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Pediatric cancer, bone marrow transplant, pediatric blood diseases

Bronwen E Shaw, Ruta Brazauskas, Heather R Millard, Rachel Fonstad, Kathryn E Flynn, Amy Abernethy, Jenny Vogel, Charney Petroske, Deborah Mattila, Rebecca Drexler, Stephanie J Lee, Mary M Horowitz, J Douglas Rizzo
BACKGROUND: Allogeneic hematopoietic cell transplantation (HCT) cures many patients, but often with the risk of late effects and impaired quality of life. The value of quantifying patient-reported outcomes (PROs) is increasingly being recognized, but the routine collection of PROs is uncommon. This study evaluated the feasibility of prospective PRO collection by an outcome registry at multiple time points from unselected HCT patients undergoing transplantation at centers contributing clinical data to the Center for International Blood and Marrow Transplant Research (CIBMTR), and then it correlated the PRO data with clinical and demographic data...
December 1, 2017: Cancer
Christopher C Porter, Todd E Druley, Ayelet Erez, Roland P Kuiper, Kenan Onel, Joshua D Schiffman, Kami Wolfe Schneider, Sarah R Scollon, Hamish S Scott, Louise C Strong, Michael F Walsh, Kim E Nichols
Leukemia, the most common childhood cancer, has long been recognized to occasionally run in families. The first clues about the genetic mechanisms underlying familial leukemia emerged in 1990 when Li-Fraumeni syndrome was linked to TP53 mutations. Since this discovery, many other genes associated with hereditary predisposition to leukemia have been identified. Although several of these disorders also predispose individuals to solid tumors, certain conditions exist in which individuals are specifically at increased risk to develop myelodysplastic syndrome (MDS) and/or acute leukemia...
June 1, 2017: Clinical Cancer Research: An Official Journal of the American Association for Cancer Research
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...
January 2017: Bone Marrow Transplantation
Kimberly A Noonan, Ivan M Borrello
The clinical results achieved with immunotherapy in the past few years have now firmly established it within the cancer armamentarium. Our group has explored a novel approach to adoptive T-cell therapy utilizing marrow-infiltrating lymphocytes (MILs) initially developed with the concept of utilizing a population of T cells with a higher endogenous tumor specificity. Marrow-infiltrating lymphocytes are antigen-experienced T cells that home to and remain in the bone marrow (BM) because of the unique biology of the BM microenvironment...
November 2015: Cancer Journal
Heather Allewelt, Paul L Martin, Paul Szabolcs, Nelson Chao, Rebecca Buckley, Suhag Parikh
BACKGROUND: X-linked hyper-IgM syndrome (X-HIGM) due to mutations in the gene encoding CD40 ligand results in failure of Ig class switching and an increased propensity for recurrent sinopulmonary and other infections, and thus decreased life expectancy. Allogeneic hematopoietic stem cell transplantation (HSCT) is curative, but long-term follow-up data are limited. PROCEDURES: We conducted a retrospective analysis of seven patients who have undergone allogeneic HSCT for HIGM syndrome at Duke University Medical Center...
December 2015: Pediatric Blood & Cancer
Julia Alten, Wolfram Klapper, Ivo Leuschner, Cornelia Eckert, Rita Beier, Elisabeth Vallo, Martin Krause, Alexander Claviez, Simon Vieth, Kirsten Bleckmann, Anja Möricke, Martin Schrappe, Gunnar Cario
Histiocytic sarcoma (HS) is a rare disease with poor prognosis which may develop subsequent to acute lymphoblastic leukemia (ALL). Here we report two children treated within the AIEOP-BFM ALL 2009 trial: one patient succumbed to fulminant hemophagocytic lymphohistiocytosis triggered by HS during ALL maintenance therapy, the other patient had a smoldering course of HS for over 2 years, and subsequently died after allogeneic stem cell transplantation. In both cases, HS and ALL were clonally related and apparent return of minimal residual disease (MRD) was detected by qPCR in bone marrow...
September 2015: Pediatric Blood & Cancer
Laura Grein Cavalcanti, Karine Fátima Lyko, Renata Lins Fuentes Araújo, José Miguel Amenábar, Carmem Bonfim, Cassius Carvalho Torres-Pereira
BACKGROUND: Fanconi anaemia is a genetic disease characterized by congenital abnormalities, progressive bone marrow failure, and a higher predisposition of oral squamous cell carcinoma. The purpose of this study was to evaluate the prevalence of oral mucosa lesions in patients with Fanconi anaemia without hematopoietic stem cell transplantation (HSCT). PROCEDURE: Patients with Fanconi anaemia who had not undergone HSCT was cross-sectional evaluated for the presence of oral lesions...
June 2015: Pediatric Blood & Cancer
Javid Gaziev, Antonella Isgrò, Alessia Francesca Mozzi, Aurèlie Petain, Laurent Nguyen, Cristiano Ialongo, Vincenzo Dinallo, Pietro Sodani, Marco Marziali, Marco Andreani, Manuela Testi, Katia Paciaroni, Cristiano Gallucci, Gioia De Angelis, Cecilia Alfieri, Michela Ribersani, Guido Lucarelli
BACKGROUND: Busulfan (Bu) is an integral part of conditioning regimens for patients with sickle cell anemia (SCA) undergoing transplantation. Patients with SCA might predispose to transplant-related neurological and pulmonary toxicities due to pre-existing disease-related cerebrovascular and lung injury. Bu therapy appears to be an important contributing factor in this context. PROCEDURE: We studied the pharmacokinetics of intravenous Bu and clinical outcomes of 36 children with SCA undergoing bone marrow transplantation...
April 2015: Pediatric Blood & Cancer
Fausta Beneventi, Elena Locatelli, Giovanna Giorgiani, Marco Zecca, Tommaso Mina, Margherita Simonetta, Chiara Cavagnoli, Mara Albanese, Arsenio Spinillo
OBJECTIVE: To compare uterine and ovarian volumes and uterine artery (UA) Doppler blood flow among women who were treated with antineoplastic regimens when pediatric aged versus healthy controls. DESIGN: Case-control study. SETTING: Tertiary obstetric and gynecologic center. PATIENT(S): One hundred twenty-seven women who were treated for childhood cancer with bone marrow transplantation (BMT) and∖or chemotherapy and total body irradiation (TBI) and 64 age-matched healthy controls...
February 2015: Fertility and Sterility
Yael Dinur-Schejter, Aviva C Krauss, Odeya Erlich, Natan Gorelik, Anat Yahel, Iris Porat, Michael Weintraub, Jerry Stein, Irina Zaidman, Polina Stepensky
BACKGROUND: Treosulfan (treo) is an alkylating agent with a low acute toxicity profile that is increasingly used in hematopoietic stem cell transplantation, predominantly in non-malignant diseases. Treosulfan is usually combined with additional agents, but there is scant evidence to allow comparison between different conditioning protocols using treosulfan. We present the experience of three pediatric transplantation centers in Israel using different treosulfan-based conditioning regimens...
February 2015: Pediatric Blood & Cancer
Ana L Basquiera, Silvia Pizzi, Agustín González Correas, Pablo G Longo, Wanda C Goldman, María V Prates, Sandra Formisano, Gustavo Kusminisky, Leonardo Feldman, Adriana R Berretta, Juan J García, Raquel Staciuk
BACKGROUND: Allogeneic hematopoietic stem cell transplantation (AHSCT) represents the only curative treatment for the majority of pediatric patients with Myelodysplastic Syndrome (MDS). We aimed to evaluate overall survival (OS), disease-free survival (DFS), non-relapse mortality (NRM) and relapse incidence in children who underwent AHSCT for MDS in six institutions from Argentina. PROCEDURE: A retrospective analysis of 54 AHSCT was carried out in 52 patients (mean age: 9 years; range: 2-19; 35 males)...
January 2015: Pediatric Blood & Cancer
Michelle Kolb, Monica Bhatia, Gay-Gaddi Madina, Prakash Satwani
Steroid-refractory acute graft versus host disease (SR-aGVHD) is associated with poor prognosis. A three year old male with severe sickle cell disease (SCD) received a 7/8 HLA-matched unrelated bone marrow transplant. He developed grade III acute graft versus host disease (aGVHD) of the gut, which was refractory to methylprednisolone and five other treatment regimens. Due to very high serum Interleukin-6 (IL-6) levels, he received salvage therapy with tocilizumab with resolution of aGVHD. It may be reasonable to further prospectively study routine monitoring of IL-6 levels and the use of tocilizumab in such patients...
February 2015: Pediatric Blood & Cancer
Kaljit S Bhuller, Manraj K S Heran, John K M Wu, S Rod Rassekh
Acute graft versus host disease (GVHD) is a significant complication of bone marrow transplantation with approximately half of patients being refractory to steroids. There are numerous second-line systemic immunosuppressive treatments but the overall prognosis is poor and these therapies are associated with high mortality due to infection. An alternative approach to systemic treatment for GVHD is targeted delivery of immunosuppression. We present two pediatric cases with steroid-refractory gastrointestinal GVHD who clinically responded to intra-arterial steroid administration...
December 2014: Pediatric Blood & Cancer
Isaac See, Martha Iwamoto, Kathy Allen-Bridson, Teresa Horan, Shelley S Magill, Nicola D Thompson
OBJECTIVE: To assess challenges to implementation of a new National Healthcare Safety Network (NHSN) surveillance definition, mucosal barrier injury laboratory-confirmed bloodstream infection (MBI-LCBI). DESIGN: Multicenter field test. SETTING: Selected locations of acute care hospitals participating in NHSN central line-associated bloodstream infection (CLABSI) surveillance. METHODS: Hospital staff augmented their CLABSI surveillance for 2 months to incorporate MBI-LCBI: a primary bloodstream infection due to a selected group of organisms in patients with either neutropenia or an allogeneic hematopoietic stem cell transplant with gastrointestinal graft-versus-host disease or diarrhea...
August 2013: Infection Control and Hospital Epidemiology
Johann K Hitzler, Wensheng He, John Doyle, Mitchell Cairo, Bruce M Camitta, Ka Wah Chan, Miguel A Diaz Perez, Christopher Fraser, Thomas G Gross, John T Horan, Alana A Kennedy-Nasser, Carrie Kitko, Joanne Kurtzberg, Leslie Lehmann, Tracey O'Brien, Michael A Pulsipher, Franklin O Smith, Mei-Jie Zhang, Mary Eapen, Paul A Carpenter
Data on outcomes of allogeneic transplantation in children with Down syndrome and acute myelogenous leukemia (DS-AML) are scarce and conflicting. Early reports stress treatment-related mortality as the main barrier; a recent case series points to posttransplantation relapse. We reviewed outcome data for 28 patients with DS-AML reported to the Center for International Blood and Marrow Transplant Research between 2000 and 2009 and performed a first matched-pair analysis of 21 patients with DS-AML and 80 non-DS AML controls...
June 2013: Biology of Blood and Marrow Transplantation
Merlin G Butler, Jay E Menitove
BACKGROUND: Umbilical cord blood is a potential vast source of primitive hematopoietic stem and progenitor cells available for clinical application to reconstitute the hematopoietic system and/or restore immunological function in affected individuals requiring treatment. Cord blood can be used as an alternative source for bone marrow transplantation and its use is developing into a new field of treatment for pediatric and adult patients presenting with hematological disorders, immunological defects and specific genetic diseases...
August 2011: Journal of Assisted Reproduction and Genetics
Ines Bojanić, Branka Golubić Cepulić, Sanja Mazić
Allogeneic hematopoietic progenitor cell (HPC) transplantation is an established therapy for many hematologic disorders. HPCs may be collected from bone marrow, peripheral blood, or umbilical cord blood. In order to minimize the risk for healthy HPC donors, thorough investigation is required before donation. The donor work-up should include medical history, physical examination, ECG, chest x-ray, blood count, coagulation screening, and testing for infectious disease markers. Donors should be fully informed on the donation procedure and sign an informed consent for donation...
June 2009: Acta Medica Croatica: C̆asopis Hravatske Akademije Medicinskih Znanosti
Michael A Pulsipher, Kenneth M Boucher, Donna Wall, Haydar Frangoul, Michel Duval, Rakesh K Goyal, Peter J Shaw, Ann E Haight, Michael Grimley, Stephan A Grupp, Morris Kletzel, Richard Kadota
The role of reduced-intensity conditioning (RIC) regimens in pediatric cancer treatment is unclear. To define the efficacy of a busulfan/fludarabine/antithymocyte globulin RIC regimen in pediatric patients ineligible for myeloablative transplantation, we completed a trial at 23 institutions in the Pediatric Blood and Marrow Transplant Consortium. Forty-seven patients with hematologic malignancies were enrolled. Sustained engraftment occurred in 98%, 89%, and 90%, and full donor chimerism was achieved in 88%, 76%, and 78% of evaluable related bone marrow/peripheral blood stem cells (BM/PBSCs), unrelated BM/PBSCs, and unrelated cord blood recipients...
August 13, 2009: Blood
Hisaki Fujii, Geoff Cuvelier, Kevin She, Soudabeh Aslanian, Hiromi Shimizu, Amina Kariminia, Mark Krailo, Zhengjia Chen, Rob McMaster, Axel Bergman, Frederick Goldman, Stephen A Grupp, Donna A Wall, Andrew L Gilman, Kirk R Schultz
Numerous chronic graft-versus-host disease (cGVHD) biomarkers have been identified in limited, single-institution studies without validation. We hypothesized that plasma-derived biomarkers could diagnose, classify, and evaluate response in children with cGVHD. We performed a concomitant analysis of a number of known and predicted peripheral blood cGVHD biomarkers from a Children's Oncology Group (COG) phase 3 cGVHD therapeutic trial. A total of 52 newly diagnosed patients with extensive cGVHD were compared for time of onset after blood and marrow transplantation (BMT) (early, 3-8 months; late, > or = 9 months) with 28 time-matched controls with no cGVHD (early, 6 months after BMT; late, 12 months after BMT)...
March 15, 2008: Blood
Bassem I Razzouk, Elihu Estey, Stanley Pounds, Shelly Lensing, Sherry Pierce, Mark Brandt, Jeffrey E Rubnitz, Raul C Ribeiro, Michael Rytting, Ching-Hon Pui, Hagop Kantarjian, Sima Jeha
BACKGROUND: The prognostic significance of age among pediatric patients with acute myeloid leukemia (AML) was investigated. METHODS: The authors reviewed the outcome of 424 patients who were <or=21 years of age at the time of diagnosis of AML (excluding acute promyelocytic leukemia) between 1983 and 2002 at St. Jude Children's Research Hospital (n=288) or the M. D. Anderson Cancer Center (n=136). Two treatment eras (1983-1989 and 1990-2002) were examined because of the greater intensity of treatment during the recent era...
June 1, 2006: Cancer
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