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1,058 results on '"MacMillan, Margaret L"'

2. Racial, ethnic, and socioeconomic diversity and outcomes of patients with graft-versus-host disease: a CIBMTR analysis

Author

Farhadfar, Nosha, Rashid, Nahid, Chen, Karen, DeVos, Jakob, Wang, Tao, Ballen, Karen, Beitinjaneh, Amer, Bhatt, Vijaya Raj, Hamilton, Betty K., Hematti, Peiman, Gadalla, Shahinaz M., Solomon, Scott R., El Jurdi, Najla, Lee, Catherine J., MacMillan, Margaret L., Rangarajan, Hemalatha G., Schoemans, Hélène, Sharma, Akshay, Spellman, Stephen R., Wingard, John R., and Lee, Stephanie J.

Published
2024
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3. Anti-CD3/CD7 immunoconjugate (T-Guard) for severe, steroid-refractory GVHD: final report of BMT CTN 2002

Author

Meyers, Gabrielle, Hamadani, Mehdi, Martens, Michael, Ali, Haris, Chevallier, Patrice, Choe, Hannah, Harris, Andrew C., Holler, Ernst, van Hooren, Eric, Klaassen, Willem, Leifer, Eric, van Oosterhout, Ypke, Perez, Lia, Pusic, Iskra, Stelljes, Matthias, van der Velden, Walter, Ammatuna, Emanuele, Beauvais, David, Cornillon, Jérôme, Maziarz, Richard T., Schetelig, Johannes, Romeril, Jennifer, MacMillan, Margaret L., Levine, John E., and Socié, Gérard

Published
2023
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5. Alloengraftment without significant toxicity or GVHD in CD45 antibody-drug conjugate–conditioned Fanconi anemia mice

Author

Saha, Asim, Palchaudhuri, Rahul, Lanieri, Leanne, Hyzy, Sharon, Riddle, Megan J., Panthera, Jamie, Eide, Cindy R., Tolar, Jakub, Panoskaltsis-Mortari, Angela, Gorfinkel, Lev, Tkachev, Victor, Gerdemann, Ulrike, Alvarez-Calderon, Francesca, Palato, Elisa Rojas, MacMillan, Margaret L., Wagner, John E., Kean, Leslie S., Osborn, Mark J., Kiem, Hans-Peter, Scadden, David T., Olson, Lisa M., and Blazar, Bruce R.

Published
2024
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8. Genomic signature of Fanconi anaemia DNA repair pathway deficiency in cancer

Author

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

Published
2022
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10. Three prophylaxis regimens (tacrolimus, mycophenolate mofetil, and cyclophosphamide; tacrolimus, methotrexate, and bortezomib; or tacrolimus, methotrexate, and maraviroc) versus tacrolimus and methotrexate for prevention of graft-versus-host disease with haemopoietic cell transplantation with reduced-intensity conditioning: a randomised phase 2 trial with a non-randomised contemporaneous control group (BMT CTN 1203)

Author

Bolaños-Meade, Javier, Reshef, Ran, Fraser, Raphael, Fei, Mingwei, Abhyankar, Sunil, Al-Kadhimi, Zaid, Alousi, Amin M, Antin, Joseph H, Arai, Sally, Bickett, Kate, Chen, Yi-Bin, Damon, Lloyd E, Efebera, Yvonne A, Geller, Nancy L, Giralt, Sergio A, Hari, Parameswaran, Holtan, Shernan G, Horowitz, Mary M, Jacobsohn, David A, Jones, Richard J, Liesveld, Jane L, Logan, Brent R, MacMillan, Margaret L, Mielcarek, Marco, Noel, Pierre, Pidala, Joseph, Porter, David L, Pusic, Iskra, Sobecks, Ronald, Solomon, Scott R, Weisdorf, Daniel J, Wu, Juan, Pasquini, Marcelo C, and Koreth, John

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Clinical Trials and Supportive Activities, Hematology, Transplantation, Cancer, Rare Diseases, Clinical Research, 6.1 Pharmaceuticals, Evaluation of treatments and therapeutic interventions, Good Health and Well Being, Aged, Bortezomib, Cyclophosphamide, Drug Interactions, Female, Graft vs Host Disease, Hematopoietic Stem Cell Transplantation, Humans, Male, Maraviroc, Methotrexate, Middle Aged, Mycophenolic Acid, Tacrolimus, Transplantation Conditioning, Cardiorespiratory Medicine and Haematology, Clinical Sciences, Cardiovascular medicine and haematology
Abstract

BackgroundPrevention of graft-versus-host disease (GvHD) without malignant relapse is the overall goal of allogeneic haemopoietic cell transplantation (HCT). We aimed to evaluate regimens using either maraviroc, bortezomib, or post-transplantation cyclophosphamide for GvHD prophylaxis compared with controls receiving the combination of tacrolimus and methotrexate using a novel composite primary endpoint to identify the most promising intervention to be further tested in a phase 3 trial.MethodsIn this prospective multicentre phase 2 trial, adult patients aged 18-75 years who received reduced-intensity conditioning HCT were randomly assigned (1:1:1) by random block sizes to tacrolimus, mycophenolate mofetil, and post-transplantation cyclophosphamide (cyclophosphamide 50 mg/kg on days 3 and 4, followed by tacrolimus starting on day 5 and mycophenolate mofetil starting on day 5 at 15 mg/kg three times daily not to exceed 1 g from day 5 to day 35); tacrolimus, methotrexate, and bortezomib (bortezomib 1·3 mg/m2 intravenously on days 1, 4, and 7 after HCT); or tacrolimus, methotrexate, and maraviroc (maraviroc 300 mg orally twice daily from day -3 to day 30 after HCT). Methotrexate was administered as a 15 mg/m2 intravenous bolus on day 1 and 10 mg/m2 intravenous bolus on days 3, 6, and 11 after HCT; tacrolimus was given intravenously at a dose of 0·05 mg/kg twice daily (or oral equivalent) starting on day -3 (except the post-transplantation cyclophosphamide, as indicated), with a target level of 5-15 ng/mL. Tacrolimus was continued at least until day 90 and was tapered off by day 180. Each study group was compared separately to a contemporary non-randomised prospective cohort of patients (control group) who fulfilled the same eligibility criteria as the trial, but who were treated with tacrolimus and methotrexate at centres not participating in the trial. The primary endpoint (GvHD-free, relapse-free survival [GRFS]) was defined as the time from HCT to onset of grade 3-4 acute GvHD, chronic GvHD requiring systemic immunosuppression, disease relapse, or death. The study was analysed by modified intention to treat. The study is closed to accrual and this is the planned analysis. This trial is registered with ClinicalTrials.gov, number NCT02208037.FindingsBetween Nov 17, 2014, and May 18, 2016, 273 patients from 31 US centres were randomly assigned to the three study arms: 89 to tacrolimus, methotrexate, and bortezomib; 92 to tacrolimus, methotrexate, and maraviroc; 92 to tacrolimus, mycophenolate mofetil, and post-transplantation cyclophosphamide; and six were excluded. Between Aug 1, 2014, and Sept 14, 2016, 224 controls received tacrolimus and methotrexate. Controls were generally well matched except for more frequent comorbidities than the intervention groups and a different distribution of types of conditioning regimens used. Compared with controls, the hazard ratio for GRFS was 0·72 (90% CI 0·54-0·94; p=0·044) for tacrolimus, mycophenolate mofetil, and post-transplantation cyclophosphamide, 0·98 (0·76-1·27; p=0·92) for tacrolimus, methotrexate, and bortezomib, and 1·10 (0·86-1·41; p=0·49) for tacrolimus, methotrexate, and maraviroc. 238 patients experienced grade 3 or 4 toxicities: 12 (13%) had grade 3 and 67 (73%) grade 4 events with tacrolimus, mycophenolate mofetil, and post-transplantation cyclophosphamide; ten (11%) had grade 3 and 68 (76%) had grade 4 events with tacrolimus, methotrexate, and bortezomib; and 18 (20%) had grade 3 and 63 (68%) had grade 4 events with tacrolimus, methotrexate, and maraviroc. The most common toxicities were haematological (77 [84%] for tacrolimus, mycophenolate mofetil, and post-transplantation cyclophosphamide; 73 [82%] for tacrolimus, methotrexate, and bortezomib; and 78 [85%] for tacrolimus, methotrexate, and maraviroc) and cardiac (43 [47%], 44 [49%], and 43 [47%], respectively).InterpretationTacrolimus, mycophenolate mofetil, and post-transplantation cyclophosphamide was the most promising intervention, yielding the best GRFS; this regimen is thus being prospectively compared with tacrolimus and methotrexate in a phase 3 randomised trial.FundingUS National Health, Lung, and Blood Institute; National Cancer Institute; National Institute of Allergy and Infectious Disease; and Millennium Pharmaceuticals.

Published
2019

11. Characteristics of Graft-Versus-Host Disease (GvHD) After Post-Transplantation Cyclophosphamide Versus Conventional GvHD Prophylaxis

Author

Saliba, Rima M., Alousi, Amin M., Pidala, Joseph, Arora, Mukta, Spellman, Stephen R., Hemmer, Michael T., Wang, Tao, Abboud, Camille, Ahmed, Sairah, Antin, Joseph H., Beitinjaneh, Amer, Buchbinder, David, Byrne, Michael, Cahn, Jean-Yves, Choe, Hannah, Hanna, Rabi, Hematti, Peiman, Kamble, Rammurti T., Kitko, Carrie L., Laughlin, Mary, Lekakis, Lazaros, MacMillan, Margaret L., Martino, Rodrigo, Mehta, Parinda A., Nishihori, Taiga, Patel, Sagar S., Perales, Miguel-Angel, Rangarajan, Hemalatha G., Ringdén, Olov, Rosenthal, Joseph, Savani, Bipin N., Schultz, Kirk R., Seo, Sachiko, Teshima, Takanori, van der Poel, Marjolein, Verdonck, Leo F., Weisdorf, Daniel, Wirk, Baldeep, Yared, Jean A., Schriber, Jeffrey, Champlin, Richard E., and Ciurea, Stefan O.

Published
2022
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15. A CD45-targeted antibody-drug conjugate successfully conditions for allogeneic hematopoietic stem cell transplantation in mice

Author

Saha, Asim, Hyzy, Sharon, Lamothe, Tahirih, Hammond, Katelyn, Clark, Nicholas, Lanieri, Leanne, Bhattarai, Prashant, Palchaudhuri, Rahul, Gillard, Geoffrey O., Proctor, Jennifer, Riddle, Megan J., Panoskaltsis-Mortari, Angela, MacMillan, Margaret L., Wagner, John E., Kiem, Hans-Peter, Olson, Lisa M., and Blazar, Bruce R.

Published
2022
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16. Chronic Graft-versus-Host Disease, Nonrelapse Mortality, and Disease Relapse in Older versus Younger Adults Undergoing Matched Allogeneic Peripheral Blood Hematopoietic Cell Transplantation: A Center for International Blood and Marrow Transplant Research Analysis

Author

Bhatt, Vijaya Raj, Wang, Tao, Chen, Karen, Kitko, Carrie L., MacMillan, Margaret L., Pidala, Joseph A., Al Malki, Monzr M., Badawy, Sherif M., Beitinjaneh, Amer, Ganguly, Siddhartha, Hamilton, Betty, Hildebrandt, Gerhard C., Lekakis, Lazaros J., Liu, Hongtao, Maziarz, Richard T., Modi, Dipenkumar, Murthy, Hemant S., Preussler, Jaime M., Sharma, Akshay, Spellman, Stephen R., Arora, Mukta, and Lee, Stephanie J.

Published
2022
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18. Severity of Chronic Graft-versus-Host Disease and Late Effects Following Allogeneic Hematopoietic Cell Transplantation for Adults with Hematologic Malignancy

Author

Lee, Catherine J., Wang, Tao, Chen, Karen, Arora, Mukta, Brazauskas, Ruta, Spellman, Stephen R., Kitko, Carrie, MacMillan, Margaret L., Pidala, Joseph A., Badawy, Sherif M., Bhatt, Neel, Bhatt, Vijaya R., DeFilipp, Zachariah, Diaz, Miguel A., Farhadfar, Nosha, Gadalla, Shahinaz, Hashmi, Shahrukh, Hematti, Peiman, Hossain, Nasheed M., Inamoto, Yoshihiro, Lekakis, Lazaros J., Sharma, Akshay, Solomon, Scott, Lee, Stephanie J., and Couriel, Daniel R.

Published
2024
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19. Standardizing Definitions of Hematopoietic Recovery, Graft Rejection, Graft Failure, Poor Graft Function, and Donor Chimerism in Allogeneic Hematopoietic Cell Transplantation: A Report on Behalf of the American Society for Transplantation and Cellular Therapy

Author

Kharfan-Dabaja, Mohamed A., Kumar, Ambuj, Ayala, Ernesto, Aljurf, Mahmoud, Nishihori, Taiga, Marsh, Rebecca, Burroughs, Lauri M., Majhail, Navneet, Al-Homsi, A. Samer, Al-Kadhimi, Zaid S., Bar, Merav, Bertaina, Alice, Boelens, Jaap J., Champlin, Richard, Chaudhury, Sonali, DeFilipp, Zachariah, Dholaria, Bhagirathbhai, El-Jawahri, Areej, Fanning, Suzanne, Fraint, Ellen, Gergis, Usama, Giralt, Sergio, Hamilton, Betty K., Hashmi, Shahrukh K., Horn, Biljana, Inamoto, Yoshihiro, Jacobsohn, David A, Jain, Tania, Johnston, Laura, Kanate, Abraham S., Kansagra, Ankit, Kassim, Adetola, Kean, Leslie S., Kitko, Carrie L., Knight-Perry, Jessica, Kurtzberg, Joanne, Liu, Hien, MacMillan, Margaret L., Mahmoudjafari, Zahra, Mielcarek, Marco, Mohty, Mohamad, Nagler, Arnon, Nemecek, Eneida, Olson, Timothy S., Oran, Betul, Perales, Miguel-Angel, Prockop, Susan E., Pulsipher, Michael A., Pusic, Iskra, Riches, Marcie L., Rodriguez, Cesar, Romee, Rizwan, Rondon, Gabriela, Saad, Ayman, Shah, Nina, Shaw, Peter J., Shenoy, Shalini, Sierra, Jorge, Talano, Julie, Verneris, Michael R., Veys, Paul, Wagner, John E., Savani, Bipin N., Hamadani, Mehdi, and Carpenter, Paul A.

Published
2021
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22. 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

28. Pediatric acute promyelocytic leukemia and Fanconi anemia: Case report and literature review.

Author

Freycon, Claire, Sepulchre, Edith, Lavallée, Vincent‐Philippe, Mitchell, David, MacMillan, Margaret L., Vezina, Catherine, and Goudie, Catherine

Subjects
ACUTE promyelocytic leukemia, FANCONI'S anemia, LITERATURE reviews, ACUTE myeloid leukemia, STEM cell transplantation
Abstract

Acute promyelocytic leukemia (APL) represents 5%–10% of childhood acute myeloid leukemia (AML) and is the most curable subtype of AML. Fanconi anemia (FA) is one of the most common inherited bone marrow failure syndromes caused by biallelic pathogenic variants (PV) in specific DNA‐repair genes. Biallelic PVs in FANCD1/BRCA2 (FA‐D1) account for 3% of FA and are associated with early‐onset leukemia and a high risk of solid tumors. We report a 4 year‐old boy from non‐consanguineous parents diagnosed with standard risk APL. This child had café‐au‐lait spots and an extra thumb remnant. Genomic sequencing revealed two PV in FANCD1/BRCA2 confirming a diagnosis of FA‐D1. Chromosomal breakage studies were compatible with FA. Each parent carried one variant and had no personal history of cancer. Morphological then molecular remissions were achieved with all‐trans retinoic acid and Arsenic trioxide. This patient underwent haploidentical stem cell transplant. In addition to our patient, a literature search revealed four additional patients with APL/FA, with a total of three patients with FA‐D1. This raises the possibility of an association between such rare disorders. Practical management of APL in the setting of FA‐D1 is discussed with an overview of current evidence and knowledge gaps. [ABSTRACT FROM AUTHOR]

Published
2024
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29. Development of specific growth charts for children with Fanconi anemia.

Author

Barbus, Crystal, Rayannavar, Arpana, Miller, Bradley S., Jenkins, Mica J., Addo, O. Yaw, Rayes, Ahmad, Ahrweiler, Natasha, Olson, Alisha, Pohlkamp, Zachary, Wagner, John E., and MacMillan, Margaret L.

Abstract

Patients with Fanconi anemia (FA) are often perceived to have poor growth when general population growth curves are utilized. We hypothesize that FA patients have unique growth and aimed to create FA‐specific growth charts. Height and weight data from ages 0 to 20 years were extracted from medical records of patients treated at the Fanconi Anemia Comprehensive Care Clinic at the University of Minnesota. Height, weight, and BMI growth curves were generated and fitted to reference percentiles using the Lambda‐Mu‐Sigma method. FA‐specific percentiles were compared to WHO standards for ages 0–2 and CDC references for ages 2–20. In FA males, the 50th height‐ and weight‐for‐age percentiles overlap with the 3rd reference percentile. In FA females, only the 50th height‐for‐age percentile overlaps with the 3rd reference percentile. For weight, FA females show progressive growth failure between 6 and 24 months followed by stabilization around the 50th percentile. The FA BMI‐for‐age percentiles show similar patterns to the weight‐for‐age percentiles but have different timing of onset of adiposity rebound and broader variability in females. Growth in FA patients follows a different trajectory than available normative curves. FA‐specific growth charts may be useful to better guide accurate growth expectations, evaluations, and treatment. [ABSTRACT FROM AUTHOR]

Published
2024
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30. Risk Factors for Graft-versus-Host Disease in Haploidentical Hematopoietic Cell Transplantation Using Post-Transplant Cyclophosphamide

Author

Im, Annie, Rashidi, Armin, Wang, Tao, Hemmer, Michael, MacMillan, Margaret L., Pidala, Joseph, Jagasia, Madan, Pavletic, Steven, Majhail, Navneet S., Weisdorf, Daniel, Abdel-Azim, Hisham, Agrawal, Vaibhav, Al-Homsi, A. Samer, Aljurf, Mahmoud, Askar, Medhat, Auletta, Jeffery J., Bashey, Asad, Beitinjaneh, Amer, Bhatt, Vijaya Raj, Byrne, Michael, Cahn, Jean-Yves, Cairo, Mitchell, Castillo, Paul, Cerny, Jan, Chhabra, Saurabh, Choe, Hannah, Ciurea, Stefan, Daly, Andrew, Perez, Miguel Angel Diaz, Farhadfar, Nosha, Gadalla, Shahinaz M., Gale, Robert, Ganguly, Siddhartha, Gergis, Usama, Hanna, Rabi, Hematti, Peiman, Herzig, Roger, Hildebrandt, Gerhard C., Lad, Deepesh P., Lee, Catherine, Lehmann, Leslie, Lekakis, Lazaros, Kamble, Rammurti T., Kharfan-Dabaja, Mohamed A., Khandelwal, Pooja, Martino, Rodrigo, Murthy, Hemant S., Nishihori, Taiga, O'Brien, Tracey A., Olsson, Richard F., Patel, Sagar S., Perales, Miguel-Angel, Prestidge, Tim, Qayed, Muna, Romee, Rizwan, Schoemans, Hélène, Seo, Sachiko, Sharma, Akshay, Solh, Melhem, Strair, Roger, Teshima, Takanori, Urbano-Ispizua, Alvaro, Van der Poel, Marjolein, Vij, Ravi, Wagner, John L., William, Basem, Wirk, Baldeep, Yared, Jean A., Spellman, Steve R., Arora, Mukta, and Hamilton, Betty K.

Published
2020
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32. Facilitating resolution of life-threatening acute GVHD with human chorionic gonadotropin and epidermal growth factor

Author

Holtan, Shernan G., Hoeschen, Andrea L., Cao, Qing, Arora, Mukta, Bachanova, Veronika, Brunstein, Claudio G., Miller, Jeffrey S., Rashidi, Armin, Slungaard, Arne, Ustun, Celalettin, Vercellotti, Gregory M., Warlick, Erica D., Betts, Brian C., El Jurdi, Najla, He, Fiona, Chen, Chi, Gandhi, Isha, Wagner, John E., Blazar, Bruce R., Jacobson, Pamala Ann, Shabaneh, Ashraf, Wang, Jinhua, Panoskaltsis-Mortari, Angela, MacMillan, Margaret L., and Weisdorf, Daniel J.

Published
2020
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34. Randomized multicenter trial of sirolimus vs prednisone as initial therapy for standard-risk acute GVHD: the BMT CTN 1501 trial

Author

Pidala, Joseph, Hamadani, Mehdi, Dawson, Peter, Martens, Michael, Alousi, Amin M., Jagasia, Madan, Efebera, Yvonne A., Chhabra, Saurabh, Pusic, Iskra, Holtan, Shernan G., Ferrara, James L.M., Levine, John E., Mielcarek, Marco, Anasetti, Claudio, Antin, Joseph H., Bolaños-Meade, Javier, Howard, Alan, Logan, Brent R., Leifer, Eric S., Pritchard, Theresa S., Horowitz, Mary M., and MacMillan, Margaret L.

Published
2020
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37. A High Proportion of Abnormal Pulmonary Baseline Investigations in Pediatric BMT Patients: Prospective, Multi-Center Results from the Transpire Study.

Author

Cook, Eleanor, primary, Rose, Steven, additional, Goldfarb, Samuel B., additional, Abts, Matthew F., additional, Woods, Jason C., additional, Koo, Dr. Jane, additional, Cooper, Richard, additional, Lawson, Jessica, additional, Gilene, Stephen, additional, Ballard, Sheri, additional, MacMillan, Margaret L., additional, Baker, Scott, additional, Walkup, Laura L., additional, Edwards, Stephanie L., additional, Myers, Kasiani C, additional, Davies, Stella M., additional, and Towe, Christopher, additional

Published
2024
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38. Transpire: Lung Injury in a Longitudinal Cohort of Pediatric Hematopoietic Stem Cell Transplant Patients

Author

Koo, Dr. Jane, primary, Myers, Kasiani C, additional, Cook, Eleanor, additional, Cooper, Richard, additional, Lawson, Jessica, additional, Edwards, Stephanie L., additional, Loveless, Sara Katherine, additional, Strecker, Lauren, additional, Lake, Kelly E., additional, Towe, Christopher, additional, Patti, Jessica, additional, Stevens, Carrie Ann, additional, Walkup, Laura L., additional, Wikenheiser-Brokamp, Kathryn A., additional, MacMillan, Margaret L., additional, Lacher, Philip, additional, Griffin, Tamara, additional, Tekman, Merve, additional, Melton, Alexis, additional, Noori, Sana, additional, Zinter, Matt S., additional, Urrego, Fernando A., additional, Baker, K. Scott, additional, Abts, Matthew F., additional, Ballard, Sheri, additional, Freedman, Jason L., additional, Flynn, David, additional, Massi, Kayla, additional, Quijano, Kaitlyn N, additional, Young, Lisa R., additional, Josephson, Maureen B., additional, Allen, Julian L., additional, Camburn, Devaney M., additional, Doherty, Erin E, additional, Azamian, Mashid Sababi, additional, Arredondo, Martha, additional, Silva-Carmona, Manuel, additional, Lehmann, Leslie E., additional, Wong, Wai, additional, Gaffin, Jonathan M., additional, Sjoquist, Julia, additional, Li, Mona, additional, Goldfarb, Samuel B., additional, Woods, Jason C., additional, and Davies, Stella M., additional

Published
2024
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39. Success and Feasibility of Multiple Breath Washout: Prospective, Multi-Center Results from the Transpire Study

Author

Abts, Matthew F., primary, Silva-Carmona, Manuel, additional, Allen, Julian L., additional, Doherty, Erin E, additional, Baker, Scott, additional, MacMillan, Margaret L., additional, Genatassio, Alan, additional, Lacher, Philip, additional, Griffin, Tamara, additional, Tekman, Merve, additional, Ballard, Sheri, additional, Azamian, Mashid Sababi, additional, Schimelpfenig, Bri, additional, Koo, Jane, additional, Edwards, Stephanie L., additional, Woods, Jason C., additional, Davies, Stella M., additional, and Goldfarb, Samuel B., additional

Published
2024
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40. Measurement of Respiratory System Function By Airwave Oscillometry (AOS) in a Cohort of Children Undergoing Hematopoietic Stem Cell Transplant (HSCT)

Author

Josephson, Maureen B., primary, McDonough, Joseph M., additional, Young, Lisa R., additional, Freedman, Jason L., additional, Towe, Christopher, additional, Cook, Eleanor, additional, Gaffin, Jonathan M., additional, Koo, Dr. Jane, additional, MacMillan, Margaret L., additional, Myers, Kasiani C, additional, Lehmann, Leslie E., additional, Walkup, Laura L., additional, Wong, Wai, additional, Woods, Jason C., additional, Davies, Stella M., additional, Goldfarb, Samuel B., additional, and Allen, Julian L., additional

Published
2024
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41. Breathing New Life into an Old Problem: Pulmonary Complications after Autologous Hematopoietic Stem Cell Transplant

Author

Koo, Dr. Jane, primary, Gupta, Ashish O., additional, Marr, Anna, additional, Torabi, Makan, additional, Shanley, Ryan, additional, Myers, Kasiani C, additional, Towe, Christopher, additional, Cooper, Richard, additional, Edwards, Stephanie L., additional, Cook, Eleanor, additional, Walkup, Laura L., additional, MacMillan, Margaret L., additional, Strecker, Lauren, additional, Woods, Jason C., additional, Davies, Stella M., additional, Dandoy, Dr. Christopher E., additional, and Goldfarb, Samuel B., additional

Published
2024
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42. Graft Vs Host Disease (GVHD) in Pediatric Hematopoietic Stem Cell Transplant (HCT) Recipients and Impact on Overall Survival: A CIBMTR Analysis

Author

Nishitani, Miki, primary, Graham, Rudi-Ann Tamara, additional, Wang, Tao, additional, DeVos, Jakob D, additional, Lee, Stephanie J, additional, Spellman, Stephen R., additional, Kitko, Carrie L., additional, MacMillan, Margaret L., additional, DeFilipp, Zachariah, additional, Duncan, Christine N., additional, and Qayed, Muna, additional

Published
2024
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43. Stat5 is critical for the development and maintenance of myeloproliferative neoplasm initiated by Nf1 deficiency

Author

Sachs, Zohar, Been, Raha A, DeCoursin, Krista J, Nguyen, Hanh T, Mohd Hassan, Nurul A, Noble-Orcutt, Klara E, Eckfeldt, Craig E, Pomeroy, Emily J, Diaz-Flores, Ernesto, Geurts, Jennifer L, Diers, Miechaleen D, Hasz, Diane E, Morgan, Kelly J, MacMillan, Margaret L, Shannon, Kevin M, Largaespada, David A, and Wiesner, Stephen M

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Childhood Leukemia, Genetics, Cancer, Pediatric Cancer, Pediatric, Rare Diseases, Hematology, Neurofibromatosis, Neurosciences, 2.1 Biological and endogenous factors, Aetiology, Animals, Disease Models, Animal, Humans, Janus Kinase 2, Leukemia, Myeloid, Leukemia, Myelomonocytic, Juvenile, Mice, Myeloproliferative Disorders, Neurofibromin 1, Proto-Oncogene Proteins p21(ras), STAT5 Transcription Factor, Signal Transduction, Cardiorespiratory Medicine and Haematology, Immunology, Cardiovascular medicine and haematology
Abstract

Juvenile myelomonocytic leukemia is a rare myeloproliferative neoplasm characterized by hyperactive RAS signaling. Neurofibromin1 (encoded by the NF1 gene) is a negative regulator of RAS activation. Patients with neurofibromatosis type 1 harbor loss-of-function mutations in NF1 and have a 200- to 500-fold increased risk of juvenile myelomonocytic leukemia. Leukemia cells from patients with juvenile myelomonocytic leukemia display hypersensitivity to certain cytokines, such as granulocyte-macrophage colony-stimulating factor. The granulocyte-macrophage colony-stimulating factor receptor utilizes pre-associated JAK2 to initiate signals after ligand binding. JAK2 subsequently activates STAT5, among other downstream effectors. Although STAT5 is gaining recognition as an important mediator of growth factor signaling in myeloid leukemias, the contribution of STAT5 to the development of hyperactive RAS-initiated myeloproliferative disease has not been well described. In this study, we investigated the consequence of STAT5 attenuation via genetic and pharmacological approaches in Nf1-deficient murine models of juvenile myelomonocytic leukemia. We found that homozygous Stat5 deficiency extended the lifespan of Nf1-deficient mice and eliminated the development of myeloproliferative neoplasm associated with Nf1 gene loss. Likewise, we found that JAK inhibition with ruxolitinib attenuated myeloproliferative neoplasm in Nf1-deficient mice. Finally, we found that primary cells from a patient with KRAS-mutant juvenile myelomonocytic leukemia displayed reduced colony formation in response to JAK2 inhibition. Our findings establish a central role for STAT5 activation in the pathogenesis of juvenile myelomonocytic leukemia and suggest that targeting this pathway may be of clinical utility in these patients.

Published
2016

44. Infection Rates among Acute Leukemia Patients Receiving Alternative Donor Hematopoietic Cell Transplantation

Author

Ballen, Karen, Ahn, Kwang Woo, Chen, Min, Abdel-Azim, Hisham, Ahmed, Ibrahim, Aljurf, Mahmoud, Antin, Joseph, Bhatt, Ami S, Boeckh, Michael, Chen, George, Dandoy, Christopher, George, Biju, Laughlin, Mary J, Lazarus, Hillard M, MacMillan, Margaret L, Margolis, David A, Marks, David I, Norkin, Maxim, Rosenthal, Joseph, Saad, Ayman, Savani, Bipin, Schouten, Harry C, Storek, Jan, Szabolcs, Paul, Ustun, Celalettin, Verneris, Michael R, Waller, Edmund K, Weisdorf, Daniel J, Williams, Kirsten M, Wingard, John R, Wirk, Baldeep, Wolfs, Tom, Young, Jo-Anne H, Auletta, Jeffrey, Komanduri, Krishna V, Lindemans, Caroline, and Riches, Marcie L

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Immunology, Rare Diseases, Stem Cell Research, Infectious Diseases, Clinical Research, Regenerative Medicine, Transplantation, Hematology, Stem Cell Research - Nonembryonic - Human, 2.2 Factors relating to the physical environment, Aetiology, Infection, Good Health and Well Being, Acute Disease, Adolescent, Adult, Aged, Cord Blood Stem Cell Transplantation, Female, Hematopoietic Stem Cell Transplantation, Histocompatibility, Humans, Incidence, Infections, Leukemia, Male, Middle Aged, Retrospective Studies, Survival Analysis, Unrelated Donors, Young Adult, Umbilical cord blood, Clinical Sciences, Cardiovascular medicine and haematology
Abstract

Alternative graft sources (umbilical cord blood [UCB], matched unrelated donors [MUD], or mismatched unrelated donors [MMUD]) enable patients without a matched sibling donor to receive potentially curative hematopoietic cell transplantation (HCT). Retrospective studies demonstrate comparable outcomes among different graft sources. However, the risk and types of infections have not been compared among graft sources. Such information may influence the choice of a particular graft source. We compared the incidence of bacterial, viral, and fungal infections in 1781 adults with acute leukemia who received alternative donor HCT (UCB, n= 568; MUD, n = 930; MMUD, n = 283) between 2008 and 2011. The incidences of bacterial infection at 1 year were 72%, 59%, and 65% (P

Published
2016

45. Phase II Study of Myeloablative 7-8/8-Matched Allotransplantation with Post-Transplantation Cyclophosphamide, Tacrolimus, and Mycophenolate Mofetil

Author

Jurdi, Najla El, Hoover, Alex, O'Leary, Daniel, Cao, Qing, Gupta, Ashish, Ebens, Christen, Maakaron, Joseph E., Betts, Brian C., Rashidi, Armin, Juckett, Mark B., Lund, Troy, Bachanova, Veronika, MacMillan, Margaret L, Miller, Jeffrey S., Orchard, Paul J., Wagner, John E., Vercellotti, Gregory, Weisdorf, Daniel J., Dusenbery, Kathryn, Terezakis, Stephanie, and Holtan, Shernan G.

Published
2023
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46. Phase II, Open-Label Clinical Trial of Urinary-Derived Human Chorionic Gonadotropin/Epidermal Growth Factor for Life-Threatening Acute Graft-versus-Host Disease

Author

Holtan, Shernan G., Hoeschen, Andrea, Cao, Qing, Ustun, Celalettin, Betts, Brian C., Jurdi, Najla El, Maakaron, Joseph, Rashidi, Armin, Miller, Jeffrey S., Wagner, John E., Blazar, Bruce R., Jacobson, Pamala A., Panoskaltsis-Mortari, Angela, Weisdorf, Daniel J., and MacMillan, Margaret L.

Published
2023
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48. Bone Mineral Density in Children with Fanconi Anemia after Hematopoietic Cell Transplantation

Author

Petryk, Anna, Polgreen, Lynda E, Barnum, Jessie L, Zhang, Lei, Hodges, James S, Baker, K Scott, Wagner, John E, Steinberger, Julia, and MacMillan, Margaret L

Subjects
Pediatric, Osteoporosis, Rare Diseases, Hematology, Pediatric Cancer, Clinical Research, Cancer, Transplantation, 2.1 Biological and endogenous factors, Aetiology, Musculoskeletal, Adolescent, Adult, Allografts, Bone Density, Child, Cross-Sectional Studies, Fanconi Anemia, Female, Follow-Up Studies, Hematopoietic Stem Cell Transplantation, Humans, Male, Middle Aged, Dual energy x-ray absorptiometry scan, Bone mineral density, Bone marrow transplantation, Fanconi anemia, Children, Clinical Sciences, Immunology
Abstract

Fanconi anemia (FA) is an inherited DNA repair disorder associated with short stature and bone marrow failure, usually requiring hematopoietic cell transplantation (HCT). Although low bone mineral density (BMD) has been reported in leukemia patients after HCT, little is known about BMD in FA children after HCT (FA HCT). This study's goals were to compare BMD in FA HCT to BMD in healthy controls and in children who received HCT for hematologic malignancy (cancer HCT), and to test for associations between BMD and risk factors for bone loss. This cross-sectional study included 20 FA HCT, 13 cancer HCT, and 90 healthy controls, age-matched and

Published
2015

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