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29 results on '"Schaefer, Edo"'

1. 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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4. Adenoviral Cellular Therapy with Adenovirus (ADV)-Specific Cytotoxic T-Lymphocytes (CTLs) for Resistant Infections in Children, Adolescent, and Young Adult (CAYA) Patients Following Allogeneic Hematopoietic Stem Cell Transplantation (AlloHSCT): A Promising Approach

Author

Schaefer, Edo, primary, Vu, Christine, additional, Flower, Allyson, additional, Ayello, Janet, additional, Chu, Yaya, additional, van de Ven, Carmella, additional, Mintzer, Elizabeth, additional, Harrison, Lauren, additional, Morris, Erin, additional, Wang, Yongping, additional, Shenoy, Shalini, additional, Lee, Dean Anthony, additional, Abu-Arja, Rolla, additional, Johnson, Bryon D, additional, Verneris, Michael R., additional, Dvorak, Christopher C., additional, Chu, Julia, additional, Cooke, Kenneth R., additional, Talano, Julie A, additional, Bunin, Nancy J., additional, and Cairo, Mitchell S., additional

Published
2024
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5. Inflammation-mediated fibroblast activation and immune dysregulation in collagen VII-deficient skin

Author

Anderson-Crannage, Morgan, primary, Ascensión, Alex M., additional, Ibanez-Solé, Olga, additional, Zhu, Hongwen, additional, Schaefer, Edo, additional, Ottomanelli, Darcy, additional, Hochberg, Bruno, additional, Pan, Jian, additional, Luo, Wen, additional, Tian, Meijuan, additional, Chu, Yaya, additional, Cairo, Mitchell S., additional, Izeta, Ander, additional, and Liao, Yanling, additional

Published
2023
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6. A pilot safety, feasibility and efficacy study of an oral anticoagulant, apixaban, in secondary prophylaxis of a primary venous thromboembolism in children and adolescents

Author

Pinchinat, Ashley, primary, Levendoglu‐Tugal, Oya, additional, Perez, Yara, additional, Thatcher, Emily, additional, Otero, Neida, additional, Budhram, Arvind, additional, Mahanti, Harshini, additional, Morris, Erin, additional, Klejmont, Liana, additional, Schaefer, Edo, additional, Brudnicki, Adele, additional, Friedman, Deborah, additional, Li, Simon, additional, and Cairo, Mitchell S., additional

Published
2023
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7. Revisiting Pre-Transplant Testicular Radiation for Relapse Prophylaxis in Allogeneic Hematopoietic Cell Transplantation (HCT) for Acute Lymphoblastic Leukemia (ALL)

Author

Summers, Corinne, primary, Bhatt, Neel S., additional, Jenssen, Kari, additional, Hoover, Alex, additional, Ebens, Christen L., additional, Schaefer, Edo, additional, Cairo, Mitchell S., additional, Carpenter, Paul A., additional, Dahlberg, Ann, additional, Hadland, Brandon, additional, Bleakley, Marie, additional, and Thakar, Monica S., additional

Published
2023
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9. Determining the Safety and Efficacy of Prophylactic Defibrotide Administration in Children, Adolescents, and Young Adults with High-Risk Sickle Cell Disease (SCD) or Beta Thalassemia Major Following Myeloimmunoablative Conditioning (MAC) and Haploidentical Stem Cell Transplantation Utilizing CD34+ Selection and T-Cell (CD3) Addback (IND127812)

Author

Schaefer, Edo, primary, Milner, Jordan, additional, Hung, Justine, additional, Ayello, Janet, additional, Shi, Qiuhu, additional, Talano, Julie, additional, Moore, Theodore B., additional, Friedman, Deborah, additional, Dozor, Allen J, additional, Klejmont, Liana, additional, Mahanti, Harshini, additional, Morris, Erin, additional, Fabricatore, Ms. Sandra, additional, Mintzer, Elizabeth, additional, Guillerman, R. Paul, additional, Flower, Allyson, additional, Cooke, Kenneth R., additional, and Cairo, Mitchell S., additional

Published
2023
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10. Feasibility, Safety, and Efficacy of Liposomal Amphotericin B Followed By Micafungin Prophylaxis of Invasive Fungal Infections in Pediatric Allogeneic Stem Cell Transplantation Recipients with Hematological Malignancies

Author

Schaefer, Edo, primary, Klejmont, Liana, additional, Gardenswartz, Aliza, additional, Cassey, Paula, additional, Mejia, Monica, additional, van de Ven, Carmella, additional, Fabricatore, Ms. Sandra, additional, Harrison, Lauren, additional, Morris, Erin, additional, and Cairo, Mitchell S., additional

Published
2023
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11. 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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12. Revisiting the Role of Post-Transplant Central Nervous System Prophylaxis in Allogeneic Hematopoietic Cell Transplantation for Acute Lymphoblastic Leukemia

Author

Bhatt, Neel S., primary, Summers, Corinne, additional, Jenssen, Kari, additional, Hoover, Alex, additional, Ebens, Christen L., additional, Schaefer, Edo, additional, Cairo, Mitchell S., additional, Carpenter, Paul A., additional, Dahlberg, Ann, additional, Hadland, Brandon, additional, Bleakley, Marie, additional, and Thakar, Monica S., additional

Published
2023
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13. A Phase II Study to Evaluate the Safety and Efficacy of Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome (IND 127812)

Author

Schaefer, Edo, primary, Kanarfogel, Temima, additional, Hochberg, Jessica C, additional, Thatcher, Emily, additional, Klejmont, Liana, additional, Chu, Yaya, additional, Shi, Qiuhu, additional, Dozor, Allen J, additional, Friedman, Deborah, additional, Mahanti, Harshini, additional, Morris, Erin, additional, Fabricatore, Sandra, additional, DeFresne, John, additional, Cooke, Kenneth R., additional, and Cairo, Mitchell S, additional

Published
2022
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16. Determining the Safety and Efficacy of Prophylactic Defibrotide Administration in Children, Adolescents, and Young Adults with High-Risk Sickle Cell Disease (SCD) or Beta Thalassemia Major Following Myeloimmunoablative Conditioning (MAC) and Haploidentical Stem Cell Transplantation Utilizing CD34+ Selection and T-Cell (CD3) Addback (IND127812)

Author

Schaefer, Edo, primary, Milner, Jordan, additional, Hung, Justine, additional, Ayello, Janet, additional, Shi, Qiuhu, additional, Talano, Julie-An, additional, Moore, Theodore B., additional, Friedman, Deborah, additional, Dozor, Allen J, additional, Klejmont, Liana, additional, Mahanti, Harshini, additional, Morris, Erin, additional, Fabricatore, Sandra, additional, Mintzer, Elizabeth, additional, Flower, Allyson, additional, Cooke, Kenneth R., additional, and Cairo, Mitch S., additional

Published
2022
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20. 394 - Determining the Safety and Efficacy of Prophylactic Defibrotide Administration in Children, Adolescents, and Young Adults with High-Risk Sickle Cell Disease (SCD) or Beta Thalassemia Major Following Myeloimmunoablative Conditioning (MAC) and Haploidentical Stem Cell Transplantation Utilizing CD34+ Selection and T-Cell (CD3) Addback (IND127812)

Author

Schaefer, Edo, Milner, Jordan, Hung, Justine, Ayello, Janet, Shi, Qiuhu, Talano, Julie, Moore, Theodore B., Friedman, Deborah, Dozor, Allen J, Klejmont, Liana, Mahanti, Harshini, Morris, Erin, Fabricatore, Ms. Sandra, Mintzer, Elizabeth, Guillerman, R. Paul, Flower, Allyson, Cooke, Kenneth R., and Cairo, Mitchell S.

Published
2023
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21. 382 - Allogenic Hematopoietic Cell Transplantations Are Effective in Patients with 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., Murguía-Favela, Luis, Mousallem, Talal I., Prasad, Prof. Vinod K., Teira, Pierre, Touzot, Fabien, Bunin, Nancy J., Heimall, Jennifer R., Burroughs, Lauri M., Kapadia, Malika, Prockop, Susan, Chandra, Sharat, Chandrakasan, Shanmuganathan, Chaudhury, Sonali, Broglie, Larisa, O’Reilly, Richard J., Dávila Saldaña, Blachy J., Schaefer, Edo, Chong, Hey, Bednarski, Jeffrey J., II, Rayes, Ahmad, DeSantes, Kenneth, Kohn, Donald B., Notarangelo, Luigi D., Pai, Sung-Yun, Puck, Jennifer, Torgerson, Troy, Cowan, Morton J., Dvorak, Christopher C., Satter, Lisa Forbes, Haddad, Elie, Pulsipher, Michael, Malech, Harry L., Kang, Elizabeth M., and Leiding, Jennifer W.

Published
2023
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24. 438 - Determining the Safety and Efficacy of Prophylactic Defibrotide Administration in Children, Adolescents, and Young Adults with High-Risk Sickle Cell Disease (SCD) or Beta Thalassemia Major Following Myeloimmunoablative Conditioning (MAC) and Haploidentical Stem Cell Transplantation Utilizing CD34+ Selection and T-Cell (CD3) Addback (IND127812)

Author

Schaefer, Edo, Milner, Jordan, Hung, Justine, Ayello, Janet, Shi, Qiuhu, Talano, Julie-An, Moore, Theodore B., Friedman, Deborah, Dozor, Allen J, Klejmont, Liana, Mahanti, Harshini, Morris, Erin, Fabricatore, Sandra, Mintzer, Elizabeth, Flower, Allyson, Cooke, Kenneth R., and Cairo, Mitch S.

Published
2022
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26. Treatment of Refractory Adenovirus (ADV) Infections Using Adv-Specific Cytotoxic T-Lymphocytes (CTLs) in Children, Adolescents and Young Adults (CAYA) Post Allogeneic Hematopoietic Stem Cell Transplantation (AlloHSCT), Solid Organ Transplantation (SOT), or with Primary Immunodeficiency (PID)

Author

Schaefer, Edo, Vu, Christine, Flower, Allyson, Ayello, Janet, Chu, Yaya, Van De Ven, Carmella, Mintzer, Elizabeth, Harrison, Lauren, Morris, Erin, Wang, Yongping, Shenoy, Shalini, Lee, Dean Anthony, ABU-Arja, Rolla, Johnson, Bryon D., Verneris, Michael R, Dvorak, Christopher C, Chu, Julia, Cooke, Kenneth R., Talano, Julie-An, Bunin, Nancy, and Cairo, Mitchell S.

Abstract

Background:AlloHSCT induces long-term, cure in CAYA with malignant and non-malignant disorders, including those with primary immunodeficiencies (PID). However, post-transplant complications secondary to immunosuppression and slow or delayed immune reconstitution remains a cause of morbidity and mortality amongst AlloHSCT recipients (Satwani/Cairo, BBMT, 2009). Major complications include refractory viral infections, most importantly adenovirus (ADV), which is secondary to absent or decreased adenovirus specific T-cell immunity which occurs due to delayed immune reconstitution and/or continued immunosuppression (George/Cairo, BJH, 2012). The use of virus-specific (vs) CTLs in immunocompromised patients offer an opportunity to restore temporary virus specific immunity. From the Viral Cytotoxic T-Lymphocyte Consortium (VIRCTLC), we present safety and efficacy results on immune compromised, AlloHSCT, or SOT patients treated with familial ADV specific CTLs.

Published
2023
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27. A Phase II Study to Evaluate the Safety and Efficacy of Defibrotide and Changes in Plasma Biomarkers in Sickle Cell Disease-Related Acute Chest Syndrome (IND 127812)

Author

Schaefer, Edo, Anderson-Crannage, Morgan, Hochberg, Jessica, Kanarfogel, Temima, Ktena, Yiouli Panayiota, Herrick, John, Thatcher, Emily, Shi, Qiuhu, Hochberg, Bruno, Chu, Yaya, Krishnan, Sankaran, Morris, Erin, Fabricatore, Sandra, DuFresne, John, Cooke, Kenneth R., and Cairo, Mitchell S.

Abstract

Background: Acute Chest Syndrome (ACS) is the greatest contributor to the morbidity and mortality of Sickle Cell Disease (SCD) patients with a rate of 12.8 cases per 100 patient-years. The mortality rates of ACS are 4 times higher in adults than in children (Vichinsky et al, Blood, 1997). The root cause of ACS in SCD is related to endothelial dysfunction (Paul et al, European Journal of Hematology 2011). While existing therapies target infection, inflammation, and alveolar hypoxia, they do not address endothelial dysfunction, a major contributor to SCD associated ACS. Defibrotide is a polydisperse mixture of predominantly single stranded oligonucleotides derived from porcine intestinal mucosa. Several pre-clinical studies indicate that defibrotide primarily protects endothelium, particularly in small vessels, and reduces endothelial cell injury (Falanga et al, Leukemia, 2003) (Cairo/Cooke et al, British Journal of Haematology 2020). In a randomized phase III study, a decrease in incidence of SOS/VOD was observed in pediatric hematopoietic stem cell transplantation patients who received prophylactic defibrotide (Corbacioglu et al, Lancet, 2012). We hypothesized that Defibrotide would be safe and well tolerated in children and adolescents with SCD-associated ACS.

Published
2023
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28. A Phase II Study to Evaluate the Safety and Efficacy of Defibrotide in Children with High Risk Kawasaki Disease (IND 127812)

Author

Schaefer, Edo, Shi, Qiuhu, Brandstaedter, Karen, Melchert, Kristina, Patel, Darshan, Morris, Erin, Fabricatore, Sandra, Mintzer, Elizabeth, DuFresne, John, Cooke, Kenneth R., and Cairo, Mitchell S.

Abstract

Background: Kawasaki disease (KD) is the leading cause of acquired heart disease in children in the developed world. It is an acute, generalized vasculitis, particularly in the coronary arteries, that occurs predominantly in infants and young children (Newburger et al, 2016). The acute phase of KD produces diffuse vascular inflammation that is thought to be associated with activation of the IL-1 pathway. Endothelium, located in the innermost layer of the coronary arteries, serves as an interface between the vascular media and circulating inflammatory cells and inflammatory cytokines. The coronary artery endothelium is the first target of the inflammatory attack in the early stages of KD (Qiu et al, Frontiers Cardiovascular Medicine, 2022) (Liu et al, Molecular Cell Biochem, 2021). There are pathologic changes during an acute arteritis with neutrophilic infiltration leading to necrosis in all the vessel walls, although the usual target is the medium sized extra-parenchymal muscular arteries. Current treatment is directed at reducing inflammation with IVIG and aspirin. Usually, this leads to rapid clinical improvement. However, 10-20% of patients are IVIG resistant and require further methods of reducing inflammation (Newburger et al, 2004). These patients that are IVIG resistant, have recrudescent fevers and evidence of ongoing inflammation, with subsequent increased risk for aneurysm formation (Tremoulet et al, 2008). Furthermore, other particular patient groups have been identified as having a higher risk (HR) of coronary artery aneurysms. Boys have a higher incidence of developing KD, but even accounting for that, are at a higher risk for developing coronary artery aneurysms (Belay et al, 2006). Defibrotide is a polydisperse mixture of predominantly single-stranded oligonucleotides (90% single-stranded and 10% double-stranded) derived from porcine intestinal mucosa. Several in vivo studies indicate that defibrotide primarily targets endothelium, particularly in small vessels, and appears to reduce endothelial cell injury and stabilize endothelial function (Falanga et al, Leukemia, 2003) (Cairo et al, BJH, 2020). Defibrotide is currently approved for SOS/VOD, with renal or pulmonary dysfunction post HSCT and has been well studied in infants with SOS post HSCT (Corbacioglu et al, Lancet, 2012). We hypothesized that Defibrotide will be safe and well tolerated in HR KD patients who fail IVIG and may be effective in difficult to treat KD patients, by mitigating ongoing endothelial dysfunction.

Published
2023
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29. Anemia associated with acute infection in children.

Author

Ballin A, Senecky Y, Rubinstein U, Schaefer E, Peri R, Amsel S, Vol M, Amit Y, and Boaz M

Subjects
Acute Disease, Adolescent, Child, Child, Preschool, Cross-Sectional Studies, Erythrocyte Indices, Humans, Anemia, Iron-Deficiency etiology, Infections complications
Abstract

Background: The pathogenesis of anemia associated with acute infection in children has not been well delineated., Objectives: To characterize this type of anemia in children with acute infection, mainly in relation to iron status., Methods: These two cross-sectional studies compared the prevalence and severity of anemia between outpatient febrile children and age-matched non-febrile controls., Results: In part 1 of the study, children with acute infection (n = 58) had a significant decrease in hemoglobin levels compared with 54 non-febrile controls. Mean corpuscular volume (MCV) did not change this association. Moreover, there was no significant difference in MCV, mean cell hemoglobin or red cell distribution width values between the two groups. Regarding part 2, of the 6534 blood counts obtained in community clinics, 229 were defined as "bacterial infection." Chart survey confirmed this diagnosis. White blood cell level was significantly inversely associated with hemoglobin level (r = -0.36, P < 0.0001). Anemia was significantly more prevalent among children with bacterial infection compared to those without: 21.4% vs. 14.1% (P = 0.002). Mean values of iron status parameters were all within normal limits., Conclusions: Acute illness is associated with anemia. The pathogenesis of this anemia does not appear to be associated with disruption of iron metabolism.

Published
2012

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