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Severe aplastic anemia

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https://www.readbyqxmd.com/read/29677078/therapeutic-outcomes-of-haploidentical-allogeneic-hematopoietic-stem-cell-transplantation-in-patients-with-severe-aplastic-anemia-a-multicenter-study
#1
Yonghua Li, Fengqi Duan, Haowen Xiao, Xiaoxiong Wu, Shunqing Wang, Duorong Xu, Qifa Liu, Zhiping Fan, Danian Nie, Yongrong Lai, Bingyi Wu, Dongjun Lin, Xin Du, Jianyu Weng, Xin Du, Zujun Jiang, Yan Pang, Lin Ouyang, Zenghui Liu, Leqin Zhang, Na Han, Lixuan Chen, Yang Xiao
BACKGROUND: Haploidentical donor allogeneic hematopoietic stem cell transplantation (HID-HSCT) is an alternative curative treatment for patients with severe aplastic anemia (SAA) who do not have suitable matched related donors (MRD). The aim of this study was to compare the therapeutic outcomes of HID-HSCT with those of MRD-HSCT for SAA. METHODS: A total of 235 SAA patients who underwent HID-HSCT (116) or MRD-HSCT (119) at 11 transplantation centers from January 2007 to January 2016 were included...
April 19, 2018: Transplantation
https://www.readbyqxmd.com/read/29674506/diagnostic-and-prognostic-roles-of-circulating-exosomal-micrornas-in-acquired-aplastic-anemia-and-myelodysplastic-syndromes
#2
Valentina Giudice, Lauren G Banaszak, Fernanda Gutierrez-Rodrigues, Sachiko Kajigaya, Reema Panjwani, Maria Del Pilar Fernandez Ibanez, Olga Rios, Christopher K Bleck, Erin S Stempinski, Diego Quinones Raffo, Danielle M Townsley, Neal S Young
Exosomal microRNAs modulate cancer cell metabolism and the immune response. Specific exosomal microRNAs have been reported to be reliable biomarkers of several solid and hematologic malignancies. We examined the possible diagnostic and prognostic values of exosomal microRNAs in two human bone marrow failure diseases, aplastic anemia and myelodysplastic syndromes. After screening of 372 microRNAs in a discovery set (n=42) of plasma exosome samples, we constructed a custom microRNA PCR plate, including 42 microRNAs, for validation in a larger cohort (n=99), and we identified 25 differentially expressed exosomal microRNAs uniquely or frequently present in aplastic anemia and/or myelodysplastic syndromes...
April 19, 2018: Haematologica
https://www.readbyqxmd.com/read/29673308/allogeneic-stem-cell-transplantation-in-fanconi-anemia-and-other-inherited-bone-marrow-failure-disorders
#3
Regis Peffault de Latour
Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative treatment for patients with an inherited bone marrow failure syndrome (IBMFS). However, the risk of treatment-related mortality after HSCT is relatively high, with graft-versus host disease (GvHD) accounting for most of the transplant-related deaths. IBMF syndromes are rare, and case reports and small series in the literature illustrate highly heterogeneous practices in terms of indications for HSCT-, timing-, stem cell source and conditioning regimens...
April 19, 2018: Current Drug Targets
https://www.readbyqxmd.com/read/29665923/-the-effect-of-serum-cytokine-levels-prior-transplantation-on-the-outcome-of-severe-aplastic-anemia-patients-received-allogeneic-hematopoietic-stem-cell-transplantation
#4
Zhao Wang, Yuan-Yuan Shi, Xin Yang, Su-Dong Zhang, Li-Ning Zhang, Dong-Lin Yang, Yi He, Rong-Li Zhang, Er-Lie Jiang, Jia-Lin Wei, Si-Zhou Feng, Min-Zhe Han
OBJECTIVE: To identify the role of serum cytokine levels prior allogeneic hematopoietic stem cell trans-plantation (allo-HSCT) in the outcome of severe aplastic anemia (SAA) patients received allo-HSCT treatment. METHODS: The clinical data of 117 SAA patients received allo-HSCT were enrolled in this study. The overall survival (OS), graft versus host disease (GVHD) incidence and relationship of serum cytokines with OS and major transplantation complications were retrospectively analyzed...
April 2018: Zhongguo Shi Yan Xue Ye Xue za Zhi
https://www.readbyqxmd.com/read/29664113/peripartum-management-of-hla-alloimmune-platelet-refractoriness
#5
Jeremy Ryan Peña, Leanna Sudhof, Barbara O'Brien
BACKGROUND: Platelet (PLT) refractoriness presents a challenging problem for transfusion support, especially in the perioperative setting, where there is urgency for human leukocyte antigen (HLA)-compatible units, yet identification and provision of compatible PLT concentrates requires time. CASE REPORT: A 22-year-old G3P1 woman with thrombocytopenia due to aplastic anemia, likely autoimmune, presented in her third trimester for peripartum care and newly diagnosed HLA alloimmune PLT refractoriness...
April 17, 2018: Transfusion
https://www.readbyqxmd.com/read/29663189/a-characteristic-flow-cytometric-pattern-with-broad-forward-scatter-and-narrowed-side-scatter-helps-diagnose-immune-thrombocytopenia-itp
#6
Raita Araki, Ryosei Nishimura, Rie Kuroda, Toshihiro Fujiki, Shintaro Mase, Kazuhiro Noguchi, Yasuhiro Ikawa, Hideaki Maeba, Akihiro Yachie
Various disorders cause severe thrombocytopenia, which can lead to critical hemorrhage. Procedures that rapidly support the diagnosis and risk factors for serious bleeding were explored, with a focus on immune thrombocytopenia (ITP). Twenty-five patients with thrombocytopenia, including 13 with newly diagnosed ITP, 3 with chronic ITP, 6 with aplastic anemia (AA), and 3 with other thrombocytopenia (one acute myeloid leukemia, one acute lymphoblastic leukemia, and one hemophagocytic lymphohistiocytosis), were reviewed...
April 16, 2018: International Journal of Hematology
https://www.readbyqxmd.com/read/29611722/single-nucleotide-polymorphisms-of-fas-and-fasl-genes-and-risk-of-idiopathic-aplastic-anemia
#7
Sadia Rehman, Nusrat Saba, Madiha Naz, Parvez Ahmed, Saeeda Munir, Sumaira Sajjad, Sobia Tabassum, Lubna Naseem
FAS/FASL signaling system plays a vital role in the regulation of apoptosis, envisaged as a death process required for immune surveillance to prevent autoimmunity and tumorigenesis along with several other biological activities. Several single-nucleotide polymorphisms (SNPs) of FAS/FASL system can result in aberrant apoptosis, which can cause different cancers and autoimmune diseases. Aplastic anemia (AA) is an autoimmune dysfunction characterized by peripheral blood pancytopenia associated with hypoplasia of bone marrow...
April 3, 2018: Immunological Investigations
https://www.readbyqxmd.com/read/29590742/myasthenia-gravis-associated-with-thymoma-and-aplastic-anemia-case-report
#8
Lidija Dežmalj Grbelja, Radovan Vrhovac, Monika Ulamec
Myasthenia gravis is associated in 10 to 15 percent of patients with thymic tumors, rarely with aplastic anemia. We report a 45-year-old male diagnosed with myasthenia gravis -associated with thymoma. We started treatment with pyridostigmine. After thymectomy, the patient -received 30 irradiation sessions. In the postoperative course, he had mild worsening of myasthenia gravis, which improved with prednisone. Five months later, he developed severe aplastic anemia. He was dependent on blood supplement. After allogeneic transplantation of bone marrow, he improved but later he -developed graft versus host disease...
December 2017: Acta Clinica Croatica
https://www.readbyqxmd.com/read/29584887/national-survey-of-myeloablative-total-body-irradiation-prior-to-hematopoietic-stem-cell-transplantation-in-japan-survey-of-the-japanese-radiation-oncology-study-group-jrosg
#9
Naoya Ishibashi, Toshinori Soejima, Hiroki Kawaguchi, Takeshi Akiba, Masatoshi Hasegawa, Kouichi Isobe, Hitoshi Ito, Michiko Imai, Yasuo Ejima, Masaharu Hata, Keisuke Sasai, Emiko Shimoda, Toshiya Maebayashi, Masahiko Oguchi, Tetsuo Akimoto
A myeloablative regimen that includes total-body irradiation (TBI) before hematopoietic stem cell transplantation results in higher patient survival rates than achieved with regimens without TBI. The TBI protocol, however, varies between institutions. In October 2015, the Japanese Radiation Oncology Study Group initiated a national survey of myeloablative TBI (covering 2010-2014). Among the 186 Japanese institutions performing TBI, 90 (48%) responded. The 82 institutions that had performed myeloablative TBI during this period treated 2698 patients with malignant disease [leukemia (2082 patients, 77...
March 23, 2018: Journal of Radiation Research
https://www.readbyqxmd.com/read/29573075/plasmodium-falciparum-infection-transmitted-by-transfusion-a-cause-of-hemophagocytic-syndrome-after-bone-marrow-tranplantation-in-a-non-endemic-country
#10
Saloua Ladeb, Nour Ben Abdejlil, Najla Fakhfakh, Amel Lakhal, Dorra Belloumi, Leila Ben Hamed, Aicha Kallel, Lamia Torjman, Rym El Fatimi, Slama Hmida, Kalthoum Kallel, Tarek Ben Othman
A 27-year-old man with severe aplastic anemia underwent bone marrow transplantation from his HLA identical brother in July 2016. Conditioning included ATGAM 30 mg/kg for 3 days and Cyclophosphamide 50 mg/kg for 4 days. The patient received several platelet and red blood cell transfusions before and after the conditioning. The patient received broad spectrum antibiotics and caspofungin because persistant febrile neutropenia without bacteriological or mycological documentation. Hemophagocytic syndrome was diagnosed on day +12...
March 23, 2018: Transplant Infectious Disease: An Official Journal of the Transplantation Society
https://www.readbyqxmd.com/read/29564363/acute-epiglottitis-in-the-immunocompromised-host-case-report-and-review-of-the-literature
#11
REVIEW
Cheng Chen, Mukil Natarajan, David Bianchi, Georg Aue, John H Powers
We present a case of acute epiglottitis in a 16-year-old with severe aplastic anemia. He was admitted with a history suggestive of a severe upper airway infection and an absolute neutrophil count of 0 per cubic millimeter. Despite his immunocompromised state, he presented with the classical signs and symptoms of epiglottitis. We review here the presentation and comorbidities of immunocompromised patients with epiglottitis. In addition, the appropriate choice of empirical antibiotic therapy is important for the management of epiglottitis in immunocompromised patients, especially in the post- Haemophilus influenza type B vaccination era...
March 2018: Open Forum Infectious Diseases
https://www.readbyqxmd.com/read/29563590/successful-outcomes-of-second-hematopoietic-stem-cell-transplantation-with-total-nodal-irradiation-and-atg-conditioning-for-graft-failure-in-adult-patients-with-severe-aplastic-anemia
#12
Seung-Ah Yahng, Sung-Soo Park, Young-Woo Jeon, Jae-Ho Yoon, Seung-Hwan Shin, Sung-Eun Lee, Byung-Sik Cho, Ki-Seong Eom, Yoo-Jin Kim, Seok Lee, Chang-Ki Min, Hee-Je Kim, Seok-Goo Cho, Dong-Wook Kim, Woo-Sung Min, Jong Wook Lee
Data regarding the optimal approach for second allogeneic hematopoietic stem cell transplantation (HSCT) after graft failure (GF) in acquired severe aplastic anemia (SAA) are still limited and heterogeneous. We examined 24 patients who underwent second HLA-matched sibling donor (MSD) peripheral blood HSCT for GF. The reconditioning regimen (TNI-750/ATG) consisted of a single dose of total nodal irradiation (TNI, 750 cGy) and antithymocyte globulin (ATG; Thymoglobulin® , 1.25 mg/kg/day for 3 days). All but one patient achieved successful engraftment of neutrophils (median 12 days, range 5-21) and platelets (median 15 days, range 9-316)...
March 21, 2018: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/29562465/-impact-on-platelet-recovery-of-recombinant-human-thrombopoietin-in-severe-aplastic-anemia-patients-with-allogeneic-hematopoietic-stem-cell-transplantation
#13
Y Song, F Zhou, N X Song, X M Liu, Z Yu, L N Xie, X C Song, X Li
Objective: To investigate and analyze the impact on PLT recovery of recombinant human thrombopoietin (rhTPO) in severe aplastic anemia (SAA) patients with allogeneic hematopoietic stem cell transplantation (allo-HSCT). Methods: A retrospective analysis of Hematology Division of General Hospital of Jinan Military Command was conducted in the 85 SAA cases who treated with allo-HSCT from January 2010 to March 2017. According to the administration of medicines for platelets, 85 patients were divided into rhTPO group ( n =29), rhIL-11 group ( n =27) and blank group ( n =29), respectively...
March 14, 2018: Zhonghua Xue Ye Xue za Zhi, Zhonghua Xueyexue Zazhi
https://www.readbyqxmd.com/read/29562463/-rapamycin-affect-the-apoptosis-of-splenic-cd4-cd25-regulatory-t-cells-of-mouse-severe-aplastic-anemia-model
#14
Z H Lin, H Liu, L Zhu, X Yang, Y P Zhang, J Qian, H Y Liu
Objective: To explore the effects and possible mechanism of rapamycin (RAPA) on apoptosis of CD4+ CD25+ Tregs from the mouse severe aplastic anemia (SAA) model. Methods: The BALB/c female SAA model mice were induced by interferon-gamma in combination with busulphan. The SAA model mice were intraperitoneal injection with RAPA at daily dose of 0.5 mg/kg for 5 days (the RAPA-treated group, n =15) in the SAA group ( n =15) and the un-treated group ( n =15) were control. Bone marrow hematopoiesis changes were observed by the patho-morphological examination of femurs...
March 14, 2018: Zhonghua Xue Ye Xue za Zhi, Zhonghua Xueyexue Zazhi
https://www.readbyqxmd.com/read/29562461/-comparison-of-different-styles-of-allogeneic-hematopoietic-stem-cell-transplantation-as-first-line-treatment-treated-with-severe-aplastic-anemia-in-children-and-adolescents
#15
S W Yang, R J Ma, J J Zhao, H F Zhong, X L Yuan, L Jiang, J Yang, P C Lei, Y Zhang, Y W Fu, D M Wan, Z M Zhu
Objective: To evaluate the efficacy of allogeneic hematopoietic stem cell transplantation (allo-HSCT) from different donors as first-line treatment for children and adolescents with severe aplastic anemia (SAA) . Methods: The clinical data of 79 children and adolescents with SAA diagnosed from January 2013 to December 2016 in Henan Province were retrospectively analyzed. There were 50 males and 29 females, with a median age of 14(4-18) years. 40 cases received matched sibling transplantation (MSD-HSCT), 17 with unrelated donor transplantation (UD-HSCT), and 22 with haploidentical transplantation (haplo-HSCT)...
March 14, 2018: Zhonghua Xue Ye Xue za Zhi, Zhonghua Xueyexue Zazhi
https://www.readbyqxmd.com/read/29562449/-the-life-span-of-red-blood-cell-in-patients-with-severe-very-severe-aplastic-anemia
#16
L Ye, J Guo, L P Jing, G X Peng, K Zhou, Y Li, Y Li, J P Li, H H Fan, L Song, F K Zhang, L Zhang
Objective: To explore the life span of red blood cells (RBC) in patients with severe/very severe aplastic anemia (SAA/VSAA). Methods: Clinical data of 128 SAA/VSAA patients from November 2016 to April 2017 were retrospectively analyzed, and 13 healthy volunteers in the same period was used as normal control. The endogenous Breath Carbon Monoxide (CO) test was used to detect the life span of RBC in SAA/VSAA patients, and the effect of immunosuppressive therapy (IST) on the life span of RBC in these patients was explored...
February 14, 2018: Zhonghua Xue Ye Xue za Zhi, Zhonghua Xueyexue Zazhi
https://www.readbyqxmd.com/read/29562447/-posaconazole-as-primary-prevention-of-fungal-infection-in-intensive-immunosuppressive-therapy-for-severe-aplastic-anemia
#17
M Chen, J L Zhuang, M H Duan, W Zhang, J Li, T N Zhu, H C Cai, X X Cao, J Feng, C Yang, Y Zhang, L Zhang, D B Zhou, B Han
Objective: To Evaluate the efficacy and safety of posaconazole as primary prevention of invasive fungal disease (IFD) in patients with severe aplastic anemia (SAA) treated with anti-thymus/lymphocyte immunoglobulin (ATG/ALG) combined with cyclosporine intensive immunosuppressive therapy (IST). Methods: A retrospective analysis of clinical data of 58 SAA patients who received IST of anti-thymocyte immunoglobulin combining cyclosporine and antifungal prophylaxis during April 2013 to May 2017 in Peking Union Medical College Hospital was performed...
February 14, 2018: Zhonghua Xue Ye Xue za Zhi, Zhonghua Xueyexue Zazhi
https://www.readbyqxmd.com/read/29551028/-clinical-efficacy-and-safety-of-porcine-antihuman-lymphocyte-immunoglobulin-in-alternative-donor-allogeneic-hematopoietic-cell-transplantation-for-severe-aplastic-anemia
#18
X Chen, J L Wei, Y Huang, E L Jiang, Q L Ma, W H Zhai, Y He, R L Zhang, D L Yang, J F Yao, G X Zhang, S Z Feng, M Z Han
Objective: To compare eficacy and safety of porcine antihuman lymphocyte immunoglobulin (pALG) and rabbit antithymocyte immunoglobulin (rATG) as a part of alternative donor allogeneic hematopoietic stem cell transplantation (AD allo-HSCT) for severe aplastic anemia (SAA). Methods: The clinical data of 46 SAA patients received AD allo-HSCT from January 2006 to November 2016 were retrospectively analyzed. The cohort of patients were divided into two groups based on rATG or pALG as a part of conditioning regimen to compare implantation rate, transplantation related complications and outcome...
January 14, 2018: Zhonghua Xue Ye Xue za Zhi, Zhonghua Xueyexue Zazhi
https://www.readbyqxmd.com/read/29549173/the-clinical-spectrum-of-pyruvate-kinase-deficiency-data-from-the-pyruvate-kinase-deficiency-natural-history-study
#19
Rachael F Grace, Paola Bianchi, Eduard J van Beers, Stefan W Eber, Bertil Glader, Hassan M Yaish, Jenny M Despotovic, Jennifer A Rothman, Mukta Sharma, Melissa M McNaull, Elisa Fermo, Kimberly Lezon-Geyda, D Holmes Morton, Ellis J Neufeld, Satheesh Chonat, Nina Kollmar, Christine M Knoll, Kevin Kuo, Janet L Kwiatkowski, Dagmar Pospíŝilová, Yves D Pastore, Alexis A Thompson, Peter E Newburger, Yaddanapudi Ravindranath, Winfred C Wang, Marcin W Wlodarski, Heng Wang, Susanne Holzhauer, Vicky R Breakey, Joachim Kunz, Sujit Sheth, Melissa J Rose, Heather A Bradeen, Nolan Neu, Dongjing Guo, Hasan Al-Sayegh, Wendy B London, Patrick G Gallagher, Alberto Zanella, Wilma Barcellini
An international, multicenter registry was established to collect retrospective and prospective clinical data on patients with pyruvate kinase (PK) deficiency, the most common glycolytic defect causing congenital non-spherocytic hemolytic anemia. Medical history and laboratory and radiologic data were retrospectively collected at enrollment in 254 patients with molecularly confirmed PK deficiency. Perinatal complications were common, including anemia requiring transfusions, hyperbilirubinemia, hydrops, and prematurity...
March 16, 2018: Blood
https://www.readbyqxmd.com/read/29532539/co-transplantation-of-haploidentical-hematopoietic-stem-cell-and-allogeneic-bone-marrow-derived-mesenchymal-stromal-cells-as-a-first-line-treatment-in-very-severe-aplastic-anemia-patients-with-refractory-infections
#20
Chunyan Yue, Yingying Ding, Yang Gao, Li Li, Yan Pang, Zenghui Liu, Hang Zhang, Yang Xiao, Zujun Jiang, Haowen Xiao
OBJECTIVES: In patients with very severe aplastic anemia (VSAA), neutropenia is prolonged and persistent, resulting in refractory overwhelming infections. Hematopoiesis recovery is urgently needed. METHODS: Six patients with de novo VSAA lacking HLA-identical sibling donors and those who experienced refractory infections, underwent haploidentical related donor (HRD) hematopoietic stem cell transplantation (HSCT) as a first-line therapy. The conditioning regimen consisted of busulfan, cyclophosphamide and rabbit antithymocyte globulin...
March 12, 2018: European Journal of Haematology
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