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bone marrow transplant pediatric

Xiaoli Wu, Xuefeng Feng, Xiaoqing Zhao, Futian Ma, Na Liu, Hongming Guo, Chaonan Li, Huan Du, Baoxi Zhang
BACKGROUND/AIMS: Acute and chronic leukemia are severe malignant cancers worldwide, and can occur in pediatric patients. Since bone marrow cell transplantation is seriously limited by the availability of the immune-paired donor sources, the therapy for pediatric leukemia (PL) remains challenging. Autophagy is essential for the regulation of cell survival in the harsh environment. However, the role of autophagy in the survival of PL cells under the oxidative stress, e.g. chemotherapy, remain ill-defined...
October 17, 2016: Cellular Physiology and Biochemistry
Andrew C Dietz, Christine N Duncan, Blanche P Alter, Dorine Bresters, Morton J Cowan, Luigi Notarangelo, Philip S Rosenberg, Shalini Shenoy, Roderick Skinner, Mark C Walters, John Wagner, K Scott Baker, Michael A Pulsipher
An international consensus conference sponsored by the Pediatric Blood and Marrow Transplant consortium entitled, "Late Effects Screening and Recommendations Following Allogeneic Hematopoietic Cell Transplant for Immune Deficiency and Non-malignant Hematologic Disease was held in Minneapolis, Minnesota on May 10-11, 2016. The purpose of the conference was to address the unmet need for a greater understanding of and the screening for long-term complications in the growing population of survivors of transplantation for non-malignant disorders...
October 10, 2016: Biology of Blood and Marrow Transplantation
Brian Y Chan, Kara G Gill, Susan L Rebsamen, Jie C Nguyen
The bone marrow is one of the largest organs in the body and is visible in every magnetic resonance (MR) imaging study. It is composed of a combination of hematopoietic red marrow and fatty yellow marrow, and its composition changes throughout life in response to normal maturation (red to yellow conversion) and stress (yellow to red reconversion). MR imaging is highly sensitive for detection of altered marrow signal intensity, and the T1-weighted spin-echo sequence provides the most robust contrast between yellow marrow and disease...
October 2016: Radiographics: a Review Publication of the Radiological Society of North America, Inc
Ting Yang, Qiaoxian Lin, Jinhua Ren, Ping Chen, Xiaohong Yuan, Xiaofeng Luo, Tingbo Liu, Jing Zheng, Zhihong Zheng, Xiaoyun Zheng, Xinji Chen, Langhui Zhang, Hao Zheng, Zaisheng Chen, Xueling Hua, Shaohua Le, Jian Li, Zhizhe Chen, Jianda Hu
Haplo-HSCT has been used when HLA-matched siblings are not available. Conditioning regimens aim to reduce tumor burden prior to HSCT and provide sufficient immunoablation. We report the outcome of haplo-HSCT in 63 consecutive patients from 2/2013 to 12/2015 (19 females/44 males) with high-risk or relapsed/refractory hematological malignancies (n=29-AML; 8-sAML; 19-ALL; 5-advanced-MDS; 2-CML-BC). Median age was 20 years (range: 1.1-49). Twenty-one patients achieved remission prior to transplant, while 42 did not...
October 1, 2016: Oncotarget
Sonia Halioui-Louhaichi, Ons Azzabi, Nadia Mattoussi, Hasna Labiadh, Khadija Bousseta, Neji Tebib, Taher Gargah, Sayda Ben Becher, Mohamed Ridha Barbouch, Mohamed Bejaoui, Ahmed Maherzi
Background Primary immunodeficiencies (PID) are a group of heterogeneous and relatively rare diseases. Aim to determine the clinical characteristics, outcome and genetic data of primary immunodeficiencies in pediatrics patients. Methods A retrospective, descriptive and multicentered study, enrolling 33 children presenting a PID in Tunis, during a period of 22 years (1991-2012). Resultats a masculine predominance has been noticed with a sex ratio at 2,3. Consanguinity was found in 71% of family cases. History of early infant deaths was found in 42% of cases...
April 2016: La Tunisie Médicale
S Struski, S Lagarde, P Bories, C Puiseux, N Prade, W Cuccuini, M-P Pages, A Bidet, C Gervais, M Lafage-Pochitaloff, C Roche-Lestienne, C Barin, D Penther, N Nadal, I Radford-Weiss, M-A Collonge-Rame, B Gaillard, F Mugneret, C Lefebvre, A Petit, G Leverger, C Broccardo, I Luquet, M Pasquet, E Delabesse
Pediatric acute myeloid leukemia (AML) is a rare disease whose prognosis is highly variable according to factors such as chromosomal abnormalities. Recurrent genomic rearrangements are detected in half of pediatric AML by karyotype. NUcleoPorin 98 (NUP98) gene is rearranged with 31 different fusion partner genes. These rearrangements are frequently undetected by conventional cytogenetics, since the NUP98 gene is located at the end of the chromosome 11 short arm (11p15). By screening a series of 574 pediatric AML, we detected a NUP98 rearrangement in 22 cases (3...
October 3, 2016: Leukemia: Official Journal of the Leukemia Society of America, Leukemia Research Fund, U.K
J L Grow, B Stea, E Katsanis
No abstract text is available yet for this article.
October 1, 2016: International Journal of Radiation Oncology, Biology, Physics
Aziza T Shad, Jeffrey S Huo, Courtney Darcy, Amal Abu-Ghosh, Giuseppe Esposito, Mary-Jo Holuba, Nancy Robey, Kenneth R Cooke, Heather J Symons, Allen R Chen, Nicolas J Llosa
To date, there has been a lack of pediatric experience regarding the efficacy and tolerability of immune checkpoint inhibitors after haploidentical hematopoietic stem cell transplant (HSCT). We present the case of a 22-year-old female with multiple-relapsed Hodgkin lymphoma (HL) who presented with a new relapse after haploidentical (post-haplo) HSCT. Anti-PD-1 therapy with nivolumab resulted in significant objective disease response and clinical improvement without notable side effects, including the absence of a graft-versus-host disease (GVHD)...
September 21, 2016: Pediatric Blood & Cancer
S H Lum, A Turner, M Guiver, D Bonney, T Martland, E Davies, M Newbould, J Brown, S Morfopoulou, J Breuer, R Wynn
Neuroinvasive astrovirus (VA1-HMO-C) is an emerging life-threatening infection in immunocompromised hosts. We describe an 8-month-old child who died of VA1/HMO-C encephalitis following bone marrow transplantation. The diagnosis was only made post-mortem using RNA deep sequencing of the brain. Repeat analysis of the post-mortem brain tissue using PCR specific primers for VA1/HMO-C was positive. Astrovirus VA1/HMO-C should be included in the evaluation of patients with similar encephalitis. This article is protected by copyright...
September 15, 2016: Transplant Infectious Disease: An Official Journal of the Transplantation Society
A A Hussein, A A Hamidieh, A Elhaddad, M Ramzi, T B Othman, F Hussain, D Dennison, P Ahmed, M Abboud, A Al-Ahmari, A Wahadneh, J Fathy, M-A Bekadja, S Al-Kindi, S Benchekroun, A Ibrahim, M Behfar, M Samra, S Ladeb, S Adil, H El-Solh, M Ayas, M Aljurf, A Ghavamzadeh, A Al-Seraihy
To describe the hematopoietic stem cell transplantation (HSCT) activities for children in the Eastern Mediterranean (EM) region, data on transplants performed for children less than 18 years of age between 1984 and 2011 in eight EM countries (Egypt, Iran, Jordan, Lebanon, Oman, Pakistan, Saudi Arabia and Tunisia) were collected. A total of 5187 transplants were performed, of which 4513 (87%) were allogeneic and 674 (13%) were autologous. Overall, the indications for transplantation were malignant diseases in 1736 (38...
September 12, 2016: Bone Marrow Transplantation
A Sirvent, P Auquier, C Oudin, Y Bertrand, S Bohrer, P Chastagner, M Poirée, J Kanold, S Thouvenin, Y Perel, D Plantaz, M-D Tabone, K Yakouben, V Gandemer, P Lutz, N Sirvent, C Vercasson, J Berbis, H Chambost, G Leverger, A Baruchel, G Michel
Data on post-transplant iron overload (IO) are scarce in pediatrics. We conducted a prospective multicenter cohort study (Leucémie de l'Enfant et de l'Adolescent cohort) to determine the prevalence and risk factors of IO in 384 acute leukemia survivors transplanted during childhood. Prevalence of IO (ferritin level ⩾350 ng/mL) was 42.2% (95%CI 37.2-47.2%). Factors significantly associated with IO were: 1) in univariate analysis: older age at transplant (P<0.001), allogeneic versus autologous transplantation (P<0...
September 5, 2016: Bone Marrow Transplantation
Michaël Philippe, Michael Neely, Yves Bertrand, Nathalie Bleyzac, Sylvain Goutelle
The traditional approach for model-based initial dosing is based on the use of a single vector of typical population parameters for targeting a specific exposure. This approach is theoretically ill-suited for targeting a range of exposure. The objective of this work was to develop a general approach for optimal (OPT) targeting of a drug exposure interval. After methodological purposes, we applied our method to the busulfan case. We used a nonparametric population pharmacokinetic model of intravenous busulfan to estimate the individual pharmacokinetic parameters of 163 bone marrow-transplanted children...
September 1, 2016: Clinical Pharmacokinetics
Kang An, Ying Wang, Biru Li, Changying Luo, Jianmin Wang, Chengjuan Luo, Jing Chen
BACKGROUND: There are many studies about the prognosis and possible predictive factors of mortality for pediatric allogeneic hematopoietic stem cell transplantation (HSCT) recipients requiring pediatric intensive care unit (PICU) treatment, but the related study in China is lacking. This study investigates the data of these special patients in our center. METHODS: This retrospective analysis is based on data from bone marrow center and PICU of our hospital. A total of 302 patients received allogeneic HSCT from January 2000 to December 2012, 29 of them were admitted to PICU because of various complications developed after transplantation...
August 20, 2016: BMC Pediatrics
Nicolas Waespe, Machiel Van Den Akker, Robert J Klaassen, Lani Lieberman, Meredith S Irwin, Salah S Ali, Mohamed Abdelhaleem, Bozana Zlateska, Mira Liebman, Michaela Cada, Tal Schechter, Yigal Dror
Advanced myelodysplastic syndrome harbors a high risk of progression to acute myeloid leukemia and poor prognosis. In children, there is no established treatment to prevent or delay progression to leukemia prior to hematopoietic stem cell transplantation. Azacitidine is a hypomethylating agent, which was shown to slow progression to leukemia in adults with myelodysplastic syndrome. There is little data on the efficacy of azacitidine in children. We reviewed 22 pediatric patients with advanced myelodysplastic syndrome from a single center, diagnosed between January 2000 and December 2015...
August 18, 2016: Haematologica
Lauren Arcuri
Dunbar, a registered nurse at Presbyterian Hospital in Albuquerque, N.M., knows firsthand the struggles patients and their families endure during cancer treatment. Her son underwent a bone marrow transplant at age 22. In 2011, Dunbar organized an annual race to raise funds for families who have children with cancer.
July 2016: Hospitals & Health Networks
Tomomi Hayase, Hitomi Niijima, Daisuke Tanaka, Syoya Wada, Yuta Kawahara, Yukiko Oh, Akira Morimoto
Pediatric anaplastic large cell lymphoma (ALCL) is a chemosensitive malignancy, but about 30% of patients experience relapse. In most of these patients, a second complete remission is obtainable with salvage chemotherapy, though relapse free survival rates are as low as 30-60%. Herein, we report a 6-year-old boy with relapsed anaplastic lymphoma kinase (ALK) positive ALCL successfully treated with vinblastine monotherapy followed by allogeneic hematopoietic stem cell transplantation (allo-HSCT), with a reduced intensity conditioning (RIC) regimen, from his father...
July 2016: [Rinshō Ketsueki] the Japanese Journal of Clinical Hematology
Pooja Khandelwal, Tsuyoshi Fukuda, Kana Mizuno, Ashley Teusink-Cross, Parinda A Mehta, Rebecca A Marsh, Angela D M Kashuba, Alexander A Vinks, Stella M Davies
Maraviroc is an allosteric small molecule antagonist of chemokine receptor type 5 (CCR5) and has been used in adult allogeneic hematopoietic stem cell transplant (HSCT) recipients to prevent acute graft-versus-host disease (GVHD) of the gastrointestinal (GI) tract and liver. The goal of this study was to establish feasibility and pharmacokinetic and pharmacodynamic profiles of maraviroc in pediatric HSCT recipients. Children ages 2 to 12 years were enrolled and maraviroc was added to standard GVHD prophylaxis, which included a calcineurin inhibitor and either steroids or mycophenolate mofetil...
October 2016: Biology of Blood and Marrow Transplantation
Natalia Maximova, Massimo Gregori, Francesca Barbieri, Antonio Pizzol, Aurelio Sonzogni
BACKGROUND: Liver biopsies in pediatric hematopoietic stem cell transplantation (HSCT) patients are as and effective when performed at bedside in the Bone Marrow Transplant Unit (BMTU) than in the Day Surgery Unit (DSU), with better patient compliance and lower emotional distress for these children. METHODS: The study group consisted of 45 children who underwent allogeneic HSCT. We reviewed 68 liver biopsies performed between April 2006 and September 2015. 12 (17...
2016: BMC Cancer
O Zając-Spychała, J Wachowiak, A Pieczonka, K Siewiera, J Frączkiewicz, K Kałwak, E Gorczyńska, A Chybicka, K Czyżewski, K Jachna-Sawicka, M Wysocki, J Klepacka, J Goździk, A Zaucha-Prażmo, J R Kowalczyk, J Styczyński
BACKGROUND: Infectious complications are a significant cause of hematopoietic stem cell transplantation (HSCT) failure, especially allogeneic HSCT (allo-HSCT) because of delayed immune reconstitution and graft-versus-host disease (GVHD) occurrence. Identifying the factors responsible for bacterial infections (BI) in patients undergoing HSCT will provide much more effective empirical antimicrobial treatment in this group of patients. OBJECTIVE: The aim of this study was to evaluate the epidemiology and profile of BI in patients after HSCT in 5 centers of the Polish Pediatric Group for Hematopoietic Stem Cell Transplantation in 2012-2013...
October 2016: Transplant Infectious Disease: An Official Journal of the Transplantation Society
Jared W Glenn, Mark J Cody, Meghann P McManus, Michael A Pulsipher, Joshua D Schiffman, Christian Con Yost
Overwhelming infection causes significant morbidity and mortality among patients treated with bone marrow transplantation (BMT) for primary immune deficiencies, syndromes of bone marrow failure, or cancer. The polymorphonuclear leukocyte (PMN; neutrophil) is the first responder to microbial invasion and acts within the innate immune system to contain and clear infections. PMNs contain, and possibly clear, infections in part by forming neutrophil extracellular traps (NETs). NETs are extensive lattices of extracellular DNA and decondensed chromatin decorated with antimicrobial proteins and degradative enzymes, such as histones, myeloperoxidase, and neutrophil elastase...
2016: Frontiers in Immunology
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