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3. Correction: Inotuzumab ozogamicin in pediatric patients with relapsed/refractory acute lymphoblastic leukemia

Author

Bhojwani, Deepa, Sposto, Richard, Shah, Nirali N, Rodriguez, Vilmarie, Yuan, Constance, Stetler-Stevenson, Maryalice, O’Brien, Maureen M, McNeer, Jennifer L, Quereshi, Amrana, Cabannes, Aurelie, Schlegel, Paul, Rossig, Claudia, Dalla-Pozza, Luciano, August, Keith, Alexander, Sarah, Bourquin, Jean-Pierre, Zwaan, Michel, Raetz, Elizabeth A, Loh, Mignon L, and Rheingold, Susan R

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Clinical Sciences, Oncology and Carcinogenesis, Cancer, Rare Diseases, Pediatric Cancer, Pediatric, Good Health and Well Being, Immunology, Cardiovascular medicine and haematology, Clinical sciences, Oncology and carcinogenesis
Abstract

We thank the research coordinators and following physicians at pediatric cancer centers for contributing data to this project: Prashant Hiwarkar and Jayashree Motwani, Birmingham Women's and Children's Hospital, UK; Kelly Malone, Children's Hospital of Colorado, USA; Mylene Bassal, Children's Hospital of Eastern Ontario, Canada; Yoav Messinger and Joanna Perkins, Children's Hospital of Minnesota, USA; Van Huynh, Children's Hospital of Orange County, USA; Richard Ho, Children's Hospital at Vanderbilt, USA; Joanne Chuah and Jessa Morales, Children's Hospital at Westmead, Australia; Donald Wells, Dell Children's Hospital, USA; Nicolas Boissel, Hospital Saint-Louis, France; Tannie Huang, Kaiser Permanente, USA; Stacey Marjerrison, McMaster Children's Hospital, Canada; William Carroll and Joanna Pierro, New York University Langone Medical Center, USA; Ajay Vora, Sheffield Children's Hospital, UK; Donna Lancaster, The Royal Marsden Hospital, UK; Lucie Šrámková, University Hospital Motol, Czech Republic; Chatchawin Assanasen, University of Texas Health Science Center, San Antonio, USA; Rupert Handgretinger, University of Tübingen, Germany.

Published
2019

4. Inotuzumab ozogamicin in pediatric patients with relapsed/refractory acute lymphoblastic leukemia

Author

Bhojwani, Deepa, Sposto, Richard, Shah, Nirali N, Rodriguez, Vilmarie, Yuan, Constance, Stetler-Stevenson, Maryalice, O’Brien, Maureen M, McNeer, Jennifer L, Quereshi, Amrana, Cabannes, Aurelie, Schlegel, Paul, Rossig, Claudia, Dalla-Pozza, Luciano, August, Keith, Alexander, Sarah, Bourquin, Jean-Pierre, Zwaan, Michel, Raetz, Elizabeth A, Loh, Mignon L, and Rheingold, Susan R

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Oncology and Carcinogenesis, Pediatric Research Initiative, Childhood Leukemia, Rare Diseases, Stem Cell Research, Cancer, Pediatric, Hematology, Transplantation, Clinical Research, Pediatric Cancer, Adolescent, Adult, Antibodies, Monoclonal, Humanized, Antineoplastic Agents, Child, Child, Preschool, Drug Resistance, Neoplasm, Female, Humans, Inotuzumab Ozogamicin, Male, Neoplasm Recurrence, Local, Precursor B-Cell Lymphoblastic Leukemia-Lymphoma, Prognosis, Remission Induction, Retrospective Studies, Salvage Therapy, Survival Rate, Young Adult, Clinical Sciences, Immunology, Cardiovascular medicine and haematology, Clinical sciences, Oncology and carcinogenesis
Abstract

Although inotuzumab ozogamicin (InO) is recognized as an effective agent in relapsed acute lymphoblastic leukemia (ALL) in adults, data on safety and efficacy in pediatric patients are scarce. We report the use of InO in 51 children with relapsed/refractory ALL treated in the compassionate use program. In this heavily pretreated cohort, complete remission was achieved in 67% of patients with overt marrow disease. The majority (71%) of responders were negative for minimal residual disease. Responses were observed irrespective of cytogenetic subtype or number or type of prior treatment regimens. InO was well-tolerated; grade 3 hepatic transaminitis or hyperbilirubinemia were noted in 6 (12%) and grade 3/4 infections in 11 (22%) patients. No patient developed sinusoidal obstruction syndrome (SOS) during InO therapy; however, 11 of 21 (52%) patients who underwent hematopoietic stem cell transplantation (HSCT) following InO developed SOS. Downregulation of surface CD22 was detected as a possible escape mechanism in three patients who developed a subsequent relapse after InO. We conclude that InO is a well-tolerated, effective therapy for children with relapsed ALL and prospective studies are warranted. Identification of risk factors for developing post-HSCT SOS and strategies to mitigate this risk are ongoing.

Published
2019

7. Clinical Correlates and Treatment Outcomes for Patients With Short Telomere Syndromes

Author

Mangaonkar, Abhishek A., Ferrer, Alejandro, Pinto e Vairo, Filippo, Cousin, Margot A., Kuisle, Ryan J., Klee, Eric W., Kennedy, Cassie C., Peters, Steve G., Scott, J.P., Utz, James P., Baqir, Misbah, Sekiguchi, Hiroshi, Khan, Shakila P., Rodriguez, Vilmarie, Simonetto, Douglas A., Kamath, Patrick S., Abraham, Roshini S., Wylam, Mark E., and Patnaik, Mrinal M.

Published
2018
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10. Thrombotic Microangiopathy Care Pathway: A Consensus Statement for the Mayo Clinic Complement Alternative Pathway-Thrombotic Microangiopathy (CAP-TMA) Disease-Oriented Group

Author

Go, Ronald S., Winters, Jeffrey L., Leung, Nelson, Murray, David L., Willrich, Maria A., Abraham, Roshini S., Amer, Hatem, Hogan, William J., Marshall, Ariela L., Sethi, Sanjeev, Tran, Cheryl L., Chen, Dong, Pruthi, Rajiv K., Ashrani, Aneel A., Fervenza, Fernando C., Cramer, Carl H., II, Hook, C. Christopher, Garovic, Vesna D., Yui, Jennifer C., Botero, Juliana Perez, Rodriguez, Vilmarie, Wolanskyj, Alexandra P., and Thomé, Stephan D.

Published
2016
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12. Abstract 2002: A genome-wide association study identifies novel sepsis risk loci in children with Down syndrome-associated acute lymphoblastic leukemia: A report from the Children’s Oncology Group

Author

Richard, Melissa A., primary, Devidas, Meenakshi, additional, Yang, Wenjian, additional, Woodhouse, John P., additional, Rodriguez, Vilmarie, additional, Hitzler, Johann K., additional, Schore, Reuven J., additional, Angiolillo, Anne L., additional, Burke, Michael J., additional, Salzer, Wanda L., additional, Raetz, Elizabeth A., additional, Loh, Mignon L., additional, Hunger, Stephen P., additional, Yang, Jun J., additional, Lupo, Philip J., additional, and Rabin, Karen R., additional

Published
2022
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14. sj-docx-1-cath-10.1177_10760296221078842 - Supplemental material for Andexanet Alfa Versus Prothrombin Complex Concentrates/Blood Products as Apixaban/Rivaroxaban Reversal Agents: A Survey Among Pediatric Hematologists

Author

Rodriguez, Vilmarie, Stanek, Joseph, Kerlin, Bryce A., and Dunn, Amy L.

Subjects
FOS: Clinical medicine, Cardiology, 111599 Pharmacology and Pharmaceutical Sciences not elsewhere classified
Abstract

Supplemental material, sj-docx-1-cath-10.1177_10760296221078842 for Andexanet Alfa Versus Prothrombin Complex Concentrates/Blood Products as Apixaban/Rivaroxaban Reversal Agents: A Survey Among Pediatric Hematologists by Vilmarie Rodriguez, Joseph Stanek, Bryce A. Kerlin and Amy L. Dunn in Clinical and Applied Thrombosis/Hemostasis

Published
2022
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16. Patients with Down Syndrome and High-Risk B-Acute Lymphoblastic Leukemia Demonstrate Improved Outcomes on a Modified Chemotherapy Regimen: A Report from Children's Oncology Group Study AALL1131

Author

Rodriguez, Vilmarie, Devidas, Meenakshi, Chen, Zhiguo (Bruce), Carroll, Andrew J, Heerema, Nyla A., Borowitz, Michael J., Wood, Brent L., Carroll, William L., Winick, Naomi J, Raetz, Elizabeth A., Loh, Mignon L., Burke, Michael J., Salzer, Wanda L., Hunger, Stephen P., and Rabin, Karen R.

Published
2023
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17. Apixaban Is Effective and Safe in Reducing Venous Thromboembolism (VTE) in Obese Patients with Newly Diagnosed Pediatric Acute Lymphoblastic Leukemia/Lymphoblastic Lymphoma (ALL/LL) a Sub-Study Analysis of the Prevapix-ALL/Children's Oncology Group ACCL1333 Trial

Author

Rodriguez, Vilmarie, O'Brien, Sarah, Lew, Glen, Orgel, Etan, Derr, Kimberly, Ranalli, Mark A., Schultz, Corinna L., Esbenshade, Adam J., Memaj, Arteid, Dyme, Joshua, Favatella, Nicholas, and Mitchell, Lesley

Published
2023
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18. Inhibit Clinical Trials Platform to Prevent and Eradicate Inhibitors: Feasibility Survey of Current Prophylaxis and Immune Tolerance Practices

Author

Ragni, Margaret V., primary, Xavier, Frederico, additional, Seaman, Craig D., additional, Acharya, Suchitra, additional, McGuinn, Catherine E., additional, Werner, Eric J., additional, Lawrence, Courtney Elizabeth, additional, Wheeler, Allison P., additional, Reiss, Ulrike, additional, Ayala, Irmel, additional, Cockrell, Erin, additional, Tarango, Cristina, additional, Dunn, Amy, additional, Kulkarni, Roshni, additional, Ahuja, Sanjay P, additional, Chitlur, Meera B., additional, Pipe, Steven W., additional, Malec, Lynn M, additional, Rodriguez, Vilmarie, additional, Crary, Shelley, additional, Brown, Deborah, additional, Diaz, Rosa, additional, Velez, Maria, additional, Leissinger, Cindy A., additional, Carpenter, Shannon L, additional, Knoll, Christine M., additional, Wang, Michael, additional, Young, Guy, additional, Thornburg, Courtney D, additional, Lasky, Joseph L, additional, Lucas, Tiffany Lin, additional, Hwang, Nina, additional, Vehec, Deborah, additional, Ivanco, Dana, additional, Haller, Tamara L., additional, Bertolet, Marnie, additional, and Brooks, Maria M, additional

Published
2020
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24. Low-Dose Total Body Irradiation (TBI) Use As Part of Pre-Transplant Conditioning Regimen Is Associated with Worse Outcomes in Patients with Severe Aplastic Anemia (SAA) Treated with Allogeneic Hematopoietic Stem Cell Transplantation (HCT)

Author

Mangaonkar, Abhishek, primary, Nadiminti, Kalyan, additional, Langer, Kimberly J., additional, Buradkar, Ajinkya, additional, Matin, Aasiya, additional, Hefazi, Mehrdad, additional, Alkhateeb, Hassan B., additional, Shah, Mithun V., additional, Khan, Shakila P., additional, Rodriguez, Vilmarie, additional, Hogan, William J., additional, Litzow, Mark R., additional, and Patnaik, Mrinal M., additional

Published
2020
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25. Hemoglobin Sunshine Seth: A Case Report of Low-Oxygen-Affinity Hemoglobinopathy

Author

Heidenreich, Leah S., Oliveira, Jennifer L., Holmberg, Peter J., and Rodriguez, Vilmarie

Subjects
Article Subject
Abstract

Pulse oximetry is routinely used in the newborn nursery for clinical monitoring and to detect critical congenital heart disease. The differential diagnoses for reduced peripheral oxygen saturation in an infant include congenital heart disease, respiratory distress syndrome, transient tachypnea of the newborn, persistent pulmonary hypertension of the newborn, meconium aspiration syndrome, pneumonia, pneumothorax, and sepsis. The diagnostic evaluation for neonatal hypoxemia can be invasive and expensive. When this evaluation is unrevealing, other interventions may be tried without clear benefit to the patient, including, but not limited to, supplemental oxygen. Therefore, it is important to consider alternative, albeit rare, diagnoses, including hemoglobinopathies with abnormal oxygen binding properties. Mutations in the structure of alpha- and beta-globin chains can alter the affinity of hemoglobin for oxygen, and changes in oxygen affinity may result in changes in the oxygen saturation detected by pulse oximetry. These changes may or may not be of clinical significance. This case report describes Hemoglobin Sunshine Seth, a rare low-oxygen-affinity hemoglobin variant presenting as reduced peripheral oxygen saturation in an otherwise well-appearing infant male.

Published
2020
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27. Clinical utility of fluorescence in situ hybridization‐based diagnosis of BCR‐ABL1 like ( P hiladelphia chromosome like) B ‐acute lymphoblastic leukemia

Author

Anagnostou, Theodora, primary, Knudson, Ryan A., additional, Pearce, Kathryn E., additional, Meyer, Reid G., additional, Pitel, Beth A., additional, Peterson, Jess F., additional, Baughn, Linda B., additional, Reichard, Kaaren K., additional, Ketterling, Rhett P., additional, Kloft‐Nelson, Sara M., additional, Knutson, Darlene L., additional, Khan, Shakila P., additional, Gangat, Naseema, additional, Litzow, Mark R., additional, Hogan, William J., additional, Wolanskyj, Alexandra, additional, Al‐Kali, Aref, additional, Begna, Kebede H., additional, Elliott, Michelle, additional, Pardanani, Animesh, additional, Foran, James, additional, Shah, Mithun, additional, Tefferi, Ayalew, additional, Alkhateeb, Hassan, additional, Halling, Kevin, additional, Rodriguez, Vilmarie, additional, Greipp, Patricia T., additional, and Patnaik, Mrinal M., additional

Published
2020
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28. Diffuse large B-cell lymphoma in an adolescent female presenting with Epstein-Barr virus-driven hemophagocytic lymphohistiocytosis: a case report

Author

Altaf Sadaf, Atreaga Grace M, Joshi Avni Y, and Rodriguez Vilmarie

Subjects
Medicine
Abstract

Abstract Introduction Hemophagocytic lymphohistiocytosis is characterized by multisystem inflammation, resulting from prolonged and intense activation of macrophages, histiocytes and CD8+ T-cells. Due to its variable presentation and non-specific findings, timely diagnosis can be challenging. This condition has been associated with malignancies, most commonly with lymphomas and leukemias of T-cell lineage. This case report represents the less commonly associated B-cell lymphomas. We also highlight the difficulties in managing hemophagocytosis with an evolving malignancy. This case report will add to the increasing literature on the diagnosis, complications and management of this complex disorder. Case presentation A 15-year-old Caucasian girl, previously diagnosed with Crohn’s disease and treated with 6-mercaptopurine, developed Epstein-Barr virus infection-driven hemophagocytic lymphohistiocytosis. The diagnosis was challenging due to her critical illness and the lack of enough features to fulfill diagnostic criteria at presentation (moderately elevated ferritin, normal coagulation profiles and normal triglycerides). While receiving therapy for hemophagocytic lymphohistiocytosis, she developed bulky cervical lymphadenopathy and was diagnosed with diffuse large B-cell lymphoma. Therapy for lymphoma was initiated and she tolerated the therapy well. Conclusion Hemophagocytic lymphohistiocytosis is a rare disorder, but potentially lethal if not diagnosed and treated in a timely manner. Our case highlights the importance of considering this diagnosis in critically ill patients who may not initially fulfill formal diagnostic criteria. In patients diagnosed with hemophagocytic lymphohistiocytosis, occult malignancies should be aggressively ruled out as they can manifest prior to the hemophagocytic lymphohistiocytosis diagnosis or appear during the treatment phase. An accurate diagnosis is also important because management of Epstein-Barr virus-driven hemophagocytic lymphohistiocytosis and Epstein-Barr virus-driven lymphoma differs due to the difference in pathophysiology and the involvement of different immune cell lines.

Published
2012
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30. Clinical Utility of Telomere Length-Directed Genomic Assessment in Patients with Short Telomere Syndromes

Author

Mangaonkar, Abhishek A, primary, Ferrer, Alejandro, additional, Pinto E Vairo, Filippo, additional, Cousin, Margot, additional, Kuisle, Ryan, additional, Kennedy, Cassie C, additional, Coltro, Giacomo, additional, Gangat, Naseema, additional, Hogan, William J, additional, Litzow, Mark, additional, Peters, Steve, additional, Klee, Eric W., additional, Scott, JP, additional, Utz, James P, additional, Baqir, Misbah, additional, Carmona Porquera, Eva, additional, Sekiguchi, Hiroshi, additional, Khan, Shakila P, additional, Rodriguez, Vilmarie, additional, Simonetto, Douglas A, additional, Kamath, Patrick S, additional, Joshi, Avni Y, additional, Wylam, Mark E, additional, and Patnaik, Mrinal M, additional

Published
2019
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31. Survival Outcomes Following Allogeneic Stem Cell Transplantation for Inherited Bone Marrow Failure and Myeloid Germline Predisposition Syndromes

Author

Nadiminti, Kalyan, primary, Mangaonkar, Abhishek A, additional, Langer, Kimberly J., additional, Khan, Shakila P, additional, Rodriguez, Vilmarie, additional, Gangat, Naseema, additional, Elliott, Michelle A, additional, Alkhateeb, Hassan B., additional, Shah, Mithun Vinod, additional, Litzow, Mark, additional, Hogan, William J, additional, Kenderian, Saad S., additional, and Patnaik, Mrinal M, additional

Published
2019
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32. Spectrum of Abnormalities and Clonal Transformation in Germline RUNX1 Familial Platelet Disorder and a Comparative Analysis with Somatic RUNX1 Mutations in Myeloid Neoplasms

Author

Difilippo, Emma, primary, Coltro, Giacomo, additional, Carr, Ryan M., additional, Mangaonkar, Abhishek A, additional, Binder, Moritz, additional, Khan, Shakila P, additional, Rodriguez, Vilmarie, additional, Gangat, Naseema, additional, Wolanskyj, Alexandra, additional, Pruthi, Rajiv K., additional, Chen, Dong, additional, He, Rong, additional, Viswanatha, David S, additional, Lasho, Terra, additional, Finke, Christy, additional, Tefferi, Ayalew, additional, Pardanani, Animesh, additional, Litzow, Mark, additional, and Patnaik, Mrinal M, additional

Published
2019
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33. Empiric treatment of protracted idiopathic purpura fulminans in an infant: a case report and review of the literature

Author

Davis Dawn M, Broomall Eileen M, Pundi Kavitha N, Macheret Fima, Rodriguez Vilmarie, and Brands Chad K

Subjects
Medicine
Abstract

Abstract Introduction Idiopathic purpura fulminans is a cutaneous thrombotic disorder usually caused by autoimmune-mediated protein C or S deficiency. This disorder typically presents with purpura and petechiae that eventually slowly or rapidly coalesce into extensive, necrotic eschars on the extremities. We present the first known case of idiopathic purpura fulminans consistent with prior clinical presentations in the setting of a prothrombotic genetic mutation, but without hallmark biochemical evidence of protein C or protein S deficiency. Another novel feature of our patient's presentation is that discontinuation of anti-coagulation has invariably led to recurrence and formation of new lesions, which is unexpected in idiopathic purpura fulminans because clearance of autoimmune factors should be followed by restoration of anti-coagulant function. Although this disease is rare, infants with suspected idiopathic purpura fulminans should be rapidly diagnosed and immediately anti-coagulated to prevent adverse catastrophic outcomes such as amputation and significant developmental delay. Case presentation A six-month-old Caucasian boy was brought to our pediatric hospital service with a low-grade fever and subacute, symmetric, serpiginous, stellate, necrotic eschars on his forearms, legs and feet that eventually spread non-contiguously to his toes, thighs and buttocks. In contrast to his impressive clinical presentation, his serologic evaluation was normal, and he was not responsive to corticosteroids and antibiotics. Full-thickness skin biopsies revealed dermal vessel thrombosis, leading to a diagnosis of idiopathic purpura fulminans and successful treatment with low-molecular-weight heparin, which was transitioned to warfarin. Long-term management has included chronic anti-coagulation because of recurrence of lesions with discontinuation of treatment. Conclusion In infants with necrotic eschars, it is important to first consider infectious, inflammatory and hematologic etiologies. In the absence of etiology for protracted idiopathic purpura fulminans, management should include tissue biopsy, in which thrombotic findings warrant a trial of empiric anti-coagulation. Some infants, including our patient, may need long-term anti-coagulation, especially when the underlying etiology of coagulation remains unidentified and symptoms recur when treatment is halted. Given that our patient still requires anti-coagulation, he may have a yet to be identified autoimmune-mediated mechanism for his truly idiopathic case of protracted purpura fulminans.

Published
2011
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34. Inotuzumab ozogamicin in pediatric patients with relapsed/refractory acute lymphoblastic leukemia

Author

Bhojwani, Deepa; https://orcid.org/0000-0002-7559-7927, Sposto, Richard, Shah, Nirali N, Rodriguez, Vilmarie, Yuan, Constance, Stetler-Stevenson, Maryalice, O'Brien, Maureen M, McNeer, Jennifer L, Quereshi, Amrana, Cabannes, Aurelie, Schlegel, Paul, Rossig, Claudia, Dalla-Pozza, Luciano, August, Keith, Alexander, Sarah, Bourquin, Jean-Pierre, Zwaan, Michel, Raetz, Elizabeth A, Loh, Mignon L, Rheingold, Susan R, Bhojwani, Deepa; https://orcid.org/0000-0002-7559-7927, Sposto, Richard, Shah, Nirali N, Rodriguez, Vilmarie, Yuan, Constance, Stetler-Stevenson, Maryalice, O'Brien, Maureen M, McNeer, Jennifer L, Quereshi, Amrana, Cabannes, Aurelie, Schlegel, Paul, Rossig, Claudia, Dalla-Pozza, Luciano, August, Keith, Alexander, Sarah, Bourquin, Jean-Pierre, Zwaan, Michel, Raetz, Elizabeth A, Loh, Mignon L, and Rheingold, Susan R

Abstract

Although inotuzumab ozogamicin (InO) is recognized as an effective agent in relapsed acute lymphoblastic leukemia (ALL) in adults, data on safety and efficacy in pediatric patients are scarce. We report the use of InO in 51 children with relapsed/refractory ALL treated in the compassionate use program. In this heavily pretreated cohort, complete remission was achieved in 67% of patients with overt marrow disease. The majority (71%) of responders were negative for minimal residual disease. Responses were observed irrespective of cytogenetic subtype or number or type of prior treatment regimens. InO was well-tolerated; grade 3 hepatic transaminitis or hyperbilirubinemia were noted in 6 (12%) and grade 3/4 infections in 11 (22%) patients. No patient developed sinusoidal obstruction syndrome (SOS) during InO therapy; however, 11 of 21 (52%) patients who underwent hematopoietic stem cell transplantation (HSCT) following InO developed SOS. Downregulation of surface CD22 was detected as a possible escape mechanism in three patients who developed a subsequent relapse after InO. We conclude that InO is a well-tolerated, effective therapy for children with relapsed ALL and prospective studies are warranted. Identification of risk factors for developing post-HSCT SOS and strategies to mitigate this risk are ongoing.

Published
2019

35. Correction: Inotuzumab ozogamicin in pediatric patients with relapsed/refractory acute lymphoblastic leukemia

Author

Bhojwani, Deepa; https://orcid.org/0000-0002-7559-7927, Sposto, Richard, Shah, Nirali N, Rodriguez, Vilmarie, Yuan, Constance, Stetler-Stevenson, Maryalice, O'Brien, Maureen M, McNeer, Jennifer L, Quereshi, Amrana, Cabannes, Aurelie, Schlegel, Paul, Rossig, Claudia, Dalla-Pozza, Luciano, August, Keith, Alexander, Sarah, Bourquin, Jean-Pierre, Zwaan, Michel, Raetz, Elizabeth A, Loh, Mignon L, Rheingold, Susan R, Bhojwani, Deepa; https://orcid.org/0000-0002-7559-7927, Sposto, Richard, Shah, Nirali N, Rodriguez, Vilmarie, Yuan, Constance, Stetler-Stevenson, Maryalice, O'Brien, Maureen M, McNeer, Jennifer L, Quereshi, Amrana, Cabannes, Aurelie, Schlegel, Paul, Rossig, Claudia, Dalla-Pozza, Luciano, August, Keith, Alexander, Sarah, Bourquin, Jean-Pierre, Zwaan, Michel, Raetz, Elizabeth A, Loh, Mignon L, and Rheingold, Susan R

Abstract

We thank the research coordinators and following physicians at pediatric cancer centers for contributing data to this project: Prashant Hiwarkar and Jayashree Motwani, Birmingham Women's and Children's Hospital, UK; Kelly Malone, Children's Hospital of Colorado, USA; Mylene Bassal, Children's Hospital of Eastern Ontario, Canada; Yoav Messinger and Joanna Perkins, Children's Hospital of Minnesota, USA; Van Huynh, Children's Hospital of Orange County, USA; Richard Ho, Children's Hospital at Vanderbilt, USA; Joanne Chuah and Jessa Morales, Children's Hospital at Westmead, Australia; Donald Wells, Dell Children's Hospital, USA; Nicolas Boissel, Hospital Saint-Louis, France; Tannie Huang, Kaiser Permanente, USA; Stacey Marjerrison, McMaster Children's Hospital, Canada; William Carroll and Joanna Pierro, New York University Langone Medical Center, USA; Ajay Vora, Sheffield Children's Hospital, UK; Donna Lancaster, The Royal Marsden Hospital, UK; Lucie Šrámková, University Hospital Motol, Czech Republic; Chatchawin Assanasen, University of Texas Health Science Center, San Antonio, USA; Rupert Handgretinger, University of Tübingen, Germany.

Published
2019

36. How We Approach Central Venous Catheter Safety: A Multidisciplinary Perspective.

Author

Levy, Emily R, Hutchins, Kathryn A, Schears, Gregory J, Rodriguez, Vilmarie, and Huskins, W Charles

Subjects
ANTI-infective agents, BLOODBORNE infections, CATHETERIZATION complications, CHLORHEXIDINE, DRUG utilization, HEALTH care teams, HEPARIN, INTERPROFESSIONAL relations, PATIENT safety, PEDIATRICS, PHYSIOLOGIC salines, PATIENT-centered care, CENTRAL venous catheterization, CENTRAL venous catheters, ANTIBIOTIC prophylaxis, CATHETER-related infections, ANTIMICROBIAL bandages, CATHETER-related thrombosis
Published
2020
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37. Inotuzumab ozogamicin in pediatric patients with relapsed/refractory acute lymphoblastic leukemia

Author

Bhojwani, Deepa, primary, Sposto, Richard, additional, Shah, Nirali N., additional, Rodriguez, Vilmarie, additional, Yuan, Constance, additional, Stetler-Stevenson, Maryalice, additional, O’Brien, Maureen M., additional, McNeer, Jennifer L., additional, Quereshi, Amrana, additional, Cabannes, Aurelie, additional, Schlegel, Paul, additional, Rossig, Claudia, additional, Dalla-Pozza, Luciano, additional, August, Keith, additional, Alexander, Sarah, additional, Bourquin, Jean-Pierre, additional, Zwaan, Michel, additional, Raetz, Elizabeth A., additional, Loh, Mignon L., additional, and Rheingold, Susan R., additional

Published
2018
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38. Recurrent heteroplasmy for the MT-ATP6 p.Ser148Asn (m.8969G>A) mutation in patients with syndromic congenital sideroblastic anemia of variable clinical severity

Author

Berhe, Simon, primary, Heeney, Matthew M., additional, Campagna, Dean R., additional, Thompson, John F., additional, White, Eric J., additional, Ross, Tristen, additional, Peake, Roy W. A., additional, Hanrahan, Jeffery D., additional, Rodriguez, Vilmarie, additional, Renaud, Deborah L., additional, Patnaik, Mrinal S., additional, Chang, Eugenia, additional, Bottomley, Sylvia S., additional, and Fleming, Mark D., additional

Published
2018
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41. Autoimmune Lymphoproliferative Syndrome: A Rare Cause of Disappearing HDL Syndrome

Author

Sriram, Swetha, Joshi, Avni Y., Rodriguez, Vilmarie, and Kumar, Seema

Subjects
Article Subject, lipids (amino acids, peptides, and proteins)
Abstract

The term disappearing HDL syndrome refers to development of severe high density lipoprotein cholesterol (HDL-C) deficiency in noncritically ill patients with previously normal HDL-C and triglyceride levels. Autoimmune lymphoproliferative syndrome (ALPS) is a disorder of the immune system due to an inability to regulate lymphocyte homeostasis resulting in lymphadenopathy and hepatosplenomegaly. We describe a 17-year-old boy who was evaluated in the lipid clinic for history of undetectable or low HDL-C and low density lipoprotein cholesterol (LDL-C) levels. Past medical history was significant for ALPS IA diagnosed at 10 years of age when he presented with bilateral cervical adenopathy. He was known to have a missense mutation in one allele of the FAS protein extracellular domain consistent with ALPS type 1A. HDL-C and LDL-C levels had been undetectable on multiple occasions, though lipids had not been measured prior to the diagnosis of ALPS. He had been receiving sirolimus for immunosuppression. The HDL-C and LDL-C levels correlated with disease activity and improved to normal levels during times when the activity of ALPS was controlled. This case highlights the importance of considering ALPS as a cause of low HDL-C and LDL-C levels in a child with evidence of lymphoproliferation.

Published
2016
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42. Cell-Based Therapy for Myocardial Dysfunction After Fontan Operation in Hypoplastic Left Heart Syndrome

Author

Qureshi, Muhammad Y., primary, Cabalka, Allison K., additional, Khan, Shakila P., additional, Hagler, Donald J., additional, Haile, Dawit T., additional, Cannon, Bryan C., additional, Olson, Timothy M., additional, Cantero-Peral, Susana, additional, Dietz, Allan B., additional, Radel, Darcie J., additional, Taggart, Nathan W., additional, Kelle, Angela M., additional, Rodriguez, Vilmarie, additional, Dearani, Joseph A., additional, O’Leary, Patrick W., additional, Nelson, Timothy J., additional, Cavanaugh, Karen M., additional, Miller, Jennifer M., additional, and Miller, Karen S., additional

Published
2017
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46. Clinical Spectrum of Germline Mutations with Predisposition to Myeloid Neoplasms- 2016 World Health Organization Classification Update

Author

Ho, Thanh, primary, Perez Botero, Juliana, additional, Hogan, William J, additional, Kenderian, Saad S, additional, Gangat, Naseema, additional, Tefferi, Ayalew, additional, Abraham, Roshini S, additional, Nguyen, Phuong L., additional, Oliveira, Jennifer L, additional, He, Rong, additional, Chen, Dong, additional, Viswanatha, David, additional, Rodriguez, Vilmarie, additional, Khan, Shakila, additional, and Patnaik, Mrinal M, additional

Published
2016
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