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pediatric hematopoietic stem cell transplant

Nirali N Shah, Theresa M Watson, Bonnie Yates, David J Liewehr, Seth M Steinberg, David Jacobsohn, Terry J Fry
BACKGROUND: Diagnosis of engraftment syndrome (ES) following allogeneic hematopoietic stem cell transplantation (HSCT) can be a challenge due to the systemic presentation and alternative etiologies. With a goal of establishing biomarkers to more accurately distinguish ES, we prospectively analyzed levels of cytokines during HSCT. PROCEDURES: We performed a prospective study of children ≤21 years who underwent allogeneic HSCT. Blood samples for interleukin (IL)-6, IL-8, IL-10, IL-1b, IL-12p70, interferon-γ, tumor necrosis factor alpha (TNF-α) and procalcitonin were obtained from each subject prior to conditioning, at day 0, and then biweekly through engraftment and at days 30, 60 and 100...
October 20, 2016: Pediatric Blood & Cancer
Raziyeh Tootoonchian, Fatemeh Pak, Ali M Ardekani, Nasrin Sehati, Manuchehr Abedi-Valugerdi, Parviz Kokhaei
The present study tried to explain CD56+ lymphocyte cells activities and possible prognostic role of these cells in Graft-Versus-Host-Disease (GVHD). The role of IL-12 activation and function is of interest in this study. Peripheral blood samples of 51 Hematopoietic Stem Cell Transplantation (HSCT) recipients collected at before (day -8) and after (days 7 and 14). PBMC were collected by Ficoll separation and analyzed by Flow Cytometry using triple antibody (CD45-PerCP, CD56-FITC, and CD69-PE staining and control antibody...
October 14, 2016: Transplant Immunology
Kenji Kishimoto, Ryoji Kobayashi, Daiki Hori, Hirozumi Sano, Daisuke Suzuki, Kazue Yasuda, Kunihiko Kobayashi
To determine whether pretransplant PSD affects the clinical outcomes in HSCT, a retrospective cohort analysis of 73 pediatric and adolescent patients who underwent HSCT was performed. Pretransplant PSD was defined as the presence of a fluid level or mucosal swelling or total opacity on sinus X-ray or CT examination performed before HSCT. Pretransplant PSD was observed in 21 (29%) patients. The probability of 2-year OS after HSCT was 42% in patients with pretransplant PSD (PSD group), and 64% in those without (non-PSD group) (P=...
October 17, 2016: Pediatric Transplantation
Tasuku Suzuki, Yoji Sasahara, Atsuo Kikuchi, Humihiko Kakuta, Toshihiko Kashiwabara, Takashi Ishige, Yoshiko Nakayama, Masanori Tanaka, Akihiro Hoshino, Hirokazu Kanegane, Daiki Abukawa, Shigeo Kure
PURPOSE: Pediatric inflammatory bowel disease (IBD) is a heterogeneous disorder caused by multiple factors. Although genetic and immunological analyses are required for a definitive diagnosis, no reports of a comprehensive genetic study of a Japanese population are available. METHODS: In total, 35 Japanese patients <16 years of age suffering from IBD, including 27 patients aged <6 years with very early-onset IBD, were enrolled in this multicenter study. Exome and targeted gene panel sequencing was performed for all patients...
October 17, 2016: Journal of Clinical Immunology
Marissa B Larochelle, Ryan Phan, John Craddock, Mark J Abzug, Donna Curtis, Christine C Robinson, Roger H Giller, Shaun Cosgrove, Frank Siringo, Emily McCourt, Alan G Palestine
PURPOSE: The incidence of cytomegalovirus (CMV) retinitis in the pediatric allogeneic hematopoietic stem cell transplant (HSCT) population is unknown. We report a cluster of 5 pediatric patients with CMV retinitis diagnosed in a 12-month period, and compare this to the rate of CMV viremia and retinitis in the four years prior. Presented is the ophthalmic screening protocol developed in response to this experience. DESIGN: Retrospective cross-sectional study. METHODS: A retrospective chart review was performed on patients at Children's Hospital of Colorado (CHCO) who received allogeneic HSCT between January 2010 and December 2014...
October 13, 2016: American Journal of Ophthalmology
Ying Chang, Yanhui Luo, Yuchen Zhou, Ruixin Wang, Na Song, Guanghua Zhu, Bin Wang, Maoquan Qin, Jun Yang, Yuan Sun, Chunfu Li, Xuan Zhou
BACKGROUND: Long-term health-related quality of life (HRQoL) of pediatric patients after hematopoietic stem cell transplantation (HSCT) is increasingly studied worldwide. However, few studies have been performed in China, where no uniform scale is available; the PedsQL™ Cancer Module 3.0 Chinese Mandarin version has been used to evaluate HRQoL of patients after HSCT in China. This study aimed to assess the reliability and validity of the Chinese Mandarin version of PedsQL™ 3.0 Transplant Module...
October 5, 2016: Health and Quality of Life Outcomes
Emily Riehm Meier, Angeli Rampersad
Once a fatal disease of childhood, more than 95% of patients born today with sickle cell disease (SCD) in developed countries are expected to survive into adulthood, largely because of improvements in supportive and preventive care (newborn screening, penicillin prophylaxis, transcranial Doppler (TCD) screening). Hydroxyurea (HU) therapy, the only oral medication currently available to prevent SCD complications, has become more widespread over the past 20 years. The NHLBI recommends that HU be offered to all patients with HbSS beginning at nine months of age, and the recently published Abnormal TCD with Transfusions Changing to HU (TWiTCH) trial has shown HU as an acceptable alternative to transfusion therapy for patients at high risk of stroke...
October 5, 2016: Pediatric Research
Daping Zhang, Diana S-L Chow
Mycophenolate mofetil (MMF), an ester prodrug of mycophenolic acid (MPA), is widely used as a maintenance immunosuppressive regimen in solid organ transplant patients. It is increasingly used for the prophylaxis and treatment of graft-versus-host disease (GVHD) in hematopoietic stem cell transplantation (HSCT) patients. MPA displays extensive binding to serum albumin and glucuronidation to the inactive MPA-7-O-glucuronide (MPAG). Here, we review and discuss the pertinent information regarding the clinical pharmacokinetics of MPA in HSCT patients...
September 27, 2016: European Journal of Drug Metabolism and Pharmacokinetics
Francisco Bautista, Jasper Van der Lugt, Pamela R Kearns, Francis J Mussai, C Michel Zwaan, Lucas Moreno
Survival rates in pediatric leukemia have greatly improved in the last decades but still a substantial number of patients will relapse and die. New agents are necessary to overcome the limitations of conventional chemotherapy and hematopoietic stem cell transplantation and to reduce their undesirable long-term toxicities. The identification of driving molecular alterations of leukemogenesis in subsets of patients will allow the incorporation of new-targeted therapies. Areas covered: In this article the authors present a detailed review of the most recent advances in targeted therapies for pediatric leukemias...
November 2016: Expert Opinion on Drug Discovery
Pooja Khandelwal, Chie Emoto, Tsuyoshi Fukuda, Alexander A Vinks, Lisa Neumeier, Christopher E Dandoy, Javier El-Bietar, Sharat Chandra, Stella M Davies, Jacob J Bleesing, Michael B Jordan, Parinda A Mehta, Sonata Jodele, Michael S Grimley, Ashish Kumar, Kasiani C Myers, Rebecca A Marsh
We describe a single-center prospective study of alemtuzumab as a second-line agent for steroid-refractory (SR) acute graft-versus-host disease (aGVHD) in pediatric and young adult allogeneic hematopoietic stem cell transplant recipients. Alemtuzumab was administered for grades II to IV aGVHD if patients did not improve within 5 days or worsened within 48 hours after corticosteroids. Interim analyses of alemtuzumab levels and response were performed after every 5 patients enrolled, resulting in 3 dosing cohorts, as follows: (1) ...
September 21, 2016: Biology of Blood and Marrow Transplantation
Margaret Bevans, Areej El-Jawahri, D Kathryn Tierney, Lori Wiener, William A Wood, Flora Hoodin, Erin E Kent, Paul B Jacobsen, Stephanie J Lee, Matt Hsieh, Ellen M Denzen, Karen L Syrjala
In 2016, the National Institutes of Health convened an expert consensus panel to address the research needs and best practices for patient-centered late effects of hematopoietic stem cell transplantation (HCT). The panel used a scoping review approach to efficiently survey the large body of literature in adult and pediatric HCT survivors over 1 year after transplantation. The goals of this paper are to (1) summarize the current literature describing patient-centered outcomes in survivors, including the various dimensions of health-related quality of life affected by HCT, and describe interventions tested to improve these outcomes; (2) highlight areas with sufficient evidence allowing for integration into standard practice; (3) address methodological issues that restrict progress in this field; (4) identify major gaps to guide future research; and (5) specify priority research recommendations...
September 19, 2016: Biology of Blood and Marrow Transplantation
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
Antonio Juan, Laura Alonso, Teresa Olivé, Alexandra Navarro, Elena Sulleiro, José Sánchez de Toledo, Cristina Díaz de Heredia
Acanthamoeba infections are rare and mostly occur in immunocompromised patients. Most of the reported cases after stem-cell transplantation have been diagnosed postmortem. We present the case of a 3-year-old boy with chronic graft versus host disease post-hematopoietic transplantation who was successfully treated for Acanthamoeba.
September 12, 2016: Pediatric Infectious Disease Journal
Sonia P Bohórquez, Juliana Díaz, Claudia M Rincón, Marcela Estupiñán, Mauricio Chaparro, Ana María Low-Calle, Jaime E Castellanos
INTRODUCTION: Hematopoietic stem cell transplantation in pediatric patients is an alternative treatment for different diseases. The conditioning regimen for transplant predisposes recipients to the development of infections. Viral infections by herpes simplex virus 1 (HSV-1), herpes simplex virus 2 (HSV-2), human cytomegalovirus (CMV), and Epstein-Barr virus (EBV), are the most common, and the leading cause of morbidity and mortality among these patients. These viruses lie dormant in various cell types and the reactivation of latent infections may lead to asymptomatic viral shedding in saliva...
2016: Biomédica: Revista del Instituto Nacional de Salud
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
Daniel Stark, Abby R Rosenberg, Donna Johnston, Kristin Knight, Lizzie Caperon, Elizabeth Uleryk, A Lindsay Frazier, Lillian Sung
Purpose: We identified studies that described use of any patient-reported outcome scale for hearing loss or tinnitus among children and adolescents and young adults (AYAs) with cancer or hematopoietic stem cell transplantation (HSCT) recipients. Method: In this systematic review, we performed electronic searches of OvidSP MEDLINE, EMBASE, and PsycINFO to August 2015. We included studies if they used any patient-reported scale of hearing loss or tinnitus among children and AYAs with cancer or HSCT recipients...
September 7, 2016: Journal of Speech, Language, and Hearing Research: JSLHR
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
Adlette Inati, Mario Kahale, Nada Sbeiti, Maria Domenica Cappellini, Ali T Taher, Suzanne Koussa, Therese A Nasr, Khaled M Musallam, Hussein A Abbas, John B Porter
BACKGROUND: Iron overload is well documented in patients with β-thalassemia major, and patients who have undergone hematopoietic stem cell transplantation (HSCT) remain at risk as a result of pre- and immediate post-HSCT transfusions. PROCEDURE: This is a prospective, randomized, 1-year clinical trial that compares the efficacy and safety of the once-daily oral iron chelator deferasirox versus phlebotomy for the treatment of iron overload in children with β-thalassemia major following HSCT...
August 31, 2016: Pediatric Blood & Cancer
Kristen E McCabe, Allison J Pollock, Jennifer L Rehm, Kenneth B DeSantes
The mainstay of treatment for type 1 diabetes (T1D) is exogenous insulin. Here, we report a case in which exogenous insulin requirements were eliminated after an allogeneic hematopoietic stem cell transplant for aplastic anemia in a pediatric patient recently diagnosed with T1D, and explore the validity of this approach compared with current treatments.
August 30, 2016: Pediatric Diabetes
Thomas Lehrnbecher, Paula D Robinson, Brian T Fisher, Elio Castagnola, Andreas H Groll, William J Steinbach, Theoklis E Zaoutis, Zelalem F Negeri, Joseph Beyene, Bob Phillips, Lillian Sung
We systematically reviewed and analyzed the available data for galactomannan (GM), β-D-glucan (BG), and polymerase chain reaction (PCR)-based assays to detect invasive fungal disease (IFD) in patients with pediatric cancer or undergoing hematopoietic stem cell transplantation when used as screening tools during immunosuppression or as diagnostic tests in patients presenting with symptoms such as fever during neutropenia (FN). Of 1532 studies screened, 25 studies reported on GM (n = 19), BG (n = 3), and PCR (n = 11)...
August 27, 2016: Clinical Infectious Diseases: An Official Publication of the Infectious Diseases Society of America
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