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Pediatric Bone marrow transplantation

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https://www.readbyqxmd.com/read/29207878/cd20-cd19-bispecific-car-t-cells-for-the-treatment-of-b-cell-malignancies
#1
Alexandra Martyniszyn, Ann-Christin Krahl, Maya C André, Andreas A Hombach, Hinrich Abken
The treatment of leukemia/lymphoma by chimeric antigen receptor (CAR) redirected T cells with specificity for CD19 induced complete remissions in the majority of patients with a realistic hope for cure. However, recent follow-up data revealed a substantial risk of relapse through leukemic cells which lack the CAR targeted antigen. In this situation a bispecific CAR with binding domains for CD19 and CD20 is aimed at recognizing also leukemic cells with only one cognate antigen. The anti-CD20-CD19 bispecific CAR induced a full T cell response upon engagement of CD19 or CD20 on target cells showing a true "OR" gate recognition in redirecting T cell activation...
December 5, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/29198679/treatment-of-a-pediatric-case-of-severe-hemorrhagic-cystitis-case-report-and-review-of-literature
#2
L Yang, Y Zha, J Feng, H Dong, C Zong, X Lei, N Liang, X Wang, G Gao, X Bai
Hemorrhagic cystitis is one of the complications of allogeneic hematopoietic stem cell transplantation. Treatment of hemorrhagic cystitis is difficult, especially in pediatric patients. A pediatric case of severe hemorrhagic cystitis after hematopoietic stem cell transplantation was treated in our hospital with arterial embolization combined with corticosteroid therapy because the conventional therapy was invalid for him. After the treatment, hemorrhagic cystitis was cured. During follow-up, the patient was in stable condition, with normal urine, blood cells returned to normal, bone marrow was in complete remission state, and disease-free survival for more than 8 months...
December 2017: Transplantation Proceedings
https://www.readbyqxmd.com/read/29189423/the-boy-in-the-bubble-and-the-baby-with-the-berlin-heart-the-dangers-of-bridge-to-decision-in-pediatric-mechanical-circulatory-support
#3
Seth A Hollander, Ethan J Hollander
On the day of his birth in 1971, David Vetter was "temporarily" placed in a sterile isolator to wait for a bone marrow donor who would cure his Severe Combined Immunodeficiency Syndrome. After enduring 12 years in isolation, David, now known to the world as "The Boy in the Bubble", received a bone marrow transplant from his unmatched sister and died 4 months later. Like Severe Combined Immunodeficiency Syndrome, pediatric heart failure is a rare and life-threatening condition for which organ transplantation is often the only option for survival...
November 14, 2017: ASAIO Journal: a Peer-reviewed Journal of the American Society for Artificial Internal Organs
https://www.readbyqxmd.com/read/29178692/-management-of-vaccination-in-children-receiving-an-allogeneic-marrow-transplant
#4
S Blumental, A Ferster, N Azzi, P Lepage
Over the last decades, significant advances in the diagnosis and therapeutics have considerably improved success rate from bone marrow transplant in patients suffering from otherwise life-threatening diseases, allowing now for prolonged survival and better quality of life after an allograft. However, infectious diseases remain one of the most serious complication in this population, hence associated with a high morbidity and mortality. Prevention, in particular through vaccination, constitutes a cornerstone of the management of immunocompromised hosts, since this procedure aims to protect them once back to life in community after long periods of hospitalization...
2017: Revue Médicale de Bruxelles
https://www.readbyqxmd.com/read/29173833/effect-of-parental-depression-level-on-children-s-quality-of-life-after-haematopoietic-stem-cell-transplantation
#5
Zeynep Kisecik Sengul, Naime Altay
PURPOSE: This descriptive cross-sectional study aimed to determine the impact of parental depression level on children's quality of life after haematopoietic stem cell transplantation (HSCT). METHOD: This study was conducted in a bone marrow transplantation unit of a children's hospital in Ankara, Turkey. The research sample consisted of 82 parents. Children aged 2-18 years and monitored in polyclinics 100 days after HSCT were included in the study. A sociodemographic data form, parent forms of the Pediatric Quality of Life Inventory, and the Beck Depression Inventory (BDI) were used...
December 2017: European Journal of Oncology Nursing: the Official Journal of European Oncology Nursing Society
https://www.readbyqxmd.com/read/29169651/-preservation-congelation-of-hematopoietic-stem-cell-grafts-in-a-pediatric-context-guidelines-from-the-francophone-society-of-bone-marrow-transplantation-and-cellular-therapy-sfgm-tc
#6
John De Vos, Eva de Berranger, Charlotte Jubert, Cécile Pochon, Catherine Letellier, Valérie Mialou, Anne Sirvent, Ibrahim Yakoub-Agha, Jean-Hugues Dalle
The Francophone Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC) organized the 7th allogeneic hematopoietic stem cell transplantation clinical practices harmonization workshop series in September 2016 in Lille, France. The objective of our workshop is to provide a discussion on the conservation and congelation of hematopoietic stem cells in a pediatric setting as well as our recommendations for this technique.
November 20, 2017: Bulletin du Cancer
https://www.readbyqxmd.com/read/29162395/automated-red-blood-cell-depletion-in-abo-incompatible-grafts-in-the-pediatric-setting
#7
REVIEW
Claudia Del Fante, Luigia Scudeller, Santina Recupero, Gianluca Viarengo, Stella Boghen, Antonella Gurrado, Marco Zecca, Jerard Seghatchian, Cesare Perotti
Bone marrow ABO incompatible transplantations require graft manipulation prior to infusion to avoid potentially lethal side effects. We analyzed the influence of pre-manipulation factors (temperature at arrival, transit time, time of storage at 4°C until processing and total time from collection to red blood cell depletion) on the graft quality of 21 red blood cell depletion procedures in ABO incompatible pediatric transplants. Bone marrow collections were processed using the Spectra Optia(®) (Terumo BCT) automated device...
November 14, 2017: Transfusion and Apheresis Science
https://www.readbyqxmd.com/read/29155316/influence-of-age-on-acute-and-chronic-gvhd-in-children-receiving-hla-identical-sibling-bmt-for-acute-leukemia-implications-for-prophylaxis
#8
Muna Qayed, Tao Wang, Michael T Hemmer, Stephen Spellman, Mukta Arora, Daniel Couriel, Amin Alousi, Joseph Pidala, Hisham Abdel-Azim, Mahmoud Aljuf, Mouhab Ayas, Menachem Bitan, Mitchell Cairo, Sung Won Choi, Christopher Dandoy, David Delgado, Robert Peter Gale, Gregory Hale, Haydar Frangoul, Rammurti T Kamble, Mohamed Kharfan-Dabaja, Leslie Lehman, John Levine, Margaret MacMillan, David I Marks, Taiga Nishihori, Richard F Olsson, Peiman Hematti, Olov Ringden, Ayman Saad, Prakash Satwani, Bipin N Savani, Kirk R Schultz, Sachiko Seo, Shalini Shenoy, Edmund K Waller, Lolie Yu, Mary M Horowitz, John Horan
Relapse remains the major cause of mortality post hematopoietic cell transplantation (HCT) for pediatric acute leukemia. Previous research suggests that reducing the intensity of calcineurin inhibitor based graft versus host disease (GVHD) prophylaxis may be an effective strategy in abrogating the risk of relapse in pediatric patients undergoing matched sibling donor (MSD) HCT. We reasoned that benefits of this strategy could be maximized by selectively applying it to those patients least likely to develop GVHD...
November 16, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/29153311/two-versus-three-day-upfront-use-of-granulocyte-colony-stimulating-factor-in-healthy-bone-marrow-donors-for-pediatric-bone-marrow-transplantation
#9
REVIEW
Tekin Aksu, İkbal Ok Bozkaya, Sibel Akpınar Tekgündüz, Mehtap Olcar Kanbur, Yasin Köksal, Meltem Özgüner, Namık Yaşar Özbek
In order to decrease donors' exposure to granulocyte-colony stimulating factor (G-CSF), we compared the effect of two versus three days of G-CSF priming on CD34+ yield in bone marrow (BM) harvest. Although the number of BM-CD34+ cells was higher in 3day G-CSF priming, we achieved the same number of CD34+ cells per recipient's weight in 2day G-CSF priming group, too. In addition, the number of total nucleated cells (TNC) harvested from BM were similar with two or three day regimen. But mononuclear cells (MNC) of the BM graft was higher in the 3day G-CSF priming group...
November 8, 2017: Transfusion and Apheresis Science
https://www.readbyqxmd.com/read/29147589/%C3%AE-%C3%AE-t-cell-acute-lymphoblastic-leukemia-lymphoma-discussion-of-two-pediatric-cases-and-its-distinction-from-other-mature-%C3%AE-%C3%AE-t-cell-malignancies
#10
Eric X Wei, Vasiliki Leventaki, John K Choi, Susana C Raimondi, Elizabeth M Azzato, Sheila A Shurtleff, Menchu G Ong, Diana M Veillon, James D Cotelingam, Rodney E Shackelford
Gamma delta (γδ) T-cell antigen receptor (TCR) expression and its related T-cell differentiation are not commonly reported in T-cell acute lymphoblastic leukemia/lymphoma (T-ALL). Here we report two pediatric T-ALL cases and present their clinical features, histology, immunophenotypes, cytogenetics, and molecular diagnostic findings. The first patient is a two-year-old girl with leukocytosis, circulating lymphoblasts, and a cryptic insertion of a short-arm segment at 10p12 into the long-arm segment of 11q23 resulting in an MLL and AF10 fusion transcript, which may be the first reported in γδ T-ALL...
2017: Case Reports in Hematology
https://www.readbyqxmd.com/read/29143887/diagnosis-and-treatment-of-aplastic-anemia
#11
REVIEW
Scott A Peslak, Timothy Olson, Daria V Babushok
Acquired aplastic anemia (AA) is a rare, life-threatening bone marrow failure (BMF) disorder that affects patients of all ages and is caused by lymphocyte destruction of early hematopoietic cells. Diagnosis of AA requires a comprehensive approach with prompt evaluation for inherited and secondary causes of bone marrow aplasia, while providing aggressive supportive care. The choice of frontline therapy is determined by a number of factors including AA severity, age of the patient, donor availability, and access to optimal therapies...
November 16, 2017: Current Treatment Options in Oncology
https://www.readbyqxmd.com/read/29116687/erythropoietic-protoporphyria-in-an-adult-with-sequential-liver-and-hematopoietic-stem-cell-transplantation-a-case-report
#12
Annika L Windon, Rashmi Tondon, Nathan Singh, Samir Abu-Gazala, David L Porter, J Eric Russell, Colleen Cook, Elaine Lander, Georgeine Smith, Kim M Olthoff, Abraham Shaked, Maarouf Hoteit, Emma E Furth, Marina Serper
Erythropoieitic protoporphyria (EPP) is a rare inherited disorder of the heme biosynthesis pathway resulting in the accumulation of protoporphyrins in the blood, erythrocytes, and other tissues. Because of a gene mutation in the FECH gene, ferrochelatase, the enzyme involved in the final step of heme synthesis, is deficient in these patients. While the major symptom of this disorder is photosensitivity, rarely, it can cause progressive liver disease requiring liver transplantation (LT). However, LT is not curative and only bone marrow transplantation (BMT) can correct the underlying enzymatic defect...
November 8, 2017: American Journal of Transplantation
https://www.readbyqxmd.com/read/29115715/infusion-hemolysis-after-pediatric-major-abo-mismatched-bone-marrow-transplant-comparison-of-two-red-blood-cell-depletion-techniques
#13
Robert Sheppard Nickel, Muna Qayed, Diana Worthington-White, Sean R Stowell, Kuang-Yueh Chiang
BACKGROUND: During major ABO-mismatched bone marrow transplant (BMT), the infusion of incompatible red blood cells (RBCs) that are present in the bone marrow graft can cause adverse events from hemolysis. RBC depletion of the bone marrow graft can decrease this risk, but the optimal method to prevent hemolysis is unclear. PROCEDURE: We conducted a retrospective cohort study of patients who underwent major ABO-mismatched BMT at a pediatric center and had RBC depletion with either hydroxyethyl starch (HES) sedimentation or Ficoll density gradient separation...
November 8, 2017: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/29099537/use-of-peripherally-inserted-central-venous-catheters-piccs-in-children-receiving-autologous-or-allogeneic-stem-cell-transplantation
#14
Stefano Benvenuti, Rosanna Ceresoli, Giovanni Boroni, Filippo Parolini, Fulvio Porta, Daniele Alberti
INTRODUCTION: The aim of our study was to present our experience with the use of peripherally inserted central catheters (PICCs) in pediatric patients receiving autologous or allogenic blood stem-cell transplantation. The insertion of the device in older children does not require general anesthesia and does not require a surgical procedure. METHODS: From January 2014 to January 2017, 13 PICCs were inserted as a central venous device in 11 pediatric patients submitted to 14 autologous or allogeneic stem-cell transplantation, at the Bone Marrow Transplant Unit of the Children's Hospital of Brescia...
October 31, 2017: Journal of Vascular Access
https://www.readbyqxmd.com/read/29050112/-clinical-pathologic-characteristics-and-treatment-outcomes-of-19-relapsed-pediatric-b-cell-lymphoma
#15
S Huang, L Jin, J Yang, Y L Duan, M Zhang, C J Zhou, X L Ma, Y H Zhang
Objective: To review the clinical-pathology characteristics of 19 relapsed pediatric mature B cell lymphoma and to find the risk factors for recurrence and the feasible treatment after relapse. Method: Data of 212 pediatric B cell lymphomas cases in Beijing Children's Hospital from January 2006 to June 2015 were collected retrospectively. All the patients were treated according to the B cell lymphoma regimen of Beijing Children's Hospital. During the study period, 19 of 212 cases were relapsed; the clinio-pathological characteristics of relapsed patients before treatment and after relapse were analyzed retrospectively, the treatment outcomes after relapse were summarized and the patients were followed-up...
October 2, 2017: Zhonghua Er Ke za Zhi. Chinese Journal of Pediatrics
https://www.readbyqxmd.com/read/29050111/-a-long-term-follow-up-report-of-pediatric-relapsed-wilms-tumor-after-retreatment
#16
T Y Wang, C Pan, Y J Gao, W T Hu, Q D Ye, M Zhou, J Y Tang
Objective: To investigate the long-term efficacy and prognostic factors of pediatric relapsed Wilms tumor (WT) after retreatment. Method: Sixteen children in Shanghai Children's Medical Center with relapsed Wilms tumor were enrolled consecutively in this study between April 2006 and June 2016. All patients were diagnosed according to pathology, imaging and medical and surgical oncologist's assistance. Relapse treatment included surgical excision, chemotherapy and selective radiation therapy. The clinical features, long-term outcomes and prognostic factors of patients were analyzed retrospectively...
October 2, 2017: Zhonghua Er Ke za Zhi. Chinese Journal of Pediatrics
https://www.readbyqxmd.com/read/29021921/post-transplant-lymphoproliferative-disorder-ptld-single-institutional-experience-of-141-patients
#17
Rohit Bishnoi, Ravneet Bajwa, Aaron J Franke, William Paul Skelton, Yu Wang, Niraj M Patel, William Birdsall Slayton, Fei Zou, Nam H Dang
BACKGROUND: Post-transplant lymphoproliferative disorder is a well-recognized but rare complication of hematopoietic stem cell and solid organ transplant. Due to rarity of this disease, retrospective studies from major transplant centers has been the main source to provide treatment guidelines, which are still in evolution. The sample size of this study is among one of the largest study on PTLD till date reported throughout the world. METHODS: This study was performed at University of Florida which is one of the largest transplant center in South East United States...
2017: Experimental Hematology & Oncology
https://www.readbyqxmd.com/read/28985203/cell-therapy-for-diverse-central-nervous-system-disorders-inherited-metabolic-diseases-and-autism
#18
REVIEW
Jessica M Sun, Joanne Kurtzberg
The concept of utilizing human cells for the treatment of medical conditions is not new. In its simplest form, blood product transfusion as treatment of severe hemorrhage has been practiced since the 1800s. The advent of hematopoietic stem cell transplantation (HSCT) began with the development of bone marrow transplantation for hematologic malignancies in the mid-1900s and is now standard of care for many hematologic disorders. In the past few decades, HSCT has expanded to additional sources of donor cells, a wider range of indications, and the development of novel cell products...
October 6, 2017: Pediatric Research
https://www.readbyqxmd.com/read/28985199/renal-lineage-cells-as-a-source-for-renal-regeneration
#19
REVIEW
Oren Pleniceanu, Dorit Omer, Orit Harari-Steinberg, Benjamin Dekel
The mammalian kidney is a highly complex organ, composed of various cell types within a unique structural framework. Nonetheless, in recent years, giant leaps in our understanding of nephrogenesis and the origin of new cells in the adult kidney, have resulted in novel routes to regenerate damaged nephrons. While several strategies can be envisioned to achieve this aim, one common theme is the reliance on renal lineage cells, since extra-renal cells, such as bone-marrow derived cells, have been shown to be devoid of renal differentiation capacity...
October 6, 2017: Pediatric Research
https://www.readbyqxmd.com/read/28945662/incidence-and-outcome-of-pericardial-effusion-in-pediatric-patients-after-hematopoietic-stem-cell-transplant-a-single-institution-experience
#20
Melissa Diamond, Catalina Ruiz-Mesa, Fernando F Corrales-Medina, Leonardo J Tamariz, Edward Ziga, Sethuraman Swaminathan
BACKGROUND: Pericardial effusion (PE) is a known complication after hematopoietic stem cell transplant (HSCT). Limited data is currently available regarding the incidence and outcomes of PE in pediatric HSCT. METHODS: We conducted a retrospective study on a cohort of patients who underwent HSCT between 2004 and 2015. Risk factors associated with development of PE were evaluated. RESULTS: In 111 HSCT, stem cell source was bone marrow in 37 (33...
September 22, 2017: Journal of Pediatric Hematology/oncology
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