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Mechanisms of Fibril Formation and Cellular Response
Published in Martha Skinner, John L. Berk, Lawreen H. Connors, David C. Seldin, XIth International Symposium on Amyloidosis, 2007
Martha Skinner, John L. Berk, Lawreen H. Connors, David C. Seldin
Autologous stem cell transplantation has been an effective tool for the treatment of hematologic malignancies for over 20 years. The routine administration of filgrastim has been demonstrated to reduce infections, shorten hospital stays, and reduce the overall duration of granulocytopenia. In amyloidosis patients, however, the administration of growth factor, particularly to those patients with amyloid cardiomyopathy or amyloid nephrotic syndrome, results in significant problems of fluid retention and occasionally respiratory distress syndrome. Deaths during apheresis of these patients have been reported. As a consequence of these complications, we have attempted to support patients following conditioning chemotherapy without growth factor. We found that the median time to achieve a neutrophil count of 500/uL, platelet counts of 20,000 and 50,000, respectively, were 14, 14, and 18 days, respectively. Since the incidence of non-staphylococcus-coagulase-negative bacteremia was only 16%, and the median time spent in the hospital was just slightly greater than a week, it would appear feasible as well as reasonable to withhold growth factor following autologous stem cell transplant for patients with AL.
Melphalan Plus High-Dose Dexamethasone in AL Patients Who are Not Candidates for Stem Cell Transplantation
Published in Gilles Grateau, Robert A. Kyle, Martha Skinner, Amyloid and Amyloidosis, 2004
Giovanni Palladini, Vittorio Perfetii, Laura Obici, Francesca Lavatelli, Riccardo Caccialanza, Fausto Adami, Giobatta Cavallero, Roberto Rustichelli, Giovambattista Virga, Giampaolo Merlini
The prognosis of patients with systemic AL is poor, with a median survival of 12–18 months and the main prognostic determinant is amyloid heart involvement (2). At present, therapy of AL is aimed at suppressing the plasma cell clone producing the amyloidogenic light-chain. The most effective treatment so far is autologous stem cell transplantation (ASCT), which can induce complete remissions in more than 50% of patients who can undergo this procedure (2, 3). However, a considerable proportion of AL patients is not eligible for ASCT due to advanced disease (3). The patients in whom ASCT is contraindicated are those in greatest need of prompt care. They are usually treated with oral melphalan plus prednisone (MP). However, response rate to this regimen is unsatisfactory and time to response can be unaffordably long. Our previous experience indicated that MP can provide a 37% response rate with a long time to response (median 9 months) (4). Kyle et al. reported that 28% of AL patients respond to MP and 30% of them require to be treated for more than 1 year (5). Time to response to high-dose dexamethasone (HD-Dex) is shorter (median 4 months), but response rate to HD-Dex is still only 35% (6). In an attempt of synergizing the therapeutic actions of melphalan and HD-Dex, we treated with this association 46 patients who were not eligible to ASCT.
BONE MARROW AND STEM CELL TRANSPLANTATION
Published in James Bishop, Cancer Facts, 1999
Autologous stem cell transplantation is now widely used for treatment of a variety of common solid tumours, including breast, small cell lung, ovarian, and soft tissue sarcomas. With the exception of paediatric tumours, results are generally disappointing.
Infections in hematopoietic stem cell transplant patients admitted to Hematology intensive care unit: a single-center study
Published in Hematology, 2021
Tuğba Barlas, Kamil İnci, Gulbin Aygencel, Melda Türkoğlu, Özlem Güzel Tunçcan, Ferda Can, Lale Aydın Kaynar, Zübeyde Nur Özkurt, Zeynep Arzu Yeğin, Münci Yağcı
HSCT patients admitted to Hematology ICU of Gazi University Hospital between 01 January 2014 and 01 September 2017 were identified. For eight patients with more than one admission to ICU, the first admission data were included. Four patients who died within the first 24 h of ICU admission and six patients who had more than one HSCT procedure were excluded. As a result, a total of 62 patients were included. Thirty-eight (61.3%) patients were male and the median age of the patients was 55.5 [34.25–62] years. The most common hematological diseases presented as indications for HSCT were multiple myeloma in 18 (29%), lymphoma in 17 (27.4%) and acute myeloid leukemia in 14 (22.6%) patients. About 26 (41.9%) patients received autologous stem cell transplantation and 36 (58.1%) patients received allogeneic stem cell transplantation. Peripheral stem cell sources were used in all stem cell transplantations. The conditioning regimens were myeloablative in 25 (40.3%), non-myeloablative in 13 (21%) and reduced intensity in 24 (38.7%) patients. Total body irradiation (TBI) was performed in eight patients in the myeloablative regimen group. The disease stages were progression in 26 (41.9%) patients, remission in 20 (32.3%) and stable disease in 16 (25.8%) at the time of transplantation.
Contemporary strategies to improve outcomes for peripheral T-cell lymphoma patients following the failure of first-line therapy
Published in Expert Review of Hematology, 2020
Avyakta Kallam, James O. Armitage
Autologous stem cell transplantation is often considered in patients with chemo-sensitive disease in order to decrease the relapse rates. The Nordic Lymphoma group assigned patients with the chemo-sensitive disease to a BEAM (carmustine, etoposide, cytarabine, melphalan) based autologous stem cell transplant [14]. The overall 5 year progression-free survival (PFS) and overall survival (OS) were 51% and 44%, respectively. Patients with PTCL-NOS and AITL had a 5 year PFS of 38% and 49% and OS of 49% and 47%, respectively. Patients with ALK-positive ALCL had the best outcomes, with 5 year PFS and OS of 61% and 70%, respectively. It is unclear why patients with aggressive T cell lymphomas respond poorly to conventional B cell lymphoma regimens, however there are data emerging reporting drug resistance pathways such as altered p-glycoprotein expression in certain subsets of patients [15]. The outcomes of patients who progress following stem cell transplantation are worse.
Emerging therapies in β-thalassemia: toward a new era in management
Published in Expert Opinion on Emerging Drugs, 2020
Rayan Bou-Fakhredin, Rami Tabbikha, Hisham Daadaa, Ali T. Taher
Additional data for gene therapy in TDT patients came from the HGB-204 and HGB-205 trials (ClinicalTrials.gov numbers, NCT01745120 and NCT02151526). In these two trials, autologous CD34+ cells were obtained from 22 TDT patients and the cells were transduced ex vivo with LentiGlobin BB305 vector. Patients received a 12.8 mg/kg of myeloablative busulfan followed by 5.2 to 18.1 CD34+ BB305+ cells/kg [52]. The transduction VCN was 0.7 copies. Approximately 26 months after the infusion process, almost all patients with a non-β0/β0 genotype stopped receiving transfusions [52]. Moreover, all biologic markers of dyserythropoiesis were corrected. The median annual transfusion volume decreased by 73% in 9 patients with a β0/β0 genotype, and transfusions were discontinued in 3 patients [52]. Adverse events from the treatment were similar to those associated with autologous stem-cell transplantation. The HGB 212 and HGB 207 trials (ClinicalTrials.gov numbers NCT03207009 and NCT02906202) are currently ongoing phase III studies using the BB305 vector for TDT subjects with a β0/β0 genotype and non-β0/β0 genotypes.