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Long term follow up post blood and marrow transplant

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https://www.readbyqxmd.com/read/28392333/outcome-of-hematopoietic-cell-transplantation-for-dna-double-strand-breakage-repair-disorders
#1
James Slack, Michael H Albert, Dmitry Balashov, Bernd H Belohradsky, Alice Bertaina, Jack Bleesing, Claire Booth, Jochen Büchner, Rebecca H Buckley, Marie Ouachée-Chardin, Elena Deripapa, Katarzyna Drabko, Mary Eapen, Tobias Feuchtinger, Andrea Finocchi, H Bobby Gaspar, Sujal Ghosh, Alfred Gillio, Luis I Gonzalez-Granado, Eyal Grunebaum, Tayfun Güngör, Carsten Heilmann, Merja Helminen, Kohei Higuchi, Kohsuke Imai, Krzysztof Kalwak, Nubuo Kanazawa, Gülsün Karasu, Zeynep Y Kucuk, Alexandra Laberko, Andrzej Lange, Nizar Mahlaoui, Roland Meisel, D Moshous, Hideki Muramatsu, Suhag Parikh, Srdjan Pasic, Irene Schmid, Catharina Schuetz, Ansgar Schulz, Kirk R Schultz, Peter J Shaw, Mary A Slatter, Karl-Walter Sykora, Shinobu Tamura, Mervi Taskinen, Angela Wawer, Beata Wolska-Kus Nierz, Morton J Cowan, Alain Fischer, Andrew R Gennery
BACKGROUND: Rare DNA breakage-repair disorders predispose to infection and lympho-reticular malignancies. Hematopoietic cell transplantation (HCT) is curative but co-administered chemo- or radio-therapy is damaging due to systemic radio-sensitivity. We collected HCT outcome data for Nijmegen Breakage syndrome (NBS), DNA ligase IV deficiency (LIG4), Cernunnos-XLF deficiency and ataxia-telangiectasia. METHODS: Data from 38 centres worldwide, including indication, donor, conditioning regimen, graft-versus-host disease (GvHD) and outcome were analyzed...
April 6, 2017: Journal of Allergy and Clinical Immunology
https://www.readbyqxmd.com/read/28331055/reticular-dysgenesis-international-survey-on-clinical-presentation-transplantation-and-outcome
#2
Manfred Hoenig, Chantal Lagresle-Peyrou, Ulrich Pannicke, Luigi D Notarangelo, Fulvio Porta, Andrew R Gennery, Mary Slatter, Morton J Cowan, Polina Stepensky, Hamoud Al-Mousa, Daifulah Al-Zahrani, Sung-Yun Pai, Waleed Al Herz, Hubert B Gaspar, Paul Veys, Koichi Oshima, Kohsuke Imai, Hiromasa Yabe, Lenora M Noroski, Nico M Wulffraat, Karl-Walter Sykora, Pere Soler-Palacin, Hideki Muramatsu, Mariam Al Hilali, Despina Moshous, Klaus-Michael Debatin, Catharina Schuetz, Eva-Maria Jacobsen, Ansgar S Schulz, Klaus Schwarz, Alain Fischer, Wilhelm Friedrich, Marina Cavazzana
Reticular Dysgenesis (RD) is a rare congenital disorder defined clinically by the combination of severe combined immunodeficiency (SCID), agranulocytosis and sensorineural deafness. Mutations in the gene encoding Adenylate Kinase 2 (AK2) were identified to cause the disorder. Hematopoietic stem cell transplantation (HSCT) is the only option to cure this otherwise fatal disease. Retrospective data on clinical presentation, genetics and outcome of HSCT were collected from centers in Europe, Asia and North America for a total of 32 patients born between 1982 and 2011...
March 22, 2017: Blood
https://www.readbyqxmd.com/read/28329410/long-term-outcome-after-a-treosulfan-based-conditioning-regimen-for-patients-with-acute-myeloid-leukemia-a-report-from-the-acute-leukemia-working-party-of-the-european-society-for-blood-and-marrow-transplantation
#3
Arnon Nagler, Myriam Labopin, Dietrich Beelen, Fabio Ciceri, Liisa Volin, Avichai Shimoni, Roberto Foá, Noel Milpied, Jacopo Peccatori, Emmanuelle Polge, Audrey Mailhol, Mohamad Mohty, Bipin N Savani
BACKGROUND: Allogeneic hematopoietic cell transplantation (HCT) is a curative therapy for patients with acute myeloid leukemia (AML). However, post-HCT relapse and regimen-related toxicity remain significant barriers to long-term survival. In recent years, new conditioning regimens have been explored to improve transplantation outcomes in patients with AML. Treosulfan combines a potent immunosuppressive and antileukemic effect with a low toxicity profile. METHODS: To investigate the role of treosulfan-based conditioning, the European Society for Blood and Marrow Transplantation Acute Leukemia Working Party performed a registry analysis of 520 adult patients with AML who received treosulfan-based conditioning and underwent HCT between 2000 and 2012, including 225 patients in first complete remission, 107 in second or later complete remission, and 188 with active/advanced disease 188 (88 with primary refractory disease)...
March 22, 2017: Cancer
https://www.readbyqxmd.com/read/28281429/single-centre-experience-of-allogeneic-haemopoietic-stem-cell-transplant-in-paediatric-patients-in-cape-town-south-africa
#4
A Van Eyssen, N Novitsky, P De Wit, T Schlaphoff, V Thomas, D Pillay, M Hendricks, A Davidson
BACKGROUND: Allogeneic haemopoietic stem cell transplant (Allo-HSCT) is a specialised and costly intervention, associated with significant morbidity and mortality. It is used to treat a broad range of paediatric conditions. South Africa (SA) is an upper middle-income country with limitations on healthcare spending. The role of paediatric Allo-HSCT in this setting is reviewed. OBJECTIVES: To review paediatric patients who underwent Allo-HSCT at the Groote Schuur Hospital/University of Cape Town Private Academic Hospital transplant unit in Cape Town, South Africa, and received post-transplant care at Red Cross War Memorial Children's Hospital, over the period January 2006 - December 2014 in respect of indications for the transplant, donor sources, conditioning regimens, treatment-related morbidity and overall survival (OS)...
February 27, 2017: South African Medical Journal, Suid-Afrikaanse Tydskrif Vir Geneeskunde
https://www.readbyqxmd.com/read/28197149/treatment-of-infantile-inflammatory-bowel-disease-and-autoimmunity-by-allogeneic-stem-cell-transplantation-in-lps-responsive-beige-like-anchor-deficiency
#5
Shahrzad Bakhtiar, Laura Gámez-Díaz, Andrea Jarisch, Jan Soerensen, Bodo Grimbacher, Bernd Belohradsky, Klaus-Michael Keller, Christoph Rietschel, Thomas Klingebiel, Sibylle Koletzko, Michael H Albert, Peter Bader
Inflammatory bowel disease (IBD) in young children can be a clinical manifestation of various primary immunodeficiency syndromes. Poor clinical outcome is associated with poor quality of life and high morbidity from the complications of prolonged immunosuppressive treatment and malabsorption. In 2012, mutations in the lipopolysaccharide-responsive beige-like anchor (LRBA) gene were identified as the cause of an autoimmunity and immunodeficiency syndrome. Since then, several LRBA-deficient patients have been reported with a broad spectrum of clinical manifestations without reliable predictive prognostic markers...
2017: Frontiers in Immunology
https://www.readbyqxmd.com/read/28068510/current-knowledge-and-priorities-for-future-research-in-late-effects-after-hematopoietic-stem-cell-transplantation-hct-for-severe-combined-immunodeficiency-patients-a-consensus-statement-from-the-second-pediatric-blood-and-marrow-transplant-consortium-international
#6
REVIEW
Jennifer Heimall, Jennifer Puck, Rebecca Buckley, Thomas A Fleisher, Andrew R Gennery, Benedicte Neven, Mary Slatter, Elie Haddad, Luigi D Notarangelo, K Scott Baker, Andrew C Dietz, Christine Duncan, Michael A Pulsipher, Mort J Cowan
Severe combined immunodeficiency (SCID) is 1 of the most common indications for pediatric hematopoietic cell transplantation (HCT) in patients with primary immunodeficiency. Historically, SCID was diagnosed in infants who presented with opportunistic infections within the first year of life. With newborn screening (NBS) for SCID in most of the United States, the majority of infants with SCID are now diagnosed and treated in the first 3.5 months of life; however, in the rest of the world, the lack of NBS means that most infants with SCID still present with infections...
March 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/27821187/long-term-survival-and-late-events-after-allogeneic-stem-cell-transplantation-from-hla-matched-siblings-for-acute-myeloid-leukemia-with-myeloablative-compared-to-reduced-intensity-conditioning-a-report-on-behalf-of-the-acute-leukemia-working-party-of-european
#7
Avichai Shimoni, Myriam Labopin, Bipin Savani, Liisa Volin, Gerhard Ehninger, Jurgen Kuball, Donald Bunjes, Nicolaas Schaap, Stephane Vigouroux, Andrea Bacigalupo, Hendrik Veelken, Jorge Sierra, Matthias Eder, Dietger Niederwieser, Mohamad Mohty, Arnon Nagler
BACKGROUND: Myeloablative (MAC) and reduced-intensity conditioning (RIC) are established approaches for allogeneic stem cell transplantation (SCT) in acute myeloid leukemia (AML). Most deaths after MAC occur within the first 2 years after SCT, while patients surviving leukemia-free for 2 years can expect a favorable long-term outcome. However, there is paucity of data on the long-term outcome (beyond 10 years) and the pattern of late events following RIC due to the relative recent introduction of this approach...
November 8, 2016: Journal of Hematology & Oncology
https://www.readbyqxmd.com/read/27264632/late-effects-after-umbilical-cord-blood-transplantation-in-very-young-children-after-busulfan-based-myeloablative-conditioning
#8
Heather Allewelt, Jill El-Khorazaty, Adam Mendizabal, Mahsa Taskindoust, Paul L Martin, Vinod Prasad, Kristin Page, Jean Sanders, Joanne Kurtzberg
Infants and young children who undergo allogeneic cord blood transplantation (CBT) are at increased risk for late effects because of exposure of developing organs to chemotherapy and radiation therapy typically used in transplant conditioning regimens. Busulfan (Bu)-based myeloablative regimens were developed to eliminate radiation exposure in these young children with the hope that late effects would be minimized. We now describe the late effects in 102 consecutive patients surviving a minimum of 5 years (median follow-up, 12...
September 2016: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/27164064/long-term-follow-up-after-reduced-intensity-conditioning-and-stem-cell-transplantation-for-childhood-nonmalignant-disorders
#9
Lisa M Madden, Robert J Hayashi, Ka Wah Chan, Michael A Pulsipher, Dorothea Douglas, Gregory A Hale, Sonali Chaudhury, Paul Haut, Kimberly A Kasow, Andrew L Gilman, Lisa M Murray, Shalini Shenoy
Reduced-intensity conditioning (RIC) before hematopoietic stem cell transplantation (HCT) in children could result in fewer complications during follow-up compared with myeloablative regimens. Hence, many RIC regimens are under investigation, but long-term follow-up is essential. We describe late follow-up beyond 2 years post-HCT in 43 children with nonmalignant disorders who underwent related or unrelated donor (56%) HCT on a multicenter study using a RIC regimen (alemtuzumab, fludarabine, and melphalan) followed by bone marrow (n = 30), peripheral blood (n = 3), or umbilical cord blood (n = 10) HCT for immune dysfunction, bone marrow failure, metabolic disorders, or hemoglobinopathy...
August 2016: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/27000734/autologous-stem-cell-transplantation-versus-alternative-allogeneic-donor-transplants-in-adult-acute-leukemias
#10
REVIEW
Norbert Claude Gorin
The availability of alternative sources of stem cells including most recently T-replete haploidentical marrow or peripheral blood, and the increasing use of reduced-intensity conditioning (RIC), renders feasible an allogeneic transplant to almost all patients with acute leukemia up to 70 years of age. Autologous stem cell transplantation (ASCT) for consolidation of complete remission (CR), however, offers in some circumstances an alternative option. Although associated with a higher relapse rate, autologous transplant benefits from a lower non-relapse mortality, the absence of graft-versus-host disease (GVHD), and a better quality of life for long-term survivors...
April 2016: Seminars in Hematology
https://www.readbyqxmd.com/read/26746819/what-they-want-inclusion-of-blood-and-marrow-transplanation-survivor-preference-in-the-development-of-models-of-care-for-long-term-health-in-sydney-australia
#11
Gemma Dyer, Nicole Gilroy, Louisa Brown, Megan Hogg, Lisa Brice, Masura Kabir, Matt Greenwood, Stephen R Larsen, John Moore, Mark Hertzberg, John Kwan, Gillian Huang, Jeff Tan, Christopher Ward, Ian Kerridge
Four hundred forty-one adult allogeneic blood and marrow transplantation (BMT) survivors participated in a cross-sectional survey to assess long-term follow-up (LTFU) model of care preference. Survey instruments included the Sydney Post BMT Survey, Functional Assessment of Cancer Therapy-BMT, Depression Anxiety Stress Scales 21, the Chronic GVHD Activity Assessment-Patient Self Report (Form B), the Lee Chronic GVHD Symptom Scale and the Post-Traumatic Growth Inventory. We found most BMT survivors (74%) would prefer LTFU with their transplantation physicians alone or in combination with transplantation center-linked services (satellite clinics or telemedicine) Over one-quarter indicated a preference for receiving comprehensive post-transplantation care in a "satellite" clinic staffed by their BMT team situated closer to their place of residence, with higher income, higher educational level, and sexual morbidity being significant social factors influencing this preference...
April 2016: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/26291959/hematopoietic-stem-cell-transplantation-for-cd40-ligand-deficiency-single-institution-experience
#12
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
https://www.readbyqxmd.com/read/24999225/lost-in-transition-the-essential-need-for-long-term-follow-up-clinic-for-blood-and-marrow-transplantation-survivors
#13
REVIEW
Shahrukh Hashmi, Paul Carpenter, Nandita Khera, André Tichelli, Bipin N Savani
Because of expanding indications and improvements in supportive care, the utilization of blood and marrow cell transplantation (BMT) to treat various conditions is increasing exponentially, and currently more than 60,000 BMTs are performed annually worldwide. By the year 2030, it is projected that the number of BMT survivors will increase 5-fold, potentially resulting in one half of a million survivors in the United States alone. As the majority of survivors now live beyond the first 2 years after BMT, they are prone to a unique set of complications and late effects...
February 2015: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/24464145/long-term-outcome-of-hla-haploidentical-hematopoietic-sct-without-in-vitro-t-cell-depletion-for-adult-severe-aplastic-anemia-after-modified-conditioning-and-supportive-therapy
#14
L Gao, Y Li, Y Zhang, X Chen, L Gao, C Zhang, Y Liu, P Kong, Q Wang, Y Su, C Wang, S Wang, B Li, A Sun, X Du, D Zeng, J Li, H Liu, X Zhang
HLA-haploidentical hematopoietic SCT (HSCT) is an option for severe aplastic anemia (SAA) patients. Here, we evaluated the outcomes of 26 adult-SAA patients who received HLA-haploidentical HSCT in five transplant centers in southwestern China. Most of the patients in this study failed prior therapy and were transfused heavily before the transplantation. The patients received fludarabine+cyclophosphamide+antithymocyte globulin as conditioning regimens and then unmanipulated peripheral blood plus marrow transplantation...
April 2014: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/24441288/long-term-follow-up-of-imatinib-in-pediatric-philadelphia-chromosome-positive-acute-lymphoblastic-leukemia-children-s-oncology-group-study-aall0031
#15
K R Schultz, A Carroll, N A Heerema, W P Bowman, A Aledo, W B Slayton, H Sather, M Devidas, H W Zheng, S M Davies, P S Gaynon, M Trigg, R Rutledge, D Jorstad, N Winick, M J Borowitz, S P Hunger, W L Carroll, B Camitta
We previously reported preliminary findings that post induction imatinib mesylate (340 mg/m(2)/day), in combination with intensive chemotherapy, resulted in outcomes similar to blood and marrow transplant (BMT) for pediatric patients with Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL). We now report 5-year outcomes of imatinib plus intensive chemotherapy in 91 children (1-21 years) with and without allogeneic BMT (N=91). We explore the impacts of additional chromosomal abnormalities and minimal residual disease (MRD) by flow cytometry on outcomes...
July 2014: Leukemia: Official Journal of the Leukemia Society of America, Leukemia Research Fund, U.K
https://www.readbyqxmd.com/read/24389309/allogeneic-hematopoietic-stem-cell-transplantation-in-patients-with-polycythemia-vera-or-essential-thrombocythemia-transformed-to-myelofibrosis-or-acute-myeloid-leukemia-a-report-from-the-mpn-subcommittee-of-the-chronic-malignancies-working-party-of-the-european
#16
Federico Lussana, Alessandro Rambaldi, Maria Chiara Finazzi, Anja van Biezen, Marijke Scholten, Elena Oldani, Alessandra Carobbio, Simona Iacobelli, Jurgen Finke, Arnon Nagler, Liisa Volin, Thierry Lamy, Renate Arnold, Mohamad Mohty, Mauricette Michallet, Theo de Witte, Eduardo Olavarria, Nicolaus Kröger
The clinical course of polycythemia vera and essential thrombocythemia is potentially associated with long-term severe complications, such as evolution to myelofibrosis or acute myeloid leukemia. Allogeneic stem cell transplantation is currently the only potentially curative treatment for advanced polycythemia vera or essential thrombocythemia. We analyzed 250 consecutive patients with an initial diagnosis of polycythemia vera (n=120) or essential thrombocythemia (n=130), who underwent transplantation due to progression to myelofibrosis (n=193) or acute myeloid leukemia (n=57) and who were reported to the European Group for Blood and Marrow Transplantation registry between 1994 and 2010...
May 2014: Haematologica
https://www.readbyqxmd.com/read/24141008/improved-survival-with-ursodeoxycholic-acid-prophylaxis-in-allogeneic-stem-cell-transplantation-long-term-follow-up-of-a-randomized-study
#17
RANDOMIZED CONTROLLED TRIAL
Tapani Ruutu, Eeva Juvonen, Mats Remberger, Kari Remes, Liisa Volin, Jonas Mattsson, Anne Nihtinen, Hans Hägglund, Olle Ringdén
We report the long-term results of a prospective randomized study on the use of ursodeoxycholic acid (UDCA) for prevention of hepatic complications after allogeneic stem cell transplantation. Two hundred forty-two patients, 232 with malignant disease, were randomized to receive (n = 123) or not to receive (n = 119) UDCA from the beginning of the conditioning until 90 days post-transplantation. The results were reported after 1-year follow-up. UDCA administration reduced significantly the proportion of patients developing high serum bilirubin levels as well as the incidence of severe acute graft-versus-host disease (GVHD), liver GVHD, and intestinal GVHD...
January 2014: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/24038022/loss-of-quiescence-and-impaired-function-of-cd34-cd38-low-cells-one-year-following-autologous-stem-cell-transplantation
#18
Carolien M Woolthuis, Annet Z Brouwers-Vos, Gerwin Huls, Joost Th M de Wolf, Jan Jacob Schuringa, Edo Vellenga
Patients who have undergone autologous stem cell transplantation are subsequently more susceptible to chemotherapy-induced bone marrow toxicity. In the present study, bone marrow primitive progenitor cells were examined one year after autologous stem cell transplantation and compared with normal bone marrow and mobilized peripheral blood stem cells. Post-transplantation bone marrow contained a significantly lower percentage of quiescent cells in the CD34(+)/CD38(low) fraction compared to normal bone marrow...
December 2013: Haematologica
https://www.readbyqxmd.com/read/23727584/detection-of-minimal-residual-disease-in-patients-with-multiple-myeloma-using-clonotype-specific-pcr-primers-designed-from-dna-extracted-from-archival-bone-marrow-slides
#19
Hiroyuki Takamatsu, Yoshiyasu Ogawa, Noriko Kobayashi, Kazue Obata, Tadashi Narisawa, Kouji Nakayama, Saori Munemoto, Go Aoki, Kinya Ohata, Yoshihisa Kumano, Jun Ozaki, Ryoichi Murata, Yukio Kondo, Yasushi Terasaki, Toshiro Kurokawa, Toshihiro Miyamoto, Naomi Shimizu, Toshihiro Fukushima, Akira Yoshida, Takanori Ueda, Takashi Yoshida, Shinji Nakao
Polymerase chain reaction (PCR)-negative molecular complete remission (mCR) can be induced by stem cell transplantation in some patients with multiple myeloma (MM) and is associated with long-term progression-free survival (PFS). The detection of molecular minimal residual disease (MRD), however, requires fresh or frozen materials for designing clone-specific primers, which are not always readily available. In this study, we used DNA extracted from archival bone marrow (BM) slides for PCR to detect MRD in 50 patients with MM who received various induction therapies and autologous peripheral blood stem cell transplantation (ASCT)...
October 2013: Experimental Hematology
https://www.readbyqxmd.com/read/23257404/autologous-haematopoietic-stem-cell-transplantation-for-systemic-lupus-erythematosus-data-from-the-european-group-for-blood-and-marrow-transplantation-registry
#20
B Alchi, D Jayne, M Labopin, Alexander Demin, V Sergeevicheva, T Alexander, F Gualandi, B Gruhn, J Ouyang, P Rzepecki, G Held, A Sampol, J Voswinkel, P Ljungman, A Fassas, M Badoglio, R Saccardi, D Farge
OBJECTIVES: Patients with systemic lupus erythematosus (SLE) refractory to conventional immunosuppression suffer substantial morbidity and mortality due to active disease and treatment toxicity. Immunoablation followed by autologous stem cell transplantation (ASCT) is a novel therapeutic strategy that potentially offers new hope to these patients. METHODS: This retrospective survey reviews the efficacy and safety of ASCT in 28 SLE patients from eight centres reported to the European Group for Blood and Marrow Transplantation (EBMT) registry between 2001 and 2008...
March 2013: Lupus
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