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pediatric hematopoietic stem cell transplant

Maria Gabelli, Marco Zecca, Chiara Messina, Elisa Carraro, Barbara Buldini, Attilio Maria Rovelli, Franca Fagioli, Alice Bertaina, Edoardo Lanino, Claudio Favre, Marco Rabusin, Arcangelo Prete, Mimmo Ripaldi, Walter Barberi, Fulvio Porta, Maurizio Caniglia, Stella Santarone, Paolo D'Angelo, Giuseppe Basso, Franco Locatelli
Relapse of acute lymphoblastic leukemia (ALL) may occur in extramedullary sites, mainly central nervous system (CNS) and testis. Optimal post-remissional treatment for isolated extramedullary relapse (IEMR) is still controversial. We collected data of children treated with hematopoietic stem cell transplantation (HSCT) for ALL IEMR from 1990 to 2015 in Italy. Among 281 patients, 167 had a relapse confined to CNS, 73 to testis, 14 to mediastinum, and 27 to other organs. Ninety-seven patients underwent autologous HSCT, 79 received allogeneic HSCT from a matched family donor, 75 from a matched unrelated donor, and 30 from an HLA-haploidentical donor...
June 13, 2018: Bone Marrow Transplantation
Nicolas Boissel, André Baruchel
Adolescent and young adult (AYA) patients with acute lymphoblastic leukemia (ALL) are recognized as a unique population with specific characteristics and needs. In adolescents aged 15-20 years old, the use of full pediatric protocols is supported by many comparative studies of pediatric and adult cooperative groups. In young adults, growing evidence suggests that pediatric-inspired or even fully pediatric approaches may also dramatically improve outcomes, leading to long-term survival rates of almost 70%, despite diminishing indications of hematopoietic stem cell transplantation...
June 12, 2018: Blood
Matthew S Kelly, Lisa Spees, Richard Vinesett, Andre Stokhuyzen, Lauren McGill, Alan D Proia, Kirsten Jenkins, Mehreen Arshad, Patrick C Seed, Paul L Martin
BACKGROUND: Autopsy may confirm clinical diagnoses or identify conditions that were not suspected prior to a patient's death. Previous studies evaluating the utility of autopsy in hematopoietic stem cell transplant (HSCT) recipients yielded conflicting results. METHODS: We conducted a retrospective cohort study of children (<18 years of age) undergoing allogeneic HSCT at Duke University who died of any cause between January 1, 1995 and December 31, 2016. We evaluated associations between patient characteristics and autopsy performance using Chi-square or Fisher's exact tests...
June 9, 2018: Biology of Blood and Marrow Transplantation
Akihiro Tamura, Toshiaki Ishida, Atsuro Saito, Nobuyuki Yamamoto, Takehito Yokoi, Suguru Uemura, Nanako Nino, Takahiro Fujiwara, Teppei Tahara, Sayaka Nakamura, Aiko Kozaki, Kenji Kishimoto, Daiichiro Hasegawa, Yoshiyuki Kosaka
The dismal prognosis of pediatric acute myeloid leukemia (AML) relapsing after hematopoietic stem cell transplantation (HSCT) requires exploration of novel strategies to prevent relapse. Azacitidine (AZA) maintenance therapy could potentially reduce the recurrence rate post HSCT. Here, we presents the cases of three children with high-risk AML post HSCT who were treated with low-dose AZA maintenance therapy, demonstrating the feasibility of this therapy. Currently, all three are in complete remission for 13-41 months despite their high-risk characteristics...
June 12, 2018: Pediatric Blood & Cancer
Bo Kyung Kim, Kyung Taek Hong, Hyoung Jin Kang, Hong Yul An, Jung Yoon Choi, Che Ry Hong, Kyung Duk Park, Dong Soon Lee, Hee Young Shin
Epstein-Barr virus (EBV)-positive aggressive natural killer-cell leukemia (ANKL) is a rare malignancy of mature natural killer cells, with a very poor survival rate. Patients have a rapidly declining clinical course and a poor prognosis, with a median survival of only a few months. Herein, we describe a 16-year-old boy who was diagnosed with EBV-positive ANKL and successfully treated using combination chemotherapy and a subsequent allogeneic hematopoietic stem cell transplantation (alloHSCT). The patient is disease free 4 years and 9 months after alloHSCT...
June 8, 2018: Journal of Pediatric Hematology/oncology
Hélène Fe Gleitz, Ai Yin Liao, James R Cook, Samuel F Rowlston, Gabriella Ma Forte, Zelpha D'Souza, Claire O'Leary, Rebecca J Holley, Brian W Bigger
The pediatric lysosomal storage disorder mucopolysaccharidosis type II is caused by mutations in IDS, resulting in accumulation of heparan and dermatan sulfate, causing severe neurodegeneration, skeletal disease, and cardiorespiratory disease. Most patients manifest with cognitive symptoms, which cannot be treated with enzyme replacement therapy, as native IDS does not cross the blood-brain barrier. We tested a brain-targeted hematopoietic stem cell gene therapy approach using lentiviral IDS fused to ApoEII (IDS...
June 8, 2018: EMBO Molecular Medicine
Jacek Wachowiak, Alicja Chybicka, Jerzy R Kowalczyk, Mariusz Wysocki, Jolanta Goździk, Ewa Gorczyńska, Krzysztof Kałwak, Jan Styczyński, Katarzyna Drabko, Anna Pieczonka
The purpose of the survey was to evaluate the development and current use of hematopoietic stem cell transplantation (HSCT) in Poland between 1989-2016. The data for analysis (indication, number of performed HSCT, HSCT type, donor type, and stem cell source, year) have been collected annually using a standardized form. In Poland, between 1989-2016, the number of pediatric transplant beds grew from one to 40 and number and rate of transplants increased annually from 1/year (0.8/10 million) to 186/year (248/10 million)...
May 16, 2018: Transfusion and Apheresis Science
Trisha Bhat, Carrie C Coughlin
PURPOSE OF REVIEW: The pediatric transplant patient population is growing as the number of solid organ transplants and indications for hematopoietic stem cell transplant increase. Understanding cutaneous sequelae of pediatric transplant and treatment strategies to manage these outcomes is vital to the care of these patients. RECENT FINDINGS: Important work in the past year enhances our understanding of the cutaneous implications of pediatric transplantation, including further work in areas of malignancy, infection, and graft versus host disease as well as newly reported risks...
June 6, 2018: Current Opinion in Pediatrics
Nancy A Kernan, Paul G Richardson, Angela R Smith, Brandon M Triplett, Joseph H Antin, Leslie Lehmann, Yoav Messinger, Wei Liang, Robin Hume, William Tappe, Robert J Soiffer, Stephan A Grupp
BACKGROUND: Hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is a potentially fatal complication of conditioning for hematopoietic stem cell transplantation (HSCT) but can occur after nontransplant-associated chemotherapy. Following HSCT, VOD/SOS with multi-organ dysfunction (MOD) may be associated with >80% mortality. Defibrotide is approved to treat severe hepatic VOD/SOS post-HSCT in patients aged >1 month in the European Union and hepatic VOD/SOS with renal or pulmonary dysfunction post-HSCT in the United States...
June 6, 2018: Pediatric Blood & Cancer
Melissa Quinn, J T Fannin, Joseph Sciasci, Allison Bragg, Patrick K Campbell, Delia Carias, Kristine R Crews, David Gregornik, Sima Jeha, Gabriela Maron, Jennifer L Pauley, Hope D Swanson, Joshua Wolf, William Greene
BACKGROUND: Pneumocystis jirovecii pneumonia is a life-threatening opportunistic infection in children receiving immunosuppressive chemotherapy. Without prophylaxis, up to 25% of pediatric oncology patients receiving chemotherapy will develop Pneumocystis jirovecii pneumonia. Trimethoprim-sulfamethoxazole is the preferred agent for prophylaxis against Pneumocystis jirovecii pneumonia. Pentamidine may be an acceptable alternative for pediatric patients unable to tolerate trimethoprim-sulfamethoxazole...
June 4, 2018: Antimicrobial Agents and Chemotherapy
Santiago M C Lopez, Marian G Michaels, Michael Green
PURPOSE OF REVIEW: Adenoviruses (AdVs) infection is a self-limited disease in the majority of immunocompetent children and adults, but can cause disseminated and life-threatening illness in immunocompromised hosts. This article will discuss therapeutic strategies for AdV infection in the pediatrics transplant recipient. RECENT FINDINGS: Currently, there is no FDA approved antiviral therapy for AdV infection. Accordingly, the primary initial therapy would be decreasing immunosuppression, whenever possible...
May 24, 2018: Current Opinion in Organ Transplantation
Süreyya Savaşan
Acquired aplastic anemia (aAA) characterized by peripheral pancytopenia and bone marrow aplasia is a rare and serious disorder. Differential diagnosis includes constitutional bone marrow failure syndromes and myelodysplastic disorders. Autoimmune reaction to altered hematopoietic stem cells highlights the underlying mechanism. Matched related donor allogeneic hematopoietic stem cell transplantation is the ideal pediatric treatment; alternative approaches include immunosuppressive therapy and use of eltrombopag...
June 2018: Pediatric Clinics of North America
Matthew J Oberley, Paul S Gaynon, Deepa Bhojwani, Michael A Pulsipher, Rebecca A Gardner, Matthew C Hiemenz, Jianling Ji, Jennifer Han, Maurice R G O'Gorman, Alan S Wayne, Gordana Raca
A pediatric patient diagnosed initially with B-lymphoblastic leukemia (B-ALL) relapsed with lineage switch to acute myeloid leukemia (AML) after chimeric antigen receptor T-cell (CAR-T) therapy and hematopoietic stem cell transplant . A TCF3-ZNF384 fusion was identified at diagnosis, persisted through B-ALL relapse, and was also present in the AML relapse cell population. ZNF384-rearrangements define a molecular subtype of B-ALL characterized by a pro-B-cell immunophenotype; furthermore, ZNF384-rearrangements are prevalent in mixed-phenotype acute leukemias...
May 24, 2018: Pediatric Blood & Cancer
Takashi Mikami, Itaru Kato, Fumihito Nozaki, Katsutsugu Umeda, Tatsuya Kamitori, Keiji Tasaka, Hideto Ogata, Hidefumi Hiramatsu, Yoshiki Arakawa, Souichi Adachi
Compared to cerebral radiation-induced cavernous hemangiomas (RICHs), little is known about intraspinal RICHs. A 13-year-old male suddenly developed symptomatic spinal hemorrhage eight years after hematopoietic stem cell transplantation using a total body irradiation (TBI) based myeloablative regimen. A solitary small hemangioma was detected on follow-up T2 star weighted magnetic resonance imaging of the spine. His neurological symptoms gradually improved with supportive treatment and rehabilitation, although he experienced rebleeding 2 years later...
May 24, 2018: Pediatric Blood & Cancer
Kengo Inagaki, Chad Blackshear, Charlotte V Hobbs
BACKGROUND: Although the epidemiology of immunocompromising condition in children has evolved over time, updated epidemiology of pediatric pneumocystis infection in the US is not available. METHODS: We performed a retrospective analysis using the Kids' Inpatient Database, a nationally representative sample of US pediatric hospital discharges collected in 1997, 2000, 2003, 2006, 2009, and 2012. pneumocystis cases were identified using International Classification of Diseases, Ninth Revision, Clinical Modification code 136...
May 21, 2018: Pediatric Infectious Disease Journal
Kaiyue Peng, Xiaowen Qian, Zhiheng Huang, Junping Lu, Yuhuan Wang, Ying Zhou, Huijun Wang, Bingbing Wu, Ying Wang, Lingli Chen, Xiaowen Zhai, Ying Huang
Background: Hematopoietic stem cell transplantation is considered the only curative therapy for very early-onset inflammatory bowel disease with specific immune defects, such as interleukin-10 receptor deficiency. We performed reduced-intensity conditioning before umbilical cord blood transplantation in patients with interleukin-10 receptor-A deficiency. Methods: We enrolled 9 very early-onset inflammatory bowel disease patients with typical manifestations. We diagnosed the patients with interleukin-10 receptor-A deficiency by whole-exome sequencing...
May 18, 2018: Inflammatory Bowel Diseases
Dominder Kaur, Aneel A Ashrani, Rajiv Pruthi, Shakila P Khan, Kent Bailey, Vilmarie Rodriguez
BACKGROUND: Overall incidence of hemostatic complications in pediatric recipients of Hematopoietic Stem Cell Transplant (HSCT) is scarcely studied. This retrospective review explored the incidence and underlying risk factors of bleeding and thrombotic complications in children. PROCEDURE: Clinical characteristics, hemorrhagic events (HE), thrombotic events (TE) and follow up data were abstracted from medical records on patients aged <21 years undergoing HSCT during January 2000-June 2015...
April 26, 2018: Thrombosis Research
Gregory Wallace, Sonata Jodele, Kasiani C Myers, Christopher E Dandoy, Javier El-Bietar, Adam Nelson, Ashley Teusink-Cross, Pooja Khandelwal, Cynthia Taggart, Catherine M Gordon, Stella M Davies, Jonathan C Howell
BACKGROUND: Vitamin D (vitD) deficiency is prevalent among childhood hematopoietic stem cell transplantation (HSCT) recipients and associated with inferior transplant survival at 100 days after transplantation. Achieving and maintaining therapeutic vitD levels in HSCT recipients is extremely challenging in the first 3-6 months after transplant due to poor compliance in the setting of mucositis, and concomitant use of critical transplant drugs that interfere with vitD absorption. We sought to evaluate the safety and efficacy of a single, ultra-high dose of vitD given prior to childhood HSCT to maintain levels in a therapeutic range during the peri-transplantation period...
May 18, 2018: Biology of Blood and Marrow Transplantation
Ditte J A Løhmann, Peter H Asdahl, Jonas Abrahamsson, Shau-Yin Ha, Ólafur G Jónsson, Gertjan J L Kaspers, Minna Koskenvuo, Birgitte Lausen, Barbara De Moerloose, Josefine Palle, Bernward Zeller, Henrik Hasle
BACKGROUND: Children with acute myeloid leukemia (AML) treated similarly show different toxicity and leukemic responses. We investigated associations between neutrophil recovery time after the first induction course, infection and relapse in children treated according to NOPHO-AML 2004 and DB AML-01. PROCEDURE: Newly diagnosed patients with AML with bone marrow blast <5% between day 15 after the start of the treatment and the start of second induction course, and in complete remission after the second induction course were included (n = 279)...
May 21, 2018: Pediatric Blood & Cancer
Irena Sniecinski, Jerard Seghatchian
Clinical practice and related diagnostic, development and research [DDR] strategies in pediatric transfusion and transplantation cover a broad range of multidisciplinary studies, performed by many professionals involved in this most challenging clinical field [1]. This commentary on the current position and future perspectives in pediatric transfusion field is aimed to highlight major unresolved transfusion complications in pediatric patients, namely red blood cell and platelet alloimmunisation, and new ones such as nosocomial infection, thrombosis and multi-organ failure...
May 16, 2018: Transfusion and Apheresis Science
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