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bone marrow transplant pediatric

Xin-Yu Li, Xin Sun, Jing Chen, Mao-Quan Qin, Zuo Luan, Yi-Ping Zhu, Jian-Pei Fang
BACKGROUND: β-Thalassemia major (β-TM) has become a public health problem in mainland China. Hematopoietic stem cell transplantation (HSCT) has remained the only cure for β-TM in mainland China since 1998. METHODS: This multicenter retrospective study provides a comprehensive review of the outcomes of 50 pediatric patients with β-TM who received HSCT between 1998 and 2009 at five centers in mainland China. Both related (n = 35) and unrelated donors (n = 15) with complete human leukocyte antigen matches were included...
March 6, 2018: World Journal of Pediatrics: WJP
F Martino, R Di Mauro, K Paciaroni, J Gaziev, C Alfieri, L Greco, R Floris, S Di Girolamo, M Di Girolamo
OBJECTIVES: Sickle cell anemia (SCA) and β -thalassemia major are well-recognized beta-globin gene disorders of red blood cells associated to mortality and morbidity included bone morbidities due to ineffective erythropoiesis and bone marrow expansion, which affect every part of the skeleton. While there are an abundance of described disease manifestations of the head and neck, the manner of paranasal sinuses involvement and its relations to β-thalassemia and SCA process was not studied yet...
March 2018: International Journal of Pediatric Otorhinolaryngology
Jun Wu, Hongxi Guo, Xing Liu, Ming Li, Yujiang Cao, Xiangyang Qu, Hai Zhou, Liuqi Weng
Background and purpose: Percutaneous autologous bone marrow transplantation (PABMT) is a minimally invasive therapeutic strategy for the treatment of delayed bone union in adults, which has been confirmed by many studies. However, there is no report on PABMT application in pediatric orthopedic surgery. The aim of this article was to analyze the therapeutic effect of PABMT in children with delayed union of limb bone and its influence in relation to delayed bone union therapy, transplantation period, patients' sex, fracture location, and fracture fixation...
2018: Therapeutics and Clinical Risk Management
Chayamon Takpradit, Susan E Prockop, Nancy A Kernan, Andromachi Scaradavou, Kevin Curran, Julianne Ruggiero, Nicole Zakak, Richard J O'Reilly, Farid Boulad
BACKGROUND: The combination of cyclophosphamide (CY) and antithymocyte globulin (ATG) has been used as a standard conditioning regimen for matched related donor transplantation in patients with severe aplastic anemia. PROCEDURE: To decrease the regimen-related toxicity while maintaining appropriate engraftment and survival rates, fludarabine (FLU) was added to the regimen. Four pediatric patients received matched related donor bone marrow transplantation with CY (50 mg/kg×2) (instead of the 50 mg/kg×4 standard dosing), equine ATG (30 mg/kg×3), with the addition of FLU (30 mg/m×4)...
February 9, 2018: Journal of Pediatric Hematology/oncology
Kosuke Akiyama, Shohei Yamamoto, Yumiko Sugishita, Ryota Kaneko, Naoko Okamoto, Masaya Koganesawa, Sachio Fujita, Ryosuke Matsuno, Daisuke Toyama, Keiichi Isoyama
A 9-year-old girl was referred to our hospital because of facial palsy. Both physical and blood examination revealed hepatosplenomegaly and leukocytosis, respectively. A bone marrow examination demonstrated marked hypercellularity involving myeloblasts and lymphoblasts. Based on these results, we suspected mixed phenotype acute leukemia. However, her leukemic blasts expressed B-cell antigens, and a chromosomal analysis of her bone marrow cells revealed the following karyotype: 46, XX, t (9;22) (q34;q11.2). All her neutrophils were positive for the breakpoint cluster region-Abelson murine leukemia viral oncogene homolog 1 fusion protein...
2018: [Rinshō Ketsueki] the Japanese Journal of Clinical Hematology
Christian Nusshag, Christian Morath, Martin Zeier, Markus A Weigand, Uta Merle, Thorsten Brenner
RATIONALE: Hemophagocytic lymphohistiocytosis (HLH) is a life-threatening disease entity primarily described in children, but not less relevant in adults. It is characterized by a misdirected activation of the immune system, resulting in an uncontrolled cytokine release from macrophages and cytotoxic T-cells (CTLs). Primary HLH relies on a genetic predisposition, whereas secondary HLH develops in the context of infections, malignancies or autoimmune diseases. However, the awareness and therapeutic knowledge for HLH in adulthood is limited...
December 2017: Medicine (Baltimore)
Racha Khalaf, Carmen Cuffari
Androgen therapy has proven efficacy in treating patients with bone marrow failure who are not candidates for bone marrow transplantation. Herein, we report on a case of colonic angioectasia secondary to oxymetholone use in an adolescent patient with Hoyeraal-Hreidarsson syndrome (HHS). A 13-year-old Caucasian male with HHS characterized by cerebellar hypoplasia, developmental delay, microcephaly, esophageal strictures and myelodysplasia presented with severe hematochezia from colonic angioectasia secondary to long-term oxymetholone therapy...
January 2018: Pediatric Gastroenterology, Hepatology & Nutrition
Christina Mangurian, Wendy Packman, Nicholas S Riano, Julia Kearney
OBJECTIVE: Pediatric bone marrow transplants represent a medically stressful, potentially traumatic experience for children and caregivers, and psychological support for parental caregivers is paramount to their long-term well-being. However, many medical centers do not have protocols in place to sustain caregiver well-being during these distressing experiences. METHOD: We report on a case of a 10-month-old infant with Wiskott Aldrich Syndrome who was hospitalized for bone marrow transplantation...
January 30, 2018: Palliative & Supportive Care
Ibukunoluwa C Akinboyo, Genevieve M Crane, Lingling Chen, Ravit Arav-Boger
Human T-cell lymphotropic virus (HTLV), an infection that is endemic in certain parts of Asia, Africa, and South America, has been associated with malignancy and neurological deficits. Here, we describe a pediatric patient with chronic HTLV-I infection who developed complications associated with HTLV-I (ie, adult T-cell leukemia/lymphoma and HTLV-I-associated myelopathy/tropical spastic paraparesis). To our knowledge, this presentation in a child has never been described. The patient underwent a bone marrow transplant and, at the time of this writing, was in remission...
January 24, 2018: Journal of the Pediatric Infectious Diseases Society
Omar Irfan, Sana Mahmood, Heera Nand, Gaffar Billoo
BACKGROUND: Celiac disease may present with hematological abnormalities including long-standing anemia. Both aplastic anemia and celiac disease have a similar underlying autoimmune process but an association between the two is seldom reported. There have only been three pediatric cases reporting this association and this case is the first reported in a female pediatric patient. CASE PRESENTATION: We report a case of 6-year-old South Asian girl presenting with bruises, petechiae, and recent history of loose stools...
January 23, 2018: Journal of Medical Case Reports
Pragnya Coca, Vijay Gandhi Linga, Sadashivudu Gundeti, Ashwani Tandon
Renal lymphoma is an uncommon renal tumor in children. Unlike renal lymphomas presenting as bilateral disease and renal failure, we report a boy who presented with unilateral renal involvement. After initial nephrectomy, he achieved remission with multiagent chemotherapy but relapsed systemically within 3 months. He was initiated on salvage chemotherapy with autologous bone marrow transplant. Even though the initial manifestation was localized lymphoma eventually, it turned out to be a systemic disease. He succumbed to disease at 14 months from diagnosis...
October 2017: Indian Journal of Medical and Paediatric Oncology
Bryan T Nycz, Samuel R Dominguez, Deborah Friedman, Joanne M Hilden, Diana Ir, Charles E Robertson, Daniel N Frank
Bloodstream infections (BSI) and Clostridium difficile infections (CDI) in pediatric oncology/hematology/bone marrow transplant (BMT) populations are associated with significant morbidity and mortality. The objective of this study was to explore possible associations between altered microbiome composition and the occurrence of BSI and CDI in a cohort of pediatric oncology patients. Stool samples were collected from all patients admitted to the pediatric oncology floor from Oct.-Dec. 2012. Bacterial profiles from patient stools were determined by bacterial 16S rRNA gene profiling...
2018: PloS One
Olga Milczarek, Danuta Jarocha, Anna Starowicz-Filip, Stanislaw Kwiatkowski, Bogna Badyra, Marcin Majka
There is a need among patients suffering from drug-resistant epilepsy (DRE) for more efficient and less toxic treatments. The objective of the present study was to assess the safety, feasibility, and potential efficacy of autologous bone marrow cell transplantation in pediatric patients with DRE. Two females and two males (11 months to 6 years) were enrolled and underwent a combined therapy consisting of autologous bone marrow nucleated cells (BMNCs) transplantation (intrathecal: 0.5 × 109 ; intravenous: 0...
January 2018: Stem Cells Translational Medicine
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 that lack the CAR targeted antigen. In this situation, a bispecific CAR with binding domains for CD19 and CD20 is aimed at recognizing 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 2017: Human Gene Therapy
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
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
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
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
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.
December 2017: Bulletin du Cancer
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
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