transplantation for Severe Scleroderma

Author

Sullivan, Keith M, Goldmuntz, Ellen A, Keyes-Elstein, Lynette, McSweeney, Peter A, Pinckney, Ashley, Welch, Beverly, Mayes, Maureen D, Nash, Richard A, Crofford, Leslie J, Eggleston, Barry, Castina, Sharon, Griffith, Linda M, Goldstein, Julia S, Wallace, Dennis, Craciunescu, Oana, Khanna, Dinesh, Folz, Rodney J, Goldin, Jonathan, St Clair, E William, Seibold, James R, Phillips, Kristine, Mineishi, Shin, Simms, Robert W, Ballen, Karen, Wener, Mark H, Georges, George E, Heimfeld, Shelly, Hosing, Chitra, Forman, Stephen, Kafaja, Suzanne, Silver, Richard M, Griffing, Leroy, Storek, Jan, LeClercq, Sharon, Brasington, Richard, Csuka, Mary E, Bredeson, Christopher, Keever-Taylor, Carolyn, Domsic, Robyn T, Kahaleh, M Bashar, Medsger, Thomas, and Furst, Daniel E

Subjects
Autoimmune Disease, Transplantation, Stem Cell Research - Nonembryonic - Human, Scleroderma, Clinical Trials and Supportive Activities, Clinical Research, Stem Cell Research, Evaluation of treatments and therapeutic interventions, 6.1 Pharmaceuticals, Inflammatory and immune system, Good Health and Well Being, Adolescent, Adult, Aged, Cyclophosphamide, Disease-Free Survival, Female, Follow-Up Studies, Hematopoietic Stem Cell Transplantation, Humans, Immunosuppressive Agents, Infections, Intention to Treat Analysis, Kaplan-Meier Estimate, Male, Middle Aged, Scleroderma, Systemic, Transplantation Conditioning, Transplantation, Autologous, Young Adult, SCOT Study Investigators, Medical and Health Sciences, General & Internal Medicine
Abstract

BackgroundDespite current therapies, diffuse cutaneous systemic sclerosis (scleroderma) often has a devastating outcome. We compared myeloablative CD34+ selected autologous hematopoietic stem-cell transplantation with immunosuppression by means of 12 monthly infusions of cyclophosphamide in patients with scleroderma.MethodsWe randomly assigned adults (18 to 69 years of age) with severe scleroderma to undergo myeloablative autologous stem-cell transplantation (36 participants) or to receive cyclophosphamide (39 participants). The primary end point was a global rank composite score comparing participants with each other on the basis of a hierarchy of disease features assessed at 54 months: death, event-free survival (survival without respiratory, renal, or cardiac failure), forced vital capacity, the score on the Disability Index of the Health Assessment Questionnaire, and the modified Rodnan skin score.ResultsIn the intention-to-treat population, global rank composite scores at 54 months showed the superiority of transplantation (67% of 1404 pairwise comparisons favored transplantation and 33% favored cyclophosphamide, P=0.01). In the per-protocol population (participants who received a transplant or completed ≥9 doses of cyclophosphamide), the rate of event-free survival at 54 months was 79% in the transplantation group and 50% in the cyclophosphamide group (P=0.02). At 72 months, Kaplan-Meier estimates of event-free survival (74% vs. 47%) and overall survival (86% vs. 51%) also favored transplantation (P=0.03 and 0.02, respectively). A total of 9% of the participants in the transplantation group had initiated disease-modifying antirheumatic drugs (DMARDs) by 54 months, as compared with 44% of those in the cyclophosphamide group (P=0.001). Treatment-related mortality in the transplantation group was 3% at 54 months and 6% at 72 months, as compared with 0% in the cyclophosphamide group.ConclusionsMyeloablative autologous hematopoietic stem-cell transplantation achieved long-term benefits in patients with scleroderma, including improved event-free and overall survival, at a cost of increased expected toxicity. Rates of treatment-related death and post-transplantation use of DMARDs were lower than those in previous reports of nonmyeloablative transplantation. (Funded by the National Institute of Allergy and Infectious Diseases and the National Institutes of Health; ClinicalTrials.gov number, NCT00114530 .).

Published
2018

19. National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease: IV. The 2020 Highly morbid forms report

Author

Wolff, Daniel, Radojcic, Vedran, Lafyatis, Robert, Cinar, Resat, Rosenstein, Rachel K., Cowen, Edward W., Cheng, Guang-Shing, Sheshadri, Ajay, Bergeron, Anne, Williams, Kirsten M., Todd, Jamie L., Teshima, Takanori, Cuvelier, Geoffrey D.E., Holler, Ernst, McCurdy, Shannon R., Jenq, Robert R., Hanash, Alan M., Jacobsohn, David, Santomasso, Bianca D., Jain, Sandeep, Ogawa, Yoko, Steven, Philipp, Luo, Zhonghui Katie, Dietrich-Ntoukas, Tina, Saban, Daniel, Bilic, Ervina, Penack, Olaf, Griffith, Linda M., Cowden, Meredith, Martin, Paul J., Greinix, Hildegard T., Sarantopoulos, Stefanie, Socie, Gerard, Blazar, Bruce R., Pidala, Joseph, Kitko, Carrie L., Couriel, Daniel R., Cutler, Corey, Schultz, Kirk R., Pavletic, Steven Z., Lee, Stephanie J., and Paczesny, Sophie

Published
2021
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21. Recommendations for Screening and Management of Late Effects in Patients with Severe Combined Immunodeficiency after Allogenic Hematopoietic Cell Transplantation: A Consensus Statement from the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects after Pediatric HCT.

Author

Heimall, Jennifer, Buckley, Rebecca H, Puck, Jennifer, Fleisher, Thomas A, Gennery, Andrew R, Haddad, Elie, Neven, Benedicte, Slatter, Mary, Roderick, Skinner, Baker, K Scott, Dietz, Andrew C, Duncan, Christine, Griffith, Linda M, Notarangelo, Luigi, Pulsipher, Michael A, and Cowan, Morton J

Subjects
Humans, Severe Combined Immunodeficiency, Hematopoietic Stem Cell Transplantation, Consensus, Adolescent, Adult, Child, Child, Preschool, Infant, Infant, Newborn, Female, Male, Practice Guidelines as Topic, Allografts, Bone marrow transplantation, Long-term follow-up guidelines, Severe combined immune deficiency, Pediatric, Cancer, Rare Diseases, Transplantation, Pediatric Research Initiative, Regenerative Medicine, Hematology, Clinical Sciences, Immunology
Abstract

Severe combined immunodeficiency (SCID) is effectively treated with hematopoietic cell transplantation (HCT), with overall survival approaching 90% in contemporary reports. However, survivors are at risk for developing late complications because of the variable durability of high-quality immune function, underlying genotype of SCID, comorbidities due to infections in the pretransplantation and post-transplantation periods, and use of conditioning before transplantation. An international group of transplantation experts was convened in 2016 to review the current knowledge of late effects seen in SCID patients after HCT and to develop recommendations for screening and monitoring for late effects. This report provides recommendations for screening and management of pediatric and adult SCID patients treated with HCT.

Published
2017

22. National Institutes of Health Hematopoietic Cell Transplantation Late Effects Initiative: The Immune Dysregulation and Pathobiology Working Group Report

Author

Gea-Banacloche, Juan, Komanduri, Krishna V, Carpenter, Paul, Paczesny, Sophie, Sarantopoulos, Stefanie, Young, Jo-Anne, Kassar, Nahed El, Le, Robert Q, Schultz, Kirk R, Griffith, Linda M, Savani, Bipin N, and Wingard, John R

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Immunology, Transplantation, Clinical Research, Vaccine Related, Rare Diseases, Prevention, Emerging Infectious Diseases, Infectious Diseases, Immunization, Infection, Inflammatory and immune system, Good Health and Well Being, Hematopoietic Stem Cell Transplantation, Humans, Immune Reconstitution, Infections, Long Term Adverse Effects, National Institutes of Health (U.S.), United States, Immune reconstitution, Intravenous immunoglobulin, Late infections, Clinical Sciences, Cardiovascular medicine and haematology
Abstract

Immune reconstitution after hematopoietic stem cell transplantation (HCT) beyond 1 year is not completely understood. Many transplant recipients who are free of graft-versus-host disease (GVHD) and not receiving any immunosuppression more than 1 year after transplantation seem to be able to mount appropriate immune responses to common pathogens and respond adequately to immunizations. However, 2 large registry studies over the last 2 decades seem to indicate that infection is a significant cause of late mortality in some patients, even in the absence of concomitant GVHD. Research on this topic is particularly challenging for several reasons. First, there are not enough long-term follow-up clinics able to measure even basic immune parameters late after HCT. Second, the correlation between laboratory measurements of immune function and infections is not well known. Third, accurate documentation of infectious episodes is notoriously difficult. Finally, it is unclear what measures can be implemented to improve the immune response in a clinically relevant way. A combination of long-term multicenter prospective studies that collect detailed infectious data and store samples as well as a national or multinational registry of clinically significant infections (eg, vaccine-preventable severe infections, opportunistic infections) could begin to address our knowledge gaps. Obtaining samples for laboratory evaluation of the immune system should be both calendar and eventdriven. Attention to detail and standardization of practices regarding prophylaxis, diagnosis, and definitions of infections would be of paramount importance to obtain clean reliable data. Laboratory studies should specifically address the neogenesis, maturation, and exhaustion of the adaptive immune system and, in particular, how these are influenced by persistent alloreactivity, inflammation, and viral infection. Ideally, some of these long-term prospective studies would collect information on long-term changes in the gut microbiome and their influence on immunity. Regarding enhancement of immune function, prospective measurement of the response to vaccines late after HCT in a variety of clinical settings should be undertaken to better understand the benefits as well as the limitations of immunizations. The role of intravenous immunoglobulin is still not well defined, and studies to address it should be encouraged.

Published
2017

23. Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report

Author

Burroughs, Lauri M., Petrovic, Aleksandra, Brazauskas, Ruta, Liu, Xuerong, Griffith, Linda M., Ochs, Hans D., Bleesing, Jack J., Edwards, Stephanie, Dvorak, Christopher C., Chaudhury, Sonali, Prockop, Susan E., Quinones, Ralph, Goldman, Frederick D., Quigg, Troy C., Chandrakasan, Shanmuganathan, Smith, Angela R., Parikh, Suhag, Dávila Saldaña, Blachy J., Thakar, Monica S., Phelan, Rachel, Shenoy, Shalini, Forbes, Lisa R., Martinez, Caridad, Chellapandian, Deepak, Shereck, Evan, Miller, Holly K., Kapoor, Neena, Barnum, Jessie L., Chong, Hey, Shyr, David C., Chen, Karin, Abu-Arja, Rolla, Shah, Ami J., Weinacht, Katja G., Moore, Theodore B., Joshi, Avni, DeSantes, Kenneth B., Gillio, Alfred P., Cuvelier, Geoffrey D.E., Keller, Michael D., Rozmus, Jacob, Torgerson, Troy, Pulsipher, Michael A., Haddad, Elie, Sullivan, Kathleen E., Logan, Brent R., Kohn, Donald B., Puck, Jennifer M., Notarangelo, Luigi D., Pai, Sung-Yun, Rawlings, David J., and Cowan, Morton J.

Published
2020
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24. 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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25. Primary Immune Deficiency Treatment Consortium (PIDTC) update.

Author

Griffith, Linda M, Cowan, Morton J, Notarangelo, Luigi D, Kohn, Donald B, Puck, Jennifer M, Shearer, William T, Burroughs, Lauri M, Torgerson, Troy R, Decaluwe, Hélène, Haddad, Elie, and workshop participants

Subjects
workshop participants, Animals, Humans, Immunologic Deficiency Syndromes, Voluntary Health Agencies, Clinical Studies as Topic, Intersectoral Collaboration, Allogeneic hematopoietic cell transplantation, clinical trial, gene therapy, primary immunodeficiency, Prevention, Rare Diseases, Clinical Research, Pediatric, Inflammatory and immune system, Good Health and Well Being, Immunology, Allergy
Abstract

The Primary Immune Deficiency Treatment Consortium (PIDTC) is a collaboration of 41 North American centers studying therapy for rare primary immune deficiency diseases (PIDs), including severe combined immune deficiency (SCID), Wiskott-Aldrich syndrome (WAS), and chronic granulomatous disease (CGD). An additional 3 European centers have partnered with the PIDTC to study CGD. Natural history protocols of the PIDTC analyze outcomes of treatment for rare PIDs in multicenter longitudinal retrospective, prospective, and cross-sectional studies. Since 2009, participating centers have enrolled more than 800 subjects on PIDTC protocols for SCID, and enrollment in the studies on WAS and CGD is underway. Four pilot projects have been funded, and 12 junior investigators have received fellowship awards. Important publications of the consortium describe the outcomes of hematopoietic cell transplantation for SCID during 2000-2009, diagnostic criteria for SCID, and the pilot project of newborn screening for SCID in the Navajo Nation. The PIDTC Annual Scientific Workshops provide an opportunity to strengthen collaborations with junior investigators, patient advocacy groups, and international colleagues. Funded by the National Institute of Allergy and Infectious Diseases and the Office of Rare Diseases Research, National Center for Advancing Translational Sciences, the PIDTC has recently received renewal for another 5 years. Here we review accomplishments of the group, projects underway, highlights of recent workshops, and challenges for the future.

Published
2016

26. High-Dose Immunosuppressive Therapy and Autologous Hematopoietic Cell Transplantation for Relapsing-Remitting Multiple Sclerosis (HALT-MS): A 3-Year Interim Report

Author

Nash, Richard A, Hutton, George J, Racke, Michael K, Popat, Uday, Devine, Steven M, Griffith, Linda M, Muraro, Paolo A, Openshaw, Harry, Sayre, Peter H, Stüve, Olaf, Arnold, Douglas L, Spychala, Meagan E, McConville, Kaitlyn C, Harris, Kristina M, Phippard, Deborah, Georges, George E, Wundes, Annette, Kraft, George H, and Bowen, James D

Subjects
Biomedical and Clinical Sciences, Immunology, Multiple Sclerosis, Transplantation, Clinical Research, Regenerative Medicine, Clinical Trials and Supportive Activities, Neurodegenerative, Brain Disorders, Autoimmune Disease, Neurosciences, Hematology, Stem Cell Research, Evaluation of treatments and therapeutic interventions, 6.1 Pharmaceuticals, Adult, Combined Modality Therapy, Disease Progression, Disease-Free Survival, Female, Follow-Up Studies, Hematopoietic Stem Cell Transplantation, Humans, Immunosuppressive Agents, Magnetic Resonance Imaging, Male, Middle Aged, Multiple Sclerosis, Relapsing-Remitting, Transplantation, Autologous, Treatment Outcome
Abstract

ImportanceMost patients with relapsing-remitting (RR) multiple sclerosis (MS) who receive approved disease-modifying therapies experience breakthrough disease and accumulate neurologic disability. High-dose immunosuppressive therapy (HDIT) with autologous hematopoietic cell transplant (HCT) may, in contrast, induce sustained remissions in early MS.ObjectiveTo evaluate the safety, efficacy, and durability of MS disease stabilization through 3 years after HDIT/HCT.Design, setting, and participantsHematopoietic Cell Transplantation for Relapsing-Remitting Multiple Sclerosis (HALT-MS) is an ongoing, multicenter, single-arm, phase 2 clinical trial of HDIT/HCT for patients with RRMS who experienced relapses with loss of neurologic function while receiving disease-modifying therapies during the 18 months before enrolling. Participants are evaluated through 5 years after HCT. This report is a prespecified, 3-year interim analysis of the trial. Thirty-six patients with RRMS from referral centers were screened; 25 were enrolled.InterventionsAutologous peripheral blood stem cell grafts were CD34+ selected; the participants then received high-dose treatment with carmustine, etoposide, cytarabine, and melphalan as well as rabbit antithymocyte globulin before autologous HCT.Main outcomes and measuresThe primary end point of HALT-MS is event-free survival defined as survival without death or disease activity from any one of the following outcomes: (1) confirmed loss of neurologic function, (2) clinical relapse, or (3) new lesions observed on magnetic resonance imaging. Toxic effects are reported using National Cancer Institute Common Terminology Criteria for Adverse Events.ResultsGrafts were collected from 25 patients, and 24 of these individuals received HDIT/HCT. The median follow-up period was 186 weeks (interquartile range, 176-250) weeks). Overall event-free survival was 78.4% (90% CI, 60.1%-89.0%) at 3 years. Progression-free survival and clinical relapse-free survival were 90.9% (90% CI, 73.7%-97.1%) and 86.3% (90% CI, 68.1%-94.5%), respectively, at 3 years. Adverse events were consistent with expected toxic effects associated with HDIT/HCT, and no acute treatment-related neurologic adverse events were observed. Improvements were noted in neurologic disability, quality-of-life, and functional scores.Conclusions and relevanceAt 3 years, HDIT/HCT without maintenance therapy was effective for inducing sustained remission of active RRMS and was associated with improvements in neurologic function. Treatment was associated with few serious early complications or unexpected adverse events.

Published
2015

27. High symptom burden in female X-linked chronic granulomatous disease carriers

Author

Miranda, Mary Ann, Tsalatsanis, Athanasios, Trotter, Jessica R., Arnold, Danielle E., Squire, Jacqueline D., Kidd, Sharon, Parikh, Suhag, Marsh, Rebecca A., Griffith, Linda M., Mallhi, Kanwaldeep, Chellapandian, Deepak, Lim, Stephanie Si, Grunebaum, Eyal, Sullivan, Kathleen E., Newburger, Peter E., Dinauer, Mary C., 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., Kang, Elizabeth M., Morton, Felicia B., and Leiding, Jennifer W.

Published
2024
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28. Autologous Hematopoietic Cell Transplantation for Treatment-Refractory Relapsing Multiple Sclerosis: Position Statement from the American Society for Blood and Marrow Transplantation

Author

Cohen, Jeffrey A., Baldassari, Laura E., Atkins, Harold L., Bowen, James D., Bredeson, Christopher, Carpenter, Paul A., Corboy, John R., Freedman, Mark S., Griffith, Linda M., Lowsky, Robert, Majhail, Navneet S., Muraro, Paolo A., Nash, Richard A., Pasquini, Marcelo C., Sarantopoulos, Stefanie, Savani, Bipin N., Storek, Jan, Sullivan, Keith M., and Georges, George E.

Published
2019
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29. 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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30. Transplantation outcomes for severe combined immunodeficiency, 2000-2009.

Author

Pai, Sung-Yun, Logan, Brent R, Griffith, Linda M, Buckley, Rebecca H, Parrott, Roberta E, Dvorak, Christopher C, Kapoor, Neena, Hanson, Imelda C, Filipovich, Alexandra H, Jyonouchi, Soma, Sullivan, Kathleen E, Small, Trudy N, Burroughs, Lauri, Skoda-Smith, Suzanne, Haight, Ann E, Grizzle, Audrey, Pulsipher, Michael A, Chan, Ka Wah, Fuleihan, Ramsay L, Haddad, Elie, Loechelt, Brett, Aquino, Victor M, Gillio, Alfred, Davis, Jeffrey, Knutsen, Alan, Smith, Angela R, Moore, Theodore B, Schroeder, Marlis L, Goldman, Frederick D, Connelly, James A, Porteus, Matthew H, Xiang, Qun, Shearer, William T, Fleisher, Thomas A, Kohn, Donald B, Puck, Jennifer M, Notarangelo, Luigi D, Cowan, Morton J, and O'Reilly, Richard J

Subjects
T-Lymphocytes, Humans, Severe Combined Immunodeficiency, Graft vs Host Disease, Immunoglobulin A, Lymphocyte Count, Treatment Outcome, Transplantation Conditioning, Retreatment, Hematopoietic Stem Cell Transplantation, Incidence, Survival Rate, Retrospective Studies, Siblings, Infant, Female, Male, CD3 Complex, Clinical Research, Organ Transplantation, Regenerative Medicine, Transplantation, Rare Diseases, Pediatric, Orphan Drug, Good Health and Well Being, Medical and Health Sciences, General & Internal Medicine
Abstract

BackgroundThe Primary Immune Deficiency Treatment Consortium was formed to analyze the results of hematopoietic-cell transplantation in children with severe combined immunodeficiency (SCID) and other primary immunodeficiencies. Factors associated with a good transplantation outcome need to be identified in order to design safer and more effective curative therapy, particularly for children with SCID diagnosed at birth.MethodsWe collected data retrospectively from 240 infants with SCID who had received transplants at 25 centers during a 10-year period (2000 through 2009).ResultsSurvival at 5 years, freedom from immunoglobulin substitution, and CD3+ T-cell and IgA recovery were more likely among recipients of grafts from matched sibling donors than among recipients of grafts from alternative donors. However, the survival rate was high regardless of donor type among infants who received transplants at 3.5 months of age or younger (94%) and among older infants without prior infection (90%) or with infection that had resolved (82%). Among actively infected infants without a matched sibling donor, survival was best among recipients of haploidentical T-cell-depleted transplants in the absence of any pretransplantation conditioning. Among survivors, reduced-intensity or myeloablative pretransplantation conditioning was associated with an increased likelihood of a CD3+ T-cell count of more than 1000 per cubic millimeter, freedom from immunoglobulin substitution, and IgA recovery but did not significantly affect CD4+ T-cell recovery or recovery of phytohemagglutinin-induced T-cell proliferation. The genetic subtype of SCID affected the quality of CD3+ T-cell recovery but not survival.ConclusionsTransplants from donors other than matched siblings were associated with excellent survival among infants with SCID identified before the onset of infection. All available graft sources are expected to lead to excellent survival among asymptomatic infants. (Funded by the National Institute of Allergy and Infectious Diseases and others.).

Published
2014

31. Establishing diagnostic criteria for severe combined immunodeficiency disease (SCID), leaky SCID, and Omenn syndrome: The Primary Immune Deficiency Treatment Consortium experience

Author

Shearer, William T, Dunn, Elizabeth, Notarangelo, Luigi D, Dvorak, Christopher C, Puck, Jennifer M, Logan, Brent R, Griffith, Linda M, Kohn, Donald B, O'Reilly, Richard J, Fleisher, Thomas A, Pai, Sung-Yun, Martinez, Caridad A, Buckley, Rebecca H, and Cowan, Morton J

Subjects
Medical Biotechnology, Biomedical and Clinical Sciences, Genetics, Rare Diseases, Transplantation, Pediatric, Clinical Research, Good Health and Well Being, Humans, Immunologic Deficiency Syndromes, North America, Practice Guidelines as Topic, Retrospective Studies, Severe Combined Immunodeficiency, Allogeneic hematopoietic cell transplantation, gene therapy, primary immunodeficiency, clinical trial, Immunology, Allergy
Abstract

BackgroundThe approach to the diagnosis of severe combined immunodeficiency disease (SCID) and related disorders varies among institutions and countries.ObjectivesThe Primary Immune Deficiency Treatment Consortium attempted to develop a uniform set of criteria for diagnosing SCID and related disorders and has evaluated the results as part of a retrospective study of SCID in North America.MethodsClinical records from 2000 through 2009 at 27 centers in North America were collected on 332 children treated with hematopoietic stem cell transplantation (HCT), enzyme replacement therapy, or gene therapy for SCID and related disorders. Eligibility for inclusion in the study and classification into disease groups were established by using set criteria and applied by an expert review group.ResultsTwo hundred eighty-five (86%) of the patients were determined to be eligible, and 47 (14%) were not eligible. Of the 285 eligible patients, 84% were classified as having typical SCID; 13% were classified as having leaky SCID, Omenn syndrome, or reticular dysgenesis; and 3% had a history of enzyme replacement or gene therapy. Detection of a genotype predicting an SCID phenotype was accepted for eligibility. Reasons for noneligibility were failure to demonstrate either impaired lymphocyte proliferation or maternal T-cell engraftment. Overall (n = 332) rates of testing were as follows: proliferation to PHA, 77%; maternal engraftment, 35%; and genotype, 79% (mutation identified in 62%).ConclusionLack of complete laboratory evaluation of patients before HCT presents a significant barrier to definitive diagnosis of SCID and related disorders and prevented inclusion of subjects in our observational HCT study. This lesson is critical for patient care, as well as the design of future prospective treatment studies for such children because a well-defined and consistent study population is important for precision in outcomes analysis.

Published
2014

33. Primary Immune Deficiency Treatment Consortium (PIDTC) report.

Author

Griffith, Linda M, Cowan, Morton J, Notarangelo, Luigi D, Kohn, Donald B, Puck, Jennifer M, Pai, Sung-Yun, Ballard, Barbara, Bauer, Sarah C, Bleesing, Jack JH, Boyle, Marcia, Brower, Amy, Buckley, Rebecca H, van der Burg, Mirjam, Burroughs, Lauri M, Candotti, Fabio, Cant, Andrew J, Chatila, Talal, Cunningham-Rundles, Charlotte, Dinauer, Mary C, Dvorak, Christopher C, Filipovich, Alexandra H, Fleisher, Thomas A, Bobby Gaspar, Hubert, Gungor, Tayfun, Haddad, Elie, Hovermale, Emily, Huang, Faith, Hurley, Alan, Hurley, Mary, Iyengar, Sumathi, Kang, Elizabeth M, Logan, Brent R, Long-Boyle, Janel R, Malech, Harry L, McGhee, Sean A, Modell, Fred, Modell, Vicki, Ochs, Hans D, O'Reilly, Richard J, Parkman, Robertson, Rawlings, David J, Routes, John M, Shearer, William T, Small, Trudy N, Smith, Heather, Sullivan, Kathleen E, Szabolcs, Paul, Thrasher, Adrian, Torgerson, Troy R, Veys, Paul, Weinberg, Kenneth, Zuniga-Pflucker, Juan Carlos, and workshop participants

Subjects
workshop participants, Humans, Immunologic Deficiency Syndromes, Neonatal Screening, Hematopoietic Stem Cell Transplantation, Pilot Projects, Infant, Newborn, Societies, Scientific, ADA, Adenosine deaminase, Allogeneic hematopoietic cell transplantation, CGD, CIBMTR, Center for International Blood and Marrow Transplant Research, Chronic granulomatous disease, EBMT, ESID, European Group for Blood and Marrow Transplantation, European Society for Immunodeficiencies, GT, GVHD, Gene therapy, Graft-versus-host disease, HCT, Hematopoietic cell transplantation, IEWP, Inborn Errors Working Party, MAC, MUD, Matched unrelated donor, Myeloablative conditioning, NBS, NIAID, NIH, NK, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Natural killer, Newborn screening for SCID, PID, PIDTC, Primary Immune Deficiency Treatment Consortium, Primary immunodeficiency, RIC, Reduced-intensity conditioning, SCETIDE, SCID, Severe combined immunodeficiency, Stem Cell Transplantation for Immunodeficiencies in Europe, USIDNET, United States Immunodeficiency Network, WAS, Wiskott-Aldrich syndrome, clinical trial, gene therapy, primary immunodeficiency, Rare Diseases, Pediatric, Clinical Research, Inflammatory and immune system, Immunology, Allergy
Abstract

The Primary Immune Deficiency Treatment Consortium (PIDTC) is a network of 33 centers in North America that study the treatment of rare and severe primary immunodeficiency diseases. Current protocols address the natural history of patients treated for severe combined immunodeficiency (SCID), Wiskott-Aldrich syndrome, and chronic granulomatous disease through retrospective, prospective, and cross-sectional studies. The PIDTC additionally seeks to encourage training of junior investigators, establish partnerships with European and other International colleagues, work with patient advocacy groups to promote community awareness, and conduct pilot demonstration projects. Future goals include the conduct of prospective treatment studies to determine optimal therapies for primary immunodeficiency diseases. To date, the PIDTC has funded 2 pilot projects: newborn screening for SCID in Navajo Native Americans and B-cell reconstitution in patients with SCID after hematopoietic stem cell transplantation. Ten junior investigators have received grant awards. The PIDTC Annual Scientific Workshop has brought together consortium members, outside speakers, patient advocacy groups, and young investigators and trainees to report progress of the protocols and discuss common interests and goals, including new scientific developments and future directions of clinical research. Here we report the progress of the PIDTC to date, highlights of the first 2 PIDTC workshops, and consideration of future consortium objectives.

Published
2014

34. Posttransplantation late complications increase over time for patients with SCID: A Primary Immune Deficiency Treatment Consortium (PIDTC) landmark study

Author

Eissa, Hesham, primary, Thakar, Monica S., additional, Shah, Ami J., additional, Logan, Brent R., additional, Griffith, Linda M., additional, Dong, Huaying, additional, Parrott, Roberta E., additional, O’Reilly, Richard J., additional, Dara, Jasmeen, additional, Kapoor, Neena, additional, Forbes Satter, Lisa, additional, Chandra, Sharat, additional, Kapadia, Malika, additional, Chandrakasan, Shanmuganathan, additional, Knutsen, Alan, additional, Jyonouchi, Soma C., additional, Molinari, Lyndsay, additional, Rayes, Ahmad, additional, Ebens, Christen L., additional, Teira, Pierre, additional, Dávila Saldaña, Blachy J., additional, Burroughs, Lauri M., additional, Chaudhury, Sonali, additional, Chellapandian, Deepak, additional, Gillio, Alfred P., additional, Goldman, Fredrick, additional, Malech, Harry L., additional, DeSantes, Kenneth, additional, Cuvelier, Geoff D.E., additional, Rozmus, Jacob, additional, Quinones, Ralph, additional, Yu, Lolie C., additional, Broglie, Larisa, additional, Aquino, Victor, additional, Shereck, Evan, additional, Moore, Theodore B., additional, Vander Lugt, Mark T., additional, Mousallem, Talal I., additional, Oved, Joeseph H., additional, Dorsey, Morna, additional, Abdel-Azim, Hisham, additional, Martinez, Caridad, additional, Bleesing, Jacob H., additional, Prockop, Susan, additional, Kohn, Donald B., additional, Bednarski, Jeffrey J., additional, Leiding, Jennifer, additional, Marsh, Rebecca A., additional, Torgerson, Troy, additional, Notarangelo, Luigi D., additional, Pai, Sung-Yun, additional, Pulsipher, Michael A., additional, Puck, Jennifer M., additional, Dvorak, Christopher C., additional, Haddad, Elie, additional, Buckley, Rebecca H., additional, Cowan, Morton J., additional, and Heimall, Jennifer, additional

Published
2023
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35. SCID genotype and 6-month posttransplant CD4 count predict survival and immune recovery

Author

Haddad, Elie, Logan, Brent R., Griffith, Linda M., Buckley, Rebecca H., Parrott, Roberta E., Prockop, Susan E., Small, Trudy N., Chaisson, Jessica, Dvorak, Christopher C., Murnane, Megan, Kapoor, Neena, Abdel-Azim, Hisham, Hanson, Imelda C., Martinez, Caridad, Bleesing, Jack J.H., Chandra, Sharat, Smith, Angela R., Cavanaugh, Matthew E., Jyonouchi, Soma, Sullivan, Kathleen E., Burroughs, Lauri, Skoda-Smith, Suzanne, Haight, Ann E., Tumlin, Audrey G., Quigg, Troy C., Taylor, Candace, Dávila Saldaña, Blachy J., Keller, Michael D., Seroogy, Christine M., Desantes, Kenneth B., Petrovic, Aleksandra, Leiding, Jennifer W., Shyr, David C., Decaluwe, Hélène, Teira, Pierre, Gillio, Alfred P., Knutsen, Alan P., Moore, Theodore B., Kletzel, Morris, Craddock, John A., Aquino, Victor, Davis, Jeffrey H., Yu, Lolie C., Cuvelier, Geoffrey D.E., Bednarski, Jeffrey J., Goldman, Frederick D., Kang, Elizabeth M., Shereck, Evan, Porteus, Matthew H., Connelly, James A., Fleisher, Thomas A., Malech, Harry L., Shearer, William T., Szabolcs, Paul, Thakar, Monica S., Vander Lugt, Mark T., Heimall, Jennifer, Yin, Ziyan, Pulsipher, Michael A., Pai, Sung-Yun, Kohn, Donald B., Puck, Jennifer M., Cowan, Morton J., O'Reilly, Richard J., and Notarangelo, Luigi D.

Published
2018
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36. B-cell differentiation and IL-21 response in IL2RG/JAK3 SCID patients after hematopoietic stem cell transplantation

Author

Miggelbrink, Alexandra M., Logan, Brent R., Buckley, Rebecca H., Parrott, Roberta E., Dvorak, Christopher C., Kapoor, Neena, Abdel-Azim, Hisham, Prockop, Susan E., Shyr, David, Decaluwe, Hélène, Hanson, Imelda C., Gillio, Alfred, Dávila Saldaña, Blachy J., Eibel, Hermann, Hopkins, Gregory, Walter, Jolan E., Whangbo, Jennifer S., Kohn, Donald B., Puck, Jennifer M., Cowan, Morton J., Griffith, Linda M., Haddad, Elie, O'Reilly, Richard J., Notarangelo, Luigi D., and Pai, Sung-Yun

Published
2018
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37. The natural history of children with severe combined immunodeficiency: baseline features of the first fifty patients of the primary immune deficiency treatment consortium prospective study 6901.

Author

Dvorak, Christopher C, Cowan, Morton J, Logan, Brent R, Notarangelo, Luigi D, Griffith, Linda M, Puck, Jennifer M, Kohn, Donald B, Shearer, William T, O'Reilly, Richard J, Fleisher, Thomas A, Pai, Sung-Yun, Hanson, I Celine, Pulsipher, Michael A, Fuleihan, Ramsay, Filipovich, Alexandra, Goldman, Frederick, Kapoor, Neena, Small, Trudy, Smith, Angela, Chan, Ka-Wah, Cuvelier, Geoff, Heimall, Jennifer, Knutsen, Alan, Loechelt, Brett, Moore, Theodore, and Buckley, Rebecca H

Subjects
Humans, Severe Combined Immunodeficiency, Treatment Outcome, Hematopoietic Stem Cell Transplantation, Survival Analysis, Follow-Up Studies, Prospective Studies, Immunophenotyping, Genotype, Child, Child, Preschool, Infant, Infant, Newborn, Female, Male, Genetic Testing, Biomarkers, Genetics, Clinical Research, Transplantation, Prevention, Pediatric, Rare Diseases, Severe combined immunodeficiency, hematopoietic cell transplantation, newborn screening, Immunology
Abstract

The Primary Immune Deficiency Treatment Consortium (PIDTC) consists of 33 centers in North America. We hypothesized that the analysis of uniform data on patients with severe combined immunodeficiency (SCID) enrolled in a prospective protocol will identify variables that contribute to optimal outcomes following treatment. We report baseline clinical, immunologic, and genetic features of the first 50 patients enrolled, and the initial therapies administered, reflecting current practice in the diagnosis and treatment of both typical (n = 37) and atypical forms (n = 13) of SCID. From August 2010 to May 2012, patients with suspected SCID underwent evaluation and therapy per local center practices. Diagnostic information was reviewed by the PIDTC eligibility review panel, and hematopoietic cell transplantation (HCT) details were obtained from the Center for International Blood and Marrow Transplant Research. Most patients (92 %) had mutations in a known SCID gene. Half of the patients were diagnosed by newborn screening or family history, were younger than those diagnosed by clinical signs (median 15 vs. 181 days; P =

Published
2013

38. Immune reconstitution and survival of 100 SCID patients post–hematopoietic cell transplant: a PIDTC natural history study

Author

Heimall, Jennifer, Logan, Brent R., Cowan, Morton J., Notarangelo, Luigi D., Griffith, Linda M., Puck, Jennifer M., Kohn, Donald B., Pulsipher, Michael A., Parikh, Suhag, Martinez, Caridad, Kapoor, Neena, O'Reilly, Richard, Boyer, Michael, Pai, Sung-Yun, Goldman, Frederick, Burroughs, Lauri, Chandra, Sharat, Kletzel, Morris, Thakar, Monica, Connelly, James, Cuvelier, Geoff, Davila Saldana, Blachy J., Shereck, Evan, Knutsen, Alan, Sullivan, Kathleen E., DeSantes, Kenneth, Gillio, Alfred, Haddad, Elie, Petrovic, Aleksandra, Quigg, Troy, Smith, Angela R., Stenger, Elizabeth, Yin, Ziyan, Shearer, William T., Fleisher, Thomas, Buckley, Rebecca H., and Dvorak, Christopher C.

Published
2017
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40. Racial and Ethnic Disparities in Outcomes following Hematopoietic Stem Cell Transplant (HCT) in Patients with Severe Combined Immunodeficiency (SCID)

Author

Winestone, Lena, primary, Logan, Brent R., additional, Liu, Xuerong, additional, Buckley, Rebecca H., additional, O’Reilly, Richard J, additional, Griffith, Linda M., additional, Puck, Jennifer, additional, Dvorak, Christopher C., additional, Cowan, Morton J, additional, and Haddad, Elie, additional

Published
2023
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41. Event Free Survival in Severe Combined Immune Deficiency (SCID) Infants after Conditioned Umbilical Cord Blood Transplantation (UCBT) Benefits from Omitting Serotherapy

Author

Martinez, Caridad, primary, Logan, Brent, additional, Liu, Xuerong, additional, Dvorak, Christopher C., additional, Madden, Lisa, additional, Molinari, Lyndsay, additional, Cowan, Morton J., additional, Pai, Sung-Yun, additional, Haddad, Elie, additional, Puck, Jennifer, additional, Kohn, Donald B., additional, Griffith, Linda M., additional, Pulsipher, Michael, additional, Leiding, Jennifer W., additional, Notarangelo, Luigi D., additional, Torgerson, Troy, additional, Marsh, Rebecca A., additional, Cuvelier, Geoff D.E., additional, Prockop, Susan, additional, Buckley, Rebecca H., additional, Kuo, Caroline Y., additional, Yip, Alison, additional, Hershfield, Michael S., additional, Parrott, Roberta E, additional, Ebens, Christen L., additional, Moore, Theodore B., additional, O’Reilly, Richard J., additional, Kapadia, Malika, additional, Kapoor, Neena, additional, Satter, Lisa Forbes, additional, Burroughs, Lauri M., additional, Petrovic, Aleksandra, additional, Thakar, Monica S., additional, Chellapandian, Deepak, additional, Heimall, Jennifer R., additional, Shyr, David C., additional, Bednarski, Jeffrey J, additional, Rayes, Ahmad, additional, Chandrakasan, Shanmuganathan, additional, Quigg, Troy C., additional, Davila, Blachy J, additional, DeSantes, Kenneth, additional, Eissa, Hesham, additional, Goldman, Frederick, additional, Rozmus, Jacob, additional, Shah, Ami J, additional, Lugt, Mark Vander, additional, Keller, Michael D., additional, Sullivan, Kathleen E., additional, Jyonouchi, Soma, additional, Seroogy, Christine, additional, Decaluwe, Helene, additional, Teira, Pierre, additional, Knutsen, Alan P., additional, Kletzel, Morris, additional, Aquino, Victor, additional, Davis, Jeffrey H, additional, and Szabolcs, Paul, additional

Published
2023
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42. 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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44. National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease: III. The 2014 Biomarker Working Group Report

Author

Paczesny, Sophie, Hakim, Frances T., Pidala, Joseph, Cooke, Kenneth R., Lathrop, Julia, Griffith, Linda M., Hansen, John, Jagasia, Madan, Miklos, David, Pavletic, Steven, Parkman, Robertson, Russek-Cohen, Estelle, Flowers, Mary E.D., Lee, Stephanie, Martin, Paul, Vogelsang, Georgia, Walton, Marc, and Schultz, Kirk R.

Published
2015
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45. 116 - Event Free Survival in Severe Combined Immune Deficiency (SCID) Infants after Conditioned Umbilical Cord Blood Transplantation (UCBT) Benefits from Omitting Serotherapy

Author

Martinez, Caridad, Logan, Brent, Liu, Xuerong, Dvorak, Christopher C., Madden, Lisa, Molinari, Lyndsay, Cowan, Morton J., Pai, Sung-Yun, Haddad, Elie, Puck, Jennifer, Kohn, Donald B., Griffith, Linda M., Pulsipher, Michael, Leiding, Jennifer W., Notarangelo, Luigi D., Torgerson, Troy, Marsh, Rebecca A., Cuvelier, Geoff D.E., Prockop, Susan, Buckley, Rebecca H., Kuo, Caroline Y., Yip, Alison, Hershfield, Michael S., Parrott, Roberta E, Ebens, Christen L., Moore, Theodore B., O’Reilly, Richard J., Kapadia, Malika, Kapoor, Neena, Satter, Lisa Forbes, Burroughs, Lauri M., Petrovic, Aleksandra, Thakar, Monica S., Chellapandian, Deepak, Heimall, Jennifer R., Shyr, David C., Bednarski, Jeffrey J, Rayes, Ahmad, Chandrakasan, Shanmuganathan, Quigg, Troy C., Davila, Blachy J, DeSantes, Kenneth, Eissa, Hesham, Goldman, Frederick, Rozmus, Jacob, Shah, Ami J, Lugt, Mark Vander, Keller, Michael D., Sullivan, Kathleen E., Jyonouchi, Soma, Seroogy, Christine, Decaluwe, Helene, Teira, Pierre, Knutsen, Alan P., Kletzel, Morris, Aquino, Victor, Davis, Jeffrey H, and Szabolcs, Paul

Published
2023
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47. Transplantation for Autoimmune Diseases in North and South America: A Report of the Center for International Blood and Marrow Transplant Research

Author

Pasquini, Marcelo C., Voltarelli, Julio, Atkins, Harold L., Hamerschlak, Nelson, Zhong, Xiaobo, Ahn, Kwang Woo, Sullivan, Keith M., Carrum, George, Andrey, Jeffrey, Bredeson, Christopher N., Cairo, Mitchell, Gale, Robert Peter, Hahn, Theresa, Storek, Jan, Horowitz, Mary M., McSweeney, Peter A., Griffith, Linda M., Muraro, Paolo A., Pavletic, Steven Z., and Nash, Richard A.

Published
2012
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48. 125 - Hematopoietic Cell Transplantation in 240 Patients with Chronic Granulomatous Disease: A Pidtc Report

Author

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

Published
2022
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50. Outcomes after Hematopoietic Cell Transplant and Gene Therapy for Adenosine Deaminase (ADA) Severe Combined Immune Deficiency: A Combined Analysis from the Primary Immune Deficiency Treatment Consortium (PIDTC) 6901 and 6902 Studies

Author

Cuvelier, Geoff D.E., primary, Logan, Brent, additional, Prockop, Susan E., additional, Buckley, Rebecca H., additional, Kuo, Caroline Y., additional, Griffith, Linda M., additional, Liu, Xuerong, additional, Yip, Alison, additional, Hershfield, Michael S., additional, Parrott, Roberta E, additional, Ebens, Christen L., additional, Moore, Theodore B., additional, O’Reilly, Richard J., additional, Pai, Sung-Yun, additional, Kapadia, Malika, additional, Kapoor, Neena, additional, Forbes Satter, Lisa R., additional, Martinez, Caridad, additional, Burroughs, Lauri M., additional, Petrovic, Aleksandra, additional, Thakar, Monica S., additional, Chellapandian, Deepak, additional, Heimall, Jennifer, additional, Shyr, David C., additional, Rayes, Ahmad, additional, Bednarski II, Jeffrey J., additional, Chandra, Sharat, additional, Chandrakasan, Shanmuganathan, additional, Gillio, Alfred P., additional, Madden, Lisa, additional, Quigg, Troy C., additional, Caywood, Emi H., additional, Dávila Saldaña, Blachy J, additional, DeSantes, Kenneth, additional, Eissa, Hesham, additional, Goldman, Frederick, additional, Rozmus, Jacob, additional, Shah, Ami J, additional, Vander Lugt, Mark T., additional, Pulsipher, Mike A., additional, Notarangelo, Luigi D., additional, Cowan, Morton J., additional, Dvorak, Christopher C., additional, Haddad, Elie, additional, Puck, Jennifer, additional, and Kohn, Donald B., additional

Published
2022
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