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https://read.qxmd.com/read/30778805/haploidentical-stem-cell-transplantation-with-post-transplant-cyclophosphamide-for-primary-immune-deficiency-disorders-in-children-challenges-and-outcome-from-a-tertiary-care-center-in-south-india
#1
Ramya Uppuluri, Meena Sivasankaran, Shivani Patel, Venkateswaran Vellaichamy Swaminathan, Kesavan Melarcode Ramanan, Nikila Ravichandran, Balasubramaniam Ramakrishnan, Indira Jayakumar, Lakshman Vaidhyanathan, Revathi Raj
Haploidentical stem cell transplantation (haplo SCT) has emerged as an acceptable alternative to matched family donor transplantation for children diagnosed to have primary immune deficiency disorders (PIDs). We present data over 4 years on the challenges and efficacy of unmanipulated T cell replete haplo SCTs with post-transplant cyclophosphamide (PTCy) in children diagnosed to have PIDs. We performed a retrospective study in the pediatric blood and marrow transplantation unit where all children less than 18 years of age diagnosed to have PIDs and who underwent haplo SCT with PTCy from January 2014 to February 2018 were included in the study...
February 18, 2019: Journal of Clinical Immunology
https://read.qxmd.com/read/30758645/different-prognostic-effects-of-core-binding-factor-positive-aml-with-korean-aml-registry-data
#2
Ho-Jin Shin, Woo-Sung Min, Yoo Hong Min, June-Won Cheong, Je-Hwan Lee, In-Ho Kim, Dae Sik Hong, Jae-Sook Ahn, Hyeoung-Joon Kim, Won-Sik Lee, Chul Won Jung, Jun-Ho Jang, Young Park, Hee-Je Kim
Core-binding factor acute myeloid leukemia (CBF-AML) data in Asian countries has been rarely reported. We analyzed 392 patients with CBF-AML [281 with t(8;21), 111 with inv.(16)/t(16;16)] among data from 3041 patients with AML from the Korean AML Registry. Interestingly, del(9q) was less frequently detected in Korean than in German patients with t(8;21) (7.5% vs. 17%), and del(7q) was more frequently detected in Korean patients with inv(16). Overall survival (OS) was similar between patients in the first complete remission (CR) who received allogeneic (alloSCT) and autologous stem cell transplantation (ASCT) for CBF-AML...
February 13, 2019: Annals of Hematology
https://read.qxmd.com/read/30747249/comparison-of-the-safety-and-efficacy-of-prophylactic-donor-lymphocyte-infusion-after-haploidentical-versus-matched-sibling-pbsct-in-very-high-risk-acute-myeloid-leukemia
#3
Xiao-Ning Gao, Ji Lin, Li-Jun Wang, Fei Li, Hong-Hua Li, Shu-Hong Wang, Wen-Rong Huang, Chun-Ji Gao, Li Yu, Dai-Hong Liu
Donor lymphocyte infusion (DLI) might be used prophylactically to reduce relapse after allogeneic hematopoietic stem cell transplantation for very high-risk leukemia/lymphoma without effective targeted therapy. To compare the safety and efficacy of prophylactic DLI for prevention of relapse after allogeneic peripheral blood stem cell transplantation from haploidentical donors (HID-SCT) and matched-sibling donors (MSD-SCT) in patients with very high-risk acute myeloid leukemia (AML), we performed a retrospective analysis in a cohort of 21 HID-SCT and 13 MSD-SCT recipients, displaying similar baseline characteristics except for donor's gender distribution...
February 12, 2019: Annals of Hematology
https://read.qxmd.com/read/30714847/anti-cd19-chimeric-antigen-receptors-t-cells-for-the-treatment-of-relapsed-or-refractory-e2a-pbx1-positive-acute-lymphoblastic-leukemia-report-of-three-cases
#4
Jian Zhang, Fei Yang, Hui-Ying Qiu, Qian Wu, Dan-Qing Kong, Jin Zhou, Yue Han, De-Pei Wu
Patients with relapsed or refractory E2A-PBX1 positive acute B lymphoblastic leukemia (B-ALL) receiving anti-CD19 chimeric antigen receptor T cells (CAR-T) were retrospectively assessed to evaluate the efficacy and safety of disease burden on outcomes and to identify predictive variables. Of the three case patients, case 1 relapsed after hematopoietic stem cell transplantation. After being treated with anti-CD19 CAR-T, the patient showed minimal residual disease (MRD), and his fusion genes turned negative. Case 2, who suffered refractory leukemia, received anti-CD19 CAR-T treatment in an attempt to remove the MRD before transplantation...
February 4, 2019: Leukemia & Lymphoma
https://read.qxmd.com/read/30703225/-molecular-cytogenetic-characterization-of-five-patients-with-myeloid-leukemia-and-t-12-22-p13-q12
#5
Haigang Shao, Qian Yang, Yanlei Gong, Shuxiao Bai, Jun Zhang, Yong Wang, Juan Shen, Chunxiao Wu, Huiying Qiu, Suning Chen, Jinlan Pan
OBJECTIVE: To explore the clinical and laboratory characteristics of 5 patients with myeloid leukemia and t(12;22)(p13;q12). METHODS: Bone marrow cells were cultured for 24 h and analyzed by standard R-banding. Rearrangement of the MN1 gene was detected by fluorescence in situ hybridization (FISH) using dual color break-apart MN1 probes. MN1-ETV6 and ETV6-MN1 fusion genes were detected by reverse transcription polymerase chain reaction (RT-PCR). And the products were subjected to direct sequencing...
February 10, 2019: Zhonghua Yi Xue Yi Chuan Xue za Zhi, Zhonghua Yixue Yichuanxue Zazhi, Chinese Journal of Medical Genetics
https://read.qxmd.com/read/30696949/ixazomib-lenalidomide-and-dexamethasone-in-patients-with-newly-diagnosed-multiple-myeloma-long-term-follow-up-including-ixazomib-maintenance
#6
Shaji K Kumar, Jesus G Berdeja, Ruben Niesvizky, Sagar Lonial, Jacob P Laubach, Mehdi Hamadani, A Keith Stewart, Parameswaran Hari, Vivek Roy, Robert Vescio, Jonathan L Kaufman, Deborah Berg, Eileen Liao, S Vincent Rajkumar, Paul G Richardson
Triplet combinations containing a proteasome inhibitor are a standard of care in newly diagnosed multiple myeloma (NDMM). We examined the long-term efficacy and safety of the all-oral combination of weekly ixazomib plus lenalidomide-dexamethasone (IRd), followed by single-agent ixazomib maintenance in NDMM patients. Of 65 enrolled patients, 53 received ixazomib 4 mg (days 1, 8, and 15) plus lenalidomide 25 mg (days 1-21) and dexamethasone 40 mg (days 1, 8, 15, and 22) for up to twelve 28-day induction cycles...
January 29, 2019: Leukemia: Official Journal of the Leukemia Society of America, Leukemia Research Fund, U.K
https://read.qxmd.com/read/30680765/minimal-residual-disease-status-determined-by-multiparametric-flow-cytometry-pretransplantation-predicts-the-outcome-of-patients-with-all-receiving-unmanipulated-haploidentical-allografts
#7
Xiao-Su Zhao, Yan-Rong Liu, Lan-Ping Xu, Yu Wang, Xiao-Hui Zhang, Huan Chen, Yu-Hong Chen, Wei Han, Yu-Qian Sun, Chen-Hua Yan, Xiao-Dong Mo, Ya-Zhe Wang, Qiao-Zhen Fan, Xin-Yu Wang, Kai-Yan Liu, Xiao-Jun Huang, Ying-Jun Chang
This study evaluated the effects of pretransplantation minimal residual disease (pre-MRD) on outcomes of acute lymphoblastic leukemia (ALL) patients who underwent unmanipulated haploidentical stem cell transplantation (haplo-SCT). A retrospective study including 543 patients with ALL was performed. MRD was determined using multiparametric flow cytometry. Both in the entire cohort of patients and in subgroup cases with T-ALL or B-ALL, subjects with positive pre-MRD had a higher incidence of relapse (CIR) than those with negative pre-MRD in MSDT settings (P<0...
January 25, 2019: American Journal of Hematology
https://read.qxmd.com/read/30672611/caspofungin-for-primary-antifungal-prophylaxis-after-t-cell-replete-haploidentical-stem-cell-transplantation-with-post-transplant-cyclophosphamide
#8
Jacopo Mariotti, Chiara De Philippis, Stefania Bramanti, Barbara Sarina, Federica Tordato, Daria Pocaterra, Erminia Casari, Carmelo Carlo-Stella, Armando Santoro, Luca Castagna
OBJECTIVES: T cell-replete Haploidentical stem transplantation (Haplo-SCT) with post-transplant cyclophosphamide (PT-Cy) is at high risk of invasive fungal infections (IFI) and anti-mold active drug is required for primary antifungal prophylaxis (PAP) according to international guidelines. No data are available on the efficacy of caspofungin as PAP in this setting. METHODS: Here we report our retrospective experience with 103 consecutive patients treated with caspofungin as PAP after Haplo-SCT...
January 23, 2019: European Journal of Haematology
https://read.qxmd.com/read/30660629/controlled-release-of-corticosteroid-with-biodegradable-nanoparticles-for-treating-experimental-autoimmune-uveitis
#9
Lixia Luo, Jin Yang, Yumin Oh, Matthew J Hartsock, Shiyu Xia, Yoo-Chun Kim, Zheng Ding, Tuo Meng, Charles G Eberhart, Laura Ensign, Jennifer E Thorne, Walter J Stark, Elia J Duh, Qingguo Xu, Justin Hanes
Noninfectious uveitis is a potentially blinding ocular condition that often requires treatment with corticosteroids to prevent inflammation-related ocular complications. Severe forms of uveitis such as panuveitis that affects the whole eye often require a combination of topical and either regional or systemic corticosteroid. Regional corticosteroids are currently delivered inside the eye by intravitreal injection (e.g. Ozurdex®, an intravitreal dexamethasone implant). Intravitreal injection is associated with rare but potentially serious side effects, including endophthalmitis, retinal and vitreous hemorrhage, and retinal detachment...
January 17, 2019: Journal of Controlled Release: Official Journal of the Controlled Release Society
https://read.qxmd.com/read/30639823/induction-therapy-with-bortezomib-and-dexamethasone-and-conditioning-with-high-dose-melphalan-and-bortezomib-followed-by-autologous-stem-cell-transplantation-for-al-amyloidosis-long-term-follow-up-analysis
#10
Vishal K Gupta, Dina Brauneis, Anthony C Shelton, Karen Quillen, Shayna Sarosiek, J Mark Sloan, Vaishali Sanchorawala
In light-chain (AL) amyloidosis, the depth of hematologic response to treatment is associated with improved survival and organ responses. We conducted a clinical trial utilizing bortezomib in induction and in conditioning with melphalan prior to stem cell transplantation (SCT) for AL amyloidosis. The results of this clinical trial with a median follow-up of 36 months have been reported previously. Here we report the long-term results of this clinical trial with a median follow-up of 77 months. The objectives of this follow-up report are to describe survival, durability of hematologic and organ responses, and relapse rates...
January 10, 2019: Biology of Blood and Marrow Transplantation
https://read.qxmd.com/read/30625463/cardiovascular-outcomes-in-african-americans-with-sickle-cell-trait-and-chronic-kidney-disease
#11
Kabir O Olaniran, Nwamaka D Eneanya, Andrew S Allegretti, Sophia H Zhao, Maureen M Achebe, Ravi I Thadhani
BACKGROUND: Sickle cell trait (SCT) is common among African Americans and has been historically considered to be benign. Recently, SCT has been associated with an increased risk for chronic kidney disease (CKD) and cardiovascular disease in the general population. Our understanding of SCT has been extrapolated largely from data of patients with sickle cell disease (SCD). Notably, in SCD, the outcomes differ by sex. The effect of SCT on cardiovascular risk in the African American CKD population is unknown, and the interaction between SCT and sex on cardiovascular risk has not been investigated...
2019: American Journal of Nephrology
https://read.qxmd.com/read/30620383/impact-of-conditioning-intensity-on-outcomes-of-haploidentical-stem-cell-transplantation-for-patients-with-acute-myeloid-leukemia-over-45-years-of-age
#12
Nicole Santoro, Myriam Labopin, Fabio Ciceri, Maria Teresa Van Lint, Daniela Nasso, Didier Blaise, William Arcese, Johanna Tischer, Benedetto Bruno, Gerhard Ehninger, Yener Koc, Stella Santarone, Xiao-Jun Huang, Bipin N Savani, Mohamad Mohty, Annalisa Ruggeri, Arnon Nagler
BACKGROUND: T cell-replete haploidentical stem cell transplantation (haplo-SCT) is a valid therapeutic option for adult patients with high-risk acute myeloid leukemia (AML) lacking an HLA-matched sibling or unrelated donor. METHOD: We retrospectively analyzed the outcomes of 912 AML patients ≥45 years of age who had undergone haplo-SCT with either myeloablative conditioning (MAC; n = 373) or reduced intensity conditioning (RIC; n = 539) regimens. RESULTS: The median follow-up was 31...
January 8, 2019: Cancer
https://read.qxmd.com/read/30597277/effect-of-cyp3a4-cyp3a5-and-abcb1-polymorphisms-on-intravenous-tacrolimus-exposure-and-adverse-events-in-adult-allogeneic-stem-cell-transplant-patients
#13
Issam S Hamadeh, Qing Zhang, Nury Steuerwald, Alicia Hamilton, Lawrence J Druhan, Meredith McSwain, Yordanis Diez, Stephanie Rusin, Yimei Han, James Symanowski, Jonathan Gerber, Michael R Grunwald, Nilanjan Ghosh, Dragos Plesca, Justin Arnall, Jigar Trivedi, Belinda Avalos, Edward Copelan, Jai N Patel
Pharmacogenetics influences oral tacrolimus exposure; however, little data exist with intravenous tacrolimus. Herein, we investigated the impact of genetic polymorphisms in CYP3A4, CYP3A5, and ABCB1 on intravenous tacrolimus exposure and toxicity in adult patients receiving an allogeneic hematopoietic stem cell transplant for hematologic malignancies. Germ-line DNA was extracted from buccal swabs and genotyped for CYP3A4, CYP3A5 and ABCB1 polymorphisms. Continuous intravenous infusion of tacrolimus 0.03 mg/kg/day was initiated on day +5 post-transplant and steady-state blood concentrations were measured 4 days later...
December 28, 2018: Biology of Blood and Marrow Transplantation
https://read.qxmd.com/read/30597264/enhancement-of-oral-bioavailability-of-salmon-calcitonin-through-chitosan-modified-dual-drug-loaded-nanoparticles
#14
Lin Liu, Hui Yang, Yan Lou, Jia-Ying Wu, Jing Miao, Xiao-Yang Lu, Jian-Qing Gao
Because numerous challenges limit the effective oral delivery of protein and peptide drugs, we developed promising chitosan (CS)-modified, dual drug-loaded nanoparticles (NPs) simultaneously containing salmon calcitonin (sCT) and puerarin (PR) (CS-sCT/PR-NPs), and to explore the potential of PR as a protease inhibitor. This oral delivery system showed efficient encapsulation of sCT (75.7%) and PR (50.9%), protection of encapsulated sCT and PR from premature release in simulated gastric fluid (SGJ, pH 1.2), and sustained-release behavior in phosphate buffer saline (PBS, pH 7...
December 28, 2018: International Journal of Pharmaceutics
https://read.qxmd.com/read/30583985/sarcopenic-obesity-derived-from-pet-ct-predicts-mortality-in-lymphoma-patients-undergoing-hematopoietic-stem-cell-transplantation
#15
J Jabbour, B Manana, A Zahreddine, C Saade, M Charafeddine, A Bazarbachi, D Blaise, J El-Cheikh
BACKGROUND: Sarcopenic Obesity (SO) is associated with worse survival among chemotherapy recipients. Research on SO is scarce among lymphoma patients receiving Hematopoietic Stem Cell Transplantation (HSCT). AIM: assess prevalence of SO pre-HSCT (T0) and 3 months post-HSCT (T1) in lymphoma patients and determine the power of SO at T0 and T1 in predicting survival. METHODS: Consecutive patients (age ≥16 years) having B and T cell lymphoma who underwent SCT and who had PET/CT scan pre-SCT and 3 months post SCT were included in the study...
December 21, 2018: Current Research in Translational Medicine
https://read.qxmd.com/read/30569802/a-novel-recombinant-human-thrombopoietin-for-treating-prolonged-isolated-thrombocytopenia-after-allogeneic-stem-cell-transplantation
#16
Yu-Qian Sun, Yuan Kong, Xiao-Hui Zhang, Yu Wang, Min-Min Shi, Yang Song, Jun Kong, Hai-Xia Fu, Chen-Hua Yan, Lan-Ping Xu, Kai-Yan Liu, Xiao-Jun Huang
Patients with prolonged isolated thrombocytopenia (PT) after allogeneic stem cell transplantation (allo-SCT) are known to have a poor prognosis. However, there is no standard treatment for PT. The present study aimed to investigate the potential effect of a novel recombinant human thrombopoietin (rhTPO) in a cohort of patients with PT following allo-SCT. A total of 24 patients with PT (including delayed platelet engraftment and secondary failure of platelet recovery) were treated with rhTPO from July 1, 2016 to May 31, 2017...
December 20, 2018: Platelets
https://read.qxmd.com/read/30564300/clofarabine-busulfan-based-reduced-intensity-conditioning-regimens-provides-very-good-survivals-in-acute-myeloid-leukemia-patients-in-complete-remission-at-transplant-a-retrospective-study-on-behalf-of-the-sfgm-tc
#17
Amandine Le Bourgeois, Myriam Labopin, Mathieu Leclerc, Régis Peffault de Latour, Jean-Henri Bourhis, Patrice Ceballos, Corentin Orvain, Hélène Labussière Wallet, Karin Bilger, Didier Blaise, Marie-Thérese Rubio, Thierry Guillaume, Mohamad Mohty, Patrice Chevallier
Background: Clofarabine has been proved to have higher anti-leukemic myeloid activity compared to fludarabine, a drug extensively used as part of reduced intensity conditioning (RIC) for allogeneic stem cell transplantation (allo-SCT). Results: Eighty-four patients were included. The majority of patients had acute myeloid leukemia (AML, n = 63). Sixty-one patients were in complete remission (AML n = 55). With a median follow up of 31 months (range: 5.7-74.1), 2-year overall (OS) and disease-free (DFS) survivals, relapse incidence (RI), non-relapse mortality (NRM) and graft-versus-host disease (GVHD)/relapse free survival (GRFS) were 64...
November 27, 2018: Oncotarget
https://read.qxmd.com/read/30561765/decompensated-liver-disease-due-to-primary-hepatic-amyloidosis-is-liver-transplantation-still-mandatory
#18
Mar Riveiro-Barciela, Merche Gironella, Alicia Senín, María-Teresa Salcedo, Xavier Merino-Casabiel, Lluis Castells, Rafael Esteban, Maria Buti, Fernando Martínez-Valle
Primary hepatic amyloidosis (PHA) is extremely uncommon and usually associated with poor prognosis due to the development of decompensation. Therapy for this condition is liver transplantation (LT) and subsequent stem cell transplantation (SCT)(1). Nonetheless, because the liver disease can progress rapidly and liver donation is limited, patients may die before transplantation. This article is protected by copyright. All rights reserved.
December 18, 2018: Hepatology: Official Journal of the American Association for the Study of Liver Diseases
https://read.qxmd.com/read/30511683/association-of-persistent-minimal-residual-disease-with-poor-outcomes-of-patients-with-acute-myeloid-leukemia-undergoing-allogeneic-hematopoietic-stem-cell-transplantation
#19
Jing Liu, Xiao-Su Zhao, Yan-Rong Liu, Lan-Ping Xu, Xiao-Hui Zhang, Huan Chen, Yu-Hong Chen, Feng-Rong Wang, Wei Han, Yu-Qian Sun, Chen-Hua Yan, Fei-Fei Tang, Xiao-Dong Mo, Kai-Yan Liu, Qiao-Zhen Fan, Xiao-Jun Huang, Ying-Jun Chang
Background: Several studies have shown that detection of minimal residual disease (MRD) in acute myeloid leukemia (AML) is an independent prognostic factor. This study aimed to evaluate the significance of dynamic MRD pretransplantation on outcome of AML patients receiving allogeneic hematopoietic stem cell transplantation (allo-HSCT). Methods: We retrospectively analyzed 145 consecutive AML patients undergoing allo-HSCT in complete remission status between June 2013 and June 2016...
December 5, 2018: Chinese Medical Journal
https://read.qxmd.com/read/30504341/balancing-exercise-risk-and-benefits-lessons-learned-from-sickle-cell-trait-and-sickle-cell-anemia
#20
REVIEW
Robert I Liem
Development of exercise guidelines for individuals with sickle cell trait (SCT) and sickle cell anemia (SCA) is hampered by the need to weigh the benefits against risks of exercise in these populations. In SCT, concern for exercise collapse associated with sickle cell trait has resulted in controversial screening of student athletes for SCT. In SCA, there exists unsubstantiated concerns that high-intensity exercise may result in pain and other complications. In both, finding the "right dose" of exercise remains a challenge for patients and their providers...
November 30, 2018: Hematology—the Education Program of the American Society of Hematology
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