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Recombinant DNA Technology and Gene Therapy Using Viruses
Published in Patricia G. Melloy, Viruses and Society, 2023
There are different gene therapy approaches. For example, a patient’s cells may be removed, modified using the viral vector with the gene insert (therapeutic transgene), and then reintroduced into the body. This is known as the ex vivoapproach. Alternatively, the viral vector with the gene insert could be introduced directly to an organ or systemically to modify the target cells within the patient’s body. This is known as the in vivo approach (Figure 7.2). (Mietzsch and Agbandje-McKenna 2017; Colavito 2007; Minkoff and Baker 2004; Kurreck and Stein 2016). It should be recognized that the ex vivo gene therapy approach is built on knowledge gained from successful bone marrow transplants using patient-matched hematopoietic (blood) stem cell donors. This is known as allogeneic bone marrow transplantation. With gene therapy, now the patient’s own cells can be modified and given back to them in an approach known as an autologous bone marrow transplantation (Dunbar et al. 2018).
Acute Leukemia and Myelodysplastic Syndromes
Published in Harold R. Schumacher, William A. Rock, Sanford A. Stass, Handbook of Hematologic Pathology, 2019
Mark D. Brissette, James D. Cotelingam
Allogeneic bone marrow transplantation is usually reserved for patients in second remission (remission after relapse) and possibly for those with refractory leukemia, especially if they are young (<30 years). Autologous bone marrow reinfusion (marrow removed from the patient during remission) may be a consideration for selected patients.
Practice Paper 5: Answers
Published in Anthony B. Starr, Hiruni Jayasena, David Capewell, Saran Shantikumar, Get ahead! Medicine, 2016
Anthony B. Starr, Hiruni Jayasena, David Capewell
Allogeneic bone marrow transplantation is curative for some haematological malignancies. Patients are initially ‘conditioned’ (i.e. have their own bone marrow killed by cyclophosphamide and total body irradiation). Healthy stem cells collected from a donor are then injected into the patient. It takes around 4 weeks for the patient to make enough cells from the transplanted stem cells. There is an upper age limit of 55 years for bone marrow transplantation. Complications include infection, graft-versus-host disease, pneumonitis and secondary malignancy. In some cases, autologous bone marrow transplantation is undertaken. In this case, the patient’s own marrow is harvested and given back as a ‘rescue’ after chemotherapy. Autologous marrow transplantation is used only in patients with good remission, as there is a higher risk of relapse.
Treatment outcomes of adult Burkitt lymphoma: results with a modified LMB protocol in Brazil and feasibility of outpatient administration
Published in Journal of Chemotherapy, 2018
Wellington Fernandes da Silva Junior, Lidiane Inês da Rosa, Marcelo Belesso, Luís Alberto P. C. Lage, Vanderson Rocha, Juliana Pereira
The median follow-up was 6 years (range, 4.2–7.8 y), and another 2/32 patients died during this period, one from multiple myeloma that developed 13 years after BL diagnosis and another from therapy-related myeloid neoplasm, developed 7 years after BL diagnosis. Another patient also developed therapy-related myeloid neoplasm about 5 years after diagnosis, but that patient underwent an allogeneic bone marrow transplantation and has been in complete remission since that time. The estimated 5-year OS was 89% (95% confidence interval [CI], 73–96%) for patients treated with mLMB and 79% (CI 95%, 58–90%) for all HIV-negative patients. Only age at diagnosis (above 35 years, p = 0.002) was a risk factor to death in univariate analysis, of all variables studied (sex, CNS, bone marrow or gastrointestinal disease, and staging were also included) (Figure 1).
Advances in new drug therapies for the management of sickle cell disease
Published in Expert Opinion on Orphan Drugs, 2018
Kenneth I. Ataga, Payal C. Desai
Despite the success of allogeneic bone marrow transplantation and the curative potential of gene therapy, these treatments are not widely available, especially in resource-poor countries. The development of drug therapies for SCD that are safe, effective, and affordable is highly warranted. The increased number of drugs in testing offers hope for the availability of more pharmacologic therapies for SCD. With its complex pathophysiology, it is unlikely that individual drugs will ameliorate all of the complications of this disease. The availability of multiple drugs offers an opportunity for individualized therapy based on the presence of SCD-related complications, as well as for combination drug therapy based on different mechanisms of action and side effect profiles. It is important that clinical trials of novel drugs for SCD involve individuals in resource-poor countries, where the burden of disease is high. In addition to acute pain episodes, evaluation of the effect of novel agents on other SCD-related clinical complications, PROs, exercise capacity and validated surrogate endpoints is warranted.
Pharmacological treatment of pediatric Gaucher disease
Published in Expert Review of Clinical Pharmacology, 2018
Punita Gupta, Gregory Pastores
Prior to the introduction of ERT, patients with GD underwent symptomatic management including splenectomy and orthopedic surgery. A few patients underwent allogeneic bone marrow transplantation. Splenectomy was indicated in patients with massive splenomegaly and severe hypersplenism, poor nutritional status secondary to early satiety, growth retardation in children and various mechanical or vascular complications [36]. After splenectomy, most patients developed bony complications (typically osteonecrosis of large joints), progressive hepatomegaly with a tendency to abnormal liver function (and in severe cases, even cirrhosis), and a higher risk of death from septicemia [37,38]. Following the introduction of ERT, splenectomy is almost never needed and should be avoided [39].