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Pediatric allogenic stem cell transplant

Asaf D Yanir, Caridad A Martinez, Ghadir Sasa, Kathryn Leung, Stephen Gottschalk, Bilal Omer, Nabil Ahmed, Meenakshi Hegde, Jo Eunji, Hao Liu, Helen E Heslop, Malcolm K Brenner, Robert A Krance, Swati Naik
Hematopoietic stem cell transplantation (HSCT) is the only curative option for a subset of patients with high-risk or relapsed Acute Lymphoblastic Leukemia (ALL). Given evolving practices, it is important to continually evaluate outcomes for pediatric ALL following HSCT. Outcomes after HSCT are influenced by the type of donor used as this determines the degree and method of T-cell depletion used, and consequently, specific transplant-related morbidities. We retrospectively analyzed HSCT data from our center for transplants performed between January 2008 and May 2016, comparing outcomes between different donor types...
March 14, 2018: Biology of Blood and Marrow Transplantation
Arti Nanda, Maitham A A Husain, Waleed Al-Herz, Adla Almekaimi, Humoud Al-Sabah, Mohammad Al-Otaibi
BACKGROUND/OBJECTIVES: Allogeneic hematopoietic stem cell transplantation (HSCT) is a treatment option for many life-threatening disorders in children. Chronic graft-versus-host disease (cGVHD) is a significant complication of HSCT, and its treatment is challenging. Skin is the most common organ affected in cGVHD, with protean manifestations posing a challenge in diagnosis and management. The objective was to have a better understanding of the spectrum of chronic cutaneous GVHD (cc-GVHD) in children...
March 14, 2018: Pediatric Dermatology
Thomas Magg, Tilmann Schober, Christoph Walz, Julia Ley-Zaporozhan, Fabio Facchetti, Christoph Klein, Fabian Hauck
Epstein-Barr virus positive (EBV+ ) smooth muscle tumors (SMTs) constitute a very rare oncological entity. They usually develop in the context of secondary immunodeficiency caused by human immunodeficiency virus infection or immunosuppressive treatment after solid organ transplantation. However, in a small fraction of predominantly pediatric patients, EBV+ SMTs may occur in patients with primary immunodeficiency disorders (PIDs), such as GATA2 and CARMIL2 deficiency. In secondary immunodeficiencies and when the underlying condition can not be cured, the treatment of EBV+ SMTs is based on surgery in combination with antiretroviral and reduced or altered immunosuppressive pharmacotherapy, respectively...
2018: Frontiers in Immunology
Karoly Toth, Jacqueline F Spencer, Baoling Ying, Ann E Tollefson, Caroll B Hartline, Eric T Richard, Jiajun Fan, Jinglei Lyu, Boris A Kashemirov, Cheryl Harteg, Dawn Reyna, Elke Lipka, Mark N Prichard, Charles E McKenna, William S M Wold
Human adenoviruses (AdV) cause generally mild infections of the respiratory and GI tracts as well as some other tissues. However, AdV can cause serious infection in severely immunosuppressed individuals, especially pediatric patients undergoing allogeneic hematopoietic stem cell transplantation, where mortality rates are up to 80% with disseminated disease. Despite the seriousness of AdV disease, there are no drugs approved specifically to treat AdV infections. We report here that USC-087, an N-alkyl tyrosinamide phosphonate ester prodrug of HPMPA, the adenine analog of cidofovir, is highly effective against multiple AdV types in cell culture...
March 3, 2018: Antiviral Research
Nataliya P Buxbaum, Cemre Robinson, Ninet Sinaii, Alexander Ling, Lauren M Curtis, Steven Z Pavletic, Kristin Baird, Maya B Lodish
Pediatric allogeneic hematopoietic stem cell transplant (AHSCT) recipients with chronic graft-versus-host disease (cGVHD) are at high risk of endocrinopathies, particularly impaired bone mineral density (BMD). However, rates of BMD impairment in pediatric AHSCT recipients with cGVHD have not been well documented. We describe 33 patients with cGVHD that were referred to the NIH for the Natural History of Clinical and Biological Factors Determining Outcomes in Chronic Graft-Versus-Host Disease Study (NCT 0092235) and underwent formal BMD assessments via dual-energy X-ray absorptiometry (DEXA)...
February 26, 2018: Biology of Blood and Marrow Transplantation
Coco de Koning, Rick Admiraal, Stefan Nierkens, Jaap Jan Boelens
Human herpesvirus 6 (HHV6) viremia is a common cause of morbidity following allogeneic hematopoietic cell transplantation (HCT). We previously associated T-cell reconstitution with HHV6 viremia. Here, we investigated whether HHV6 viremia affects T-cell reconstitution after HCT in a time-dependent retrospective analysis. We included 273 pediatric patients (0.1-22.7 years; median follow-up, 58 months) receiving a first HCT between 2004 and 2014. HHV6 was screened weekly in plasma via polymerase chain reaction and occurred in 79 patients (29%) at a median time of 19 days after transplant...
February 27, 2018: Blood Advances
C M Jaramillo-Esparza, A Consuelo-Sánchez, C P Acosta-Rodríguez-Bueno, G Ramón-García, S W Sadowinski-Pine, M A Escobar-Sánchez, I Castorena-Villa, F Gaytán-Morales, R Vázquez-Frias
INTRODUCTION AND AIMS: Graft-versus-host disease (GVHD) is a common multisystemic complication of allogeneic hematopoietic cell transplantation. The most frequent presentations of graft-versus-host disease involve the skin, the gastrointestinal tract, and the liver. The aim of the present study was to know the frequency of gastrointestinal tract and liver GVHD and the characteristics of disease presentation in pediatric patients that underwent hematopoietic stem cell transplantation (HSCT) at a tertiary care hospital center in Mexico City...
February 24, 2018: Revista de Gastroenterología de México
Adrien Picod, Agnès Bonnin, Giorgia Battipaglia, Federica Giannotti, Annalisa Ruggeri, Eolia Brissot, Florent Malard, Clémence Médiavilla, Ramdane Belhocine, Anne Vekhoff, Mor Sény Gueye, Simona Lapusan, Rosa Adaeva, Françoise Isnard, Ollivier Legrand, Minh-Tam Baylatry, Anne-Christine Joly, Myriam Labopin, Rémy Duléry, Mohamad Mohty
Sinusoidal obstruction syndrome (SOS), also known as hepatic veno-occlusive disease (VOD) is a serious complication after hematopoietic stem cell transplantation (HSCT). SOS/VOD usually occurs within 3 weeks of HSCT but the 2016 EBMT diagnosis criteria have been revised to include late forms. Prophylactic use of defibrotide is recommended in the pediatric setting but its value remains uncertain in the adult population. We report here a single center series of 63 adult patients considered at high risk of SOS/VOD, receiving defibrotide prophylaxis in combination with ursodeoxycholic acid between May 2012 and August 2016...
February 22, 2018: Biology of Blood and Marrow Transplantation
Bianca Borchert-Mörlins, Nima Memaran, Martin Sauer, Britta Maecker-Kolhoff, Karl-Walter Sykora, Ricarda Blöte, Elena Bauer, Bernhard M W Schmidt, Anette Melk, Rita Beier
Advances in allogeneic hematopoietic stem cell transplantation (HSCT) in malignant and non-malignant diseases result in more long-term survivors, in whom cardiovascular (CV) disease is one leading non-cancer cause of death. This study aimed to evaluate risk factors and subclinical CV organ damage in survivors after HSCT in pediatric age. We enrolled 64 children in a cross-sectional approach 3.3 ± 3.1 years after HSCT. Anthropometric data, laboratory values, office and 24-h ambulatory blood pressure monitoring (ABPM) were evaluated, showing a high prevalence of obesity, hypertension and dyslipidemia...
February 9, 2018: Bone Marrow Transplantation
Jingbo Wang, Lei Yuan, Haoyu Cheng, Xinhong Fei, Yumin Yin, Jiangying Gu, Song Xue, Junbao He, Fan Yang, Xiaocan Wang, Yixin Yang, Weijie Zhang
There is an ongoing debate concerning the performance of salvaged allogeneic hematopoietic stem cell transplantation (allo-HSCT) in pediatric patients with acute refractory leukemia, in whom the prognosis is quite dismal. Few studies have ever been conducted on this subject. This may be partly due to missed opportunities by majority of the patients in such situations. To investigate the feasibility, evaluate the efficiency, and identify the prognostic factors of allo-HSCT in this sub-setting, the authors performed a single institution-based retrospective analysis...
January 9, 2018: Oncotarget
Ehud Even-Or, Hasan Ghandourah, Muhammad Ali, Joerg Krueger, Neil B Sweezey, Tal Schechter
BOS is the pulmonary manifestation of cGvHD post-allogeneic HSCT. Survival and treatment of this often fatal complication have not improved over the last 20 years and there is no clear standard of care. For the past 10 years, BOS was treated in our center with monthly cycles of HDPS. We reviewed the outcomes of patients with post-HSCT BOS who met the diagnostic criteria for BOS as per the NIH consensus and were treated with at least one cycle of methylprednisolone at a dose of 10-30 mg/kg/d×3 d. We collected demographic and clinical data, responses to treatment and results of pulmonary function tests at several time points...
February 7, 2018: Pediatric Transplantation
Chiara Messina, Marco Zecca, Franca Fagioli, Attilio Rovelli, Stefano Giardino, Pietro Merli, Fulvio Porta, Maurizio Aricò, Elena Sieni, Giuseppe Basso, Mimmo Ripaldi, Claudio Favre, Marta Pillon, Antonio Marzollo, Marco Rabusin, Simone Cesaro, Mattia Algeri, Maurizio Caniglia, Paolo Di Bartolomeo, Ottavio Ziino, Francesco Saglio, Arcangelo Prete, Franco Locatelli
We report on 109 patients with hemophagocytic lymphohistiocytosis (HLH) undergoing 126 procedures of allogeneic hematopoietic stem cell transplantation (HSCT) between 2000 and 2014 in centers associated with the Italian Pediatric Hematology Oncology Association. Genetic diagnosis was FHL2 (32%), FHL3 (33%) or other defined disorders known to cause HLH (15%); in the remaining patients, no genetic abnormality was found. Donor for first transplant was an HLA-matched sibling for 25 patients (23%), an unrelated donor for 73 (67%) and an HLA-partially matched family donor for 11 children (10%)...
February 1, 2018: Biology of Blood and Marrow Transplantation
Larisa Broglie, Alfred Rademaker, John Galvin, Ayita Ray, William T Tse, Reggie Duerst, Jennifer Schneiderman, Morris Kletzel, Sonali Chaudhury
BACKGROUND: Acute graft versus host disease (aGVHD) affects approximately 30-60% of patients after allogeneic hematopoietic stem cell transplantation (HCT) and our ability to predict who develops this complication and their response to treatment is limited. Fecal calprotectin has recently gained popularity as an effective marker of GI inflammation in patients with Inflammatory Bowel Disease (IBD). METHODS: Fecal calprotectin and albumin were evaluated as prognostic and predictive markers of aGVHD in 60 adult and pediatric HCT patients...
January 31, 2018: Hematology/oncology and Stem Cell Therapy
Agnieszka Zaucha-Prazmo, Jolanta Gozdzik, Robert Debski, Katarzyna Drabko, Elzbieta Sadurska, Jerzy R Kowalczyk
The aim of the study was to assess the risk of TRM in pediatric patients treated for malignant disorders with allogeneic HSCT, according to different risk factors. The treatment outcome was analyzed in 299 pediatric patients treated in pediatric transplant departments from 2006 to 2015. To compare the outcome, patients were analyzed all together and in groups according to the diagnosis, age at transplant, donor type, disease status, stem cell source, and pediatric TRM score. At the end of the observation time, 82 patients were alive, 82 died, of which 40 due to transplant-related reasons...
February 3, 2018: Pediatric Transplantation
A M Flinn, C F Roberts, M A Slatter, R Skinner, H Robson, J Lawrence, J Guest, A R Gennery
Acute graft-versus-host disease (aGVHD) complicates allogeneic hematopoietic stem cell transplantation (HSCT), and is treated with topical and/or systemic corticosteroids. Systemic corticosteroids and aGVHD damage thymic tissue. We compared thymopoietic effect of topical steroid therapy, corticosteroids and extracorporeal photopheresis (ECP) in 102 pediatric allogeneic HSCT patients. We categorized patients into 4 groups: - no aGVHD, aGVHD treated with topical or systemic steroid, or ECP. Naïve CD4+CD45RA+CD27+ T-lymphocyte values at 3, 6, 9, 12months post-HSCT were recorded: for ECP patients, values were recorded at 3, 6, 9, 12months during ECP...
January 27, 2018: Clinical Immunology: the Official Journal of the Clinical Immunology Society
Xiaohong Zhang, Wenge Hao, Tao Xu, Sha Liu, Hua Jiang
INTRODUCTION: Post-transplant lymphoproliferative disorder (PTLD) is the most common form of lymphoproliferation in childhood and is associated with significant morbidity and mortality. In this report we reviewed the case of a pediatric patient who experienced PTLD after allogeneic hematopoietic stem cell transplantation (HSCT) from a human leukocyte antigen (HLA)-identical sibling. METHODS: The clinical characteristics, diagnosis, and treatment of PTLD after sibling HSCT in a 4-year-old boy with severe β-thalassemia was retrospectively reviewed...
December 2017: Medicine (Baltimore)
Salwa S Saadeh, Mark R Litzow
Allogeneic hematopoietic stem cell transplant (allo-HSCT) has an important role in management of acute lymphoblastic leukemia (ALL). Proper patient selection is central to ensure optimal outcomes. Areas covered: This review covers various aspects of HSCT in ALL patients, including indications, donor selection, conditioning regimens, and post-transplant management. Expert commentary: Allo-HSCT is important in post-remission management of ALL but proper risk-stratification is a major challenge. Incorporation of minimal residual disease (MRD) and molecular testing will improve patient allocation...
March 2018: Expert Review of Hematology
Orsolya Horváth, Krisztián Kállay, Dorottya Csuka, Blanka Mező, György Sinkovits, Csaba Kassa, Anita Stréhn, Katalin Csordás, János Sinkó, Zoltán Prohászka, Gergely Kriván
Hematopoietic stem cell transplantation- (HSCT-) associated thrombotic microangiopathy (TA-TMA) is a multifactorial complication, and its prediction is largely unresolved.Our aim was to analyze changes of complement profile after HSCT in order to identify potential markers of TA-TMA development. Thirty- three consecutive pediatric patients (9.6 ± 4.4 years old) were included in this study who underwent allogeneic HSCT due to malignant (n=17) or non malignant (n=16) indications. Graft versus host disease (GVHD) was diagnosed using Glucksberg criteria, viral reactivation was monitored, five different TA-TMA diagnostic criteria were applied and all important clinical and laboratory parameters of TA-TMA activity were registered...
January 12, 2018: Biology of Blood and Marrow Transplantation
Karin Kosulin, Bettina Berkowitsch, Susanne Matthes, Herbert Pichler, Anita Lawitschka, Ulrike Pötschger, Gerhard Fritsch, Thomas Lion
Human adenoviruses (HAdV) are a major cause of morbidity and mortality in pediatric human stem cell transplant (HSCT) recipients. Our previous studies identified the gastrointestinal tract as a site of HAdV persistence, but the role of intestinal virus shedding pre-transplant for the risk of ensuing invasive infection has not been entirely elucidated. Molecular HAdV monitoring of serial stool samples using RQ-PCR was performed in 304 children undergoing allogeneic HSCT. Analysis of stool and peripheral blood specimens was performed pre-transplant and at short intervals until day 100 post-HSCT...
January 9, 2018: EBioMedicine
Tang-Her Jaing, Shih-Hsiang Chen, Yu-Chuan Wen, Tsung-Yen Chang, Dai-Yun Tsai, Hung-Tao Chung, Pei-Kwei Tsay
The objective of this study was to determine the incidence, risk factors, outcome, and clinical significance of pericardial effusion (PE). We retrospectively analyzed outcomes of 272 pediatric patients undergoing their first hematopoietic stem cell transplantation (HSCT) from 1998 to 2016. In total, 15% (3/20) and 5.9% (15/252) of autologous and allogeneic HSCT recipients, respectively, were identified with PE. However, there was no statistically significant difference in the incidence of PE between the 2 groups...
November 2017: Cell Transplantation
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