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1. National Landscape of Human Immunodeficiency Virus-Positive Deceased Organ Donors in the United States.

Author

Werbel, William A, Brown, Diane M, Kusemiju, Oyinkansola T, Doby, Brianna L, Seaman, Shanti M, Redd, Andrew D, Eby, Yolanda, Fernandez, Reinaldo E, Desai, Niraj M, Miller, Jernelle, Bismut, Gilad A, Kirby, Charles S, Schmidt, Haley A, Clarke, William A, Seisa, Michael, Petropoulos, Christos J, Quinn, Thomas C, Florman, Sander S, Huprikar, Shirish, Rana, Meenakshi M, Friedman-Moraco, Rachel J, Mehta, Aneesh K, Stock, Peter G, Price, Jennifer C, Stosor, Valentina, Mehta, Shikha G, Gilbert, Alexander J, Elias, Nahel, Morris, Michele I, Mehta, Sapna A, Small, Catherine B, Haidar, Ghady, Malinis, Maricar, Husson, Jennifer S, Pereira, Marcus R, Gupta, Gaurav, Hand, Jonathan, Kirchner, Varvara A, Agarwal, Avinash, Aslam, Saima, Blumberg, Emily A, Wolfe, Cameron R, Myer, Kevin, Wood, R Patrick, Neidlinger, Nikole, Strell, Sara, Shuck, Marion, Wilkins, Harry, Wadsworth, Matthew, Motter, Jennifer D, Odim, Jonah, Segev, Dorry L, Durand, Christine M, Tobian, Aaron AR, Piquant, Dominque, Link, Katherine, Hemmersbach-Miller, Marion, Pearson, Thomas, Turgeon, Nicole, Lyon, G Marshall, Kitchens, William, Huckaby, Jeryl, Lasseter, A Francie, Elbein, Rivka, Roberson, April, Ferry, Elizabeth, Klock, Ethan, Cochran, Willa V, Morrison, Michelle, Rasmussen, Sarah, Bollinger, Juli, Sugarman, Jeremy, Smith, Angela R, Thomas, Margaret, Coakley, Margaret, Timpone, Joseph, Stucke, Alyssa, Haydel, Brandy, Dieter, Rebecca, Klein, Elizabeth J, Neumann, Henry, Gallon, Lorenzo, Goudy, Leah, Callegari, Michelle, Marrazzo, Ilise, Jackson, Towanda, Pruett, Timothy, Farnsworth, Mary, Locke, Jayme E, Mompoint-Williams, Darnell, Basinger, Katherine, Mekeel, Kristin, Nguyen, Phirum, Kwan, Joanne, Srisengfa, Tab, Chin-Hong, Peter, Rogers, Rodney, Simkins, Jacques, Munoz, Carlos, and Dunn, Ty

Subjects
Pediatric Research Initiative, HIV/AIDS, Infectious Diseases, 6.1 Pharmaceuticals, Evaluation of treatments and therapeutic interventions, Infection, Good Health and Well Being, Anti-Retroviral Agents, HIV, HIV Infections, HIV Seropositivity, Humans, Integrases, Prospective Studies, Tissue Donors, United States, Viral Load, transplant, organ donation, drug resistance, HOPE in Action Investigators, Biological Sciences, Medical and Health Sciences, Microbiology
Abstract

BackgroundOrgan transplantation from donors with human immunodeficiency virus (HIV) to recipients with HIV (HIV D+/R+) presents risks of donor-derived infections. Understanding clinical, immunologic, and virologic characteristics of HIV-positive donors is critical for safety.MethodsWe performed a prospective study of donors with HIV-positive and HIV false-positive (FP) test results within the HIV Organ Policy Equity (HOPE) Act in Action studies of HIV D+/R+ transplantation (ClinicalTrials.gov NCT02602262, NCT03500315, and NCT03734393). We compared clinical characteristics in HIV-positive versus FP donors. We measured CD4 T cells, HIV viral load (VL), drug resistance mutations (DRMs), coreceptor tropism, and serum antiretroviral therapy (ART) detection, using mass spectrometry in HIV-positive donors.ResultsBetween March 2016 and March 2020, 92 donors (58 HIV positive, 34 FP), representing 98.9% of all US HOPE donors during this period, donated 177 organs (131 kidneys and 46 livers). Each year the number of donors increased. The prevalence of hepatitis B (16% vs 0%), syphilis (16% vs 0%), and cytomegalovirus (CMV; 91% vs 58%) was higher in HIV-positive versus FP donors; the prevalences of hepatitis C viremia were similar (2% vs 6%). Most HIV-positive donors (71%) had a known HIV diagnosis, of whom 90% were prescribed ART and 68% had a VL

Published
2022

2. 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

3. 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

4. Related and unrelated donor transplantation for β-thalassemia major: results of an international survey

Author

Li, Chunfu, Mathews, Vikram, Kim, Soyoung, George, Biju, Hebert, Kyle, Jiang, Hua, Li, Changgang, Zhu, Yiping, Keesler, Daniel A, Boelens, Jaap Jan, Dvorak, Christopher C, Agarwal, Rajni, Auletta, Jeffery J, Goyal, Rakesh K, Hanna, Rabi, Kasow, Kimberly, Shenoy, Shalini, Smith, Angela R, Walters, Mark C, and Eapen, Mary

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Immunology, Regenerative Medicine, Clinical Research, Transplantation, Adolescent, Adult, Age Factors, Child, Child, Preschool, Hematopoietic Stem Cell Transplantation, Histocompatibility, Humans, Myeloablative Agonists, Surveys and Questionnaires, Survival Analysis, Tissue Donors, Transplantation Conditioning, Transplantation, Homologous, Unrelated Donors, Young Adult, beta-Thalassemia, Cardiovascular medicine and haematology
Abstract

We studied 1110 patients with β-thalassemia major aged ≤25 years who received transplants with grafts from HLA-matched related (n = 677; 61%), HLA-mismatched related (n = 78; 7%), HLA-matched unrelated (n = 252; 23%), and HLA-mismatched unrelated (n = 103; 9%) donors between 2000 and 2016. Ninety percent of transplants were performed in the last decade. Eight-five percent of patients received ≥20 transfusions and 88% were inadequately chelated. All patients received myeloablative-conditioning regimen. Overall and event-free survival were highest for patients aged ≤6 years and after HLA-matched related and HLA-matched unrelated donor transplantation. The 5-year probabilities of overall survival for patients aged ≤6 years, 7 to 15 years, and 16 to 25 years, adjusted for donor type and conditioning regimen were 90%, 84%, and 63%, respectively (P < .001). The corresponding probabilities for event-free survival were 86%, 80%, and 63% (P < .001). Overall and event-free survival did not differ between HLA-matched related and HLA-matched unrelated donor transplantation (89% vs 87% and 86% vs 82%, respectively). Corresponding probabilities after mismatched related and mismatched unrelated donor transplantation were 73% vs 83% and 70% vs 78%. In conclusion, if transplantation is considered as a treatment option it should be offered early (age ≤6 years). An HLA-matched unrelated donor is a suitable alternative if an HLA-matched relative is not available.

Published
2019

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

Author

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

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

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

Published
2019

6. 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

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

Author

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

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

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

Published
2017

12. 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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16. 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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17. 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

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

Author

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

Published
2019
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19. 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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20. 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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22. 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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23. Personalized Prognostic Risk Score for Long-Term Survival for Children with Acute Leukemia after Allogeneic Transplantation

Author

Bitan, Menachem, Ahn, Kwang Woo, Millard, Heather R., Pulsipher, Michael A., Abdel-Azim, Hisham, Auletta, Jeffery J., Brown, Valerie, Chan, Ka Wah, Diaz, Miguel Angel, Dietz, Andrew, Vincent, Marta González, Guilcher, Gregory, Hale, Gregory A., Hayashi, Robert J., Keating, Amy, Mehta, Parinda, Myers, Kasiani, Page, Kristin, Prestidge, Tim, Shah, Nirali N., Smith, Angela R., Woolfrey, Ann, Thiel, Elizabeth, Davies, Stella M., and Eapen, Mary

Published
2017
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25. 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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42. Supplement to: Transplantation of 240 patients with Severe Combined Immunodeficiency (SCID) from 2000-2009: A Primary Immune Deficiency Treatment Consortium report

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 OʼReilly, Richard J.

Published
2014

47. Predictive Value of C‐Reactive Protein and Albumin for Temporal Within‐Individual Pharmacokinetic Variability of Voriconazole in Pediatric Patients Undergoing Hematopoietic Cell Transplantation.

Author

Takahashi, Takuto, Jaber, Mutaz M., Smith, Angela R., Jacobson, Pamala A., Fisher, James, and Kirstein, Mark N.

Subjects
C-reactive protein, ALBUMINS, BIOMARKERS, VORICONAZOLE, INTRAVENOUS therapy, BODY weight, CYTOCHROME P-450, BIOAVAILABILITY, METABOLIC clearance rate, PHENOMENOLOGICAL biology, PEDIATRICS, TREATMENT effectiveness, DESCRIPTIVE statistics, DRUG monitoring, HEMATOPOIETIC stem cell transplantation, PHENOTYPES, CHILDREN
Abstract

Voriconazole is a widely used antifungal agent in immunocompromised patients, but its utility is limited by its variable exposure and narrow therapeutic index. Population pharmacokinetic (PK) models have been used to characterize voriconazole PK and derive individualized dosing regimens. However, determinants of temporal within‐patient variability of voriconazole PK were not well established. We aimed to characterize temporal variability of voriconazole PK within individuals and identify predictive clinical factors. This study was conducted as a part of a single‐institution, phase I study of intravenous voriconazole in children undergoing hematopoietic cell transplantation (NCT02227797). We analyzed voriconazole PK study data collected at week 1 and again at week 2 after the start of voriconazole therapy in 59 pediatric patients undergoing HCT (age <21 years). Population PK analysis using nonlinear mixed effect modeling was performed to analyze temporal within‐individual variability of voriconazole PK by incorporating a between‐occasion variability term in the model. A 2‐compartment linear elimination model incorporating body weight and cytochrome P450 2C19 phenotype described the data. The ratio of individual voriconazole clearance between weeks 1 and 2 ranged from 0.11 to 3.3 (−9.1 to +3.3‐fold change). Incorporation of covariate effects by serum C‐reactive protein and albumin levels decreased between‐occasion variability of clearance as compared to the model without them (coefficient of variation, 41.2% and 59.5%, respectively) and improved the model fit (P <.05). As significant covariates on voriconazole PK, C‐reactive protein and albumin concentrations may potentially serve as useful biomarkers as part of therapeutic drug monitoring. [ABSTRACT FROM AUTHOR]

Published
2022
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