Gene/Protein Disease Symptom Drug Enzyme Compound
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Query: UMLS:C0021051 (immunodeficiency)
 71,517 document(s) hit in 31,850,051 MEDLINE articles (0.00 seconds)

In 1990, 7 hemophilia B patients were infected with human immunodeficiency virus type 1 (HIV-1) after exposure to a single common lot of clotting factor. The hypervariable regions V1 and V2 of the proviral env gene from the patients shared a homology between 97.5% and 100% at the time of seroconversion. To determine the in vivo diversification of these epidemiologically closely related virus strains, the patients were followed up in the early phase of HIV infection. Direct sequencing of the V1/V2 region in the env gene still revealed a very high degree of homology (96.5%-100%). In the case of the patient who showed the highest decrease of CD4+ cells, moderate genetic diversification of the virus was associated with a biological differentiation. The strain originally presenting two expressed substitutions displayed three more deviations 9 months after the first investigation (including one reversion to the consensus sequence). In addition, the virus that originally could not be cultivated could now be isolated as a low cytopathogenic agent. This study provides evidence that the high genetic homogeneity of HIV-1 observed at the time of seroconversion is maintained as a predominant consensus sequence in the following so-called latent phase of infection.
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PMID:Diversification of HIV-1 strains after infection from a unique source. 845 82

The safety and efficacy of a monoclonal antibody purified factor IX concentrate were evaluated in two continuing trials of 32 previously untreated patients with mild, moderate, or severe hemophilia B. Patients were evaluated every 2 weeks for 24 weeks and every 3 months thereafter for at least 1 year. No patients became positive for human immunodeficiency virus antibody or hepatitis C virus antibody during the trial. Two patients developed a false-positive hepatitis B core antibody, one transiently, but neither had elevated levels of alanine aminotransferase (ALT). None of the 25 patients evaluable for non-A, non-B, non-C hepatitis by strict International Society of Thrombosis and Hemostasis criteria developed elevated levels of ALT indicative of posttransfusion infection. Anaphylaxis occurred in one subject who also developed an inhibitor to factor IX (19.3 Bethesda units). Five of the eight adverse events reported (63%) were mild in severity, and the relationship of three of these to therapy was considered remote. Hemostasis with monoclonal antibody purified factor IX concentrate was excellent in all patients.
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PMID:Safety and efficacy of monoclonal antibody purified factor IX concentrate in previously untreated patients with hemophilia B. 871 76

Severe cellular immunosuppression developed in a 25-year-old man with hemophilia B who was infected with the human immunodeficiency virus (HIV). Four days after administration of sulfamethoxazole-trimethoprim (SMX-TMP) for prophylaxis against Pneumocystis carinii pneumonia (PCP), diffuse uptake of both lungs was confirmed on a 67Ga scintigram. Reticular shadows were also seen throughout both lung fields on a chest CT scan. These findings were compatible with PCP, according to the guidelines for presumptive diagnosis of the acquired immunodeficiency syndrome, published by the Centers for Disease Control and Prevention. The dose of SMX-TMP was increased, but interstitial pneumonitis worsened and was accompanied by fever, skin rash, and liver dysfunction, which are common in HIV-infected patients receiving SMX-TMP. No evidence of PCP or of any other opportunistic infection was found by bronchoalveolar lavage. Adverse reactions diminished after SMX-TMP administration was stopped. The 67Ga scintigram and chest CT findings also returned to normal. We concluded that the interstitial pneumonitis was induced by SMX-TMP. SMX-TMP is the first choice anti-PCP drug, but a high incidence of adverse reactions in patients with HIV infection has been reported. Therefore the possibility of SMX-TMP-related pulmonary toxicity must be considered in HIV-infected patients.
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PMID:[Sulfamethoxazole-trimethoprim-induced pneumonitis in a patient with hemophilia B who was infected with the human immunodeficiency virus]. 881 Jul 66

Prophylaxis has been practiced for many years in Europe and is gaining acceptance worldwide with current viral inactivation procedures. Unfortunately, the high cost of prophylaxis is currently the major obstacle to its implementation in developing countries such as Turkey. The aim of this controlled preliminary study is to evaluate the efficacy, safety, and feasibility of prophylaxis. Seven boys aged 1.5-7 years (5.0 +/- 1.8), who had severe hemophilia (six A, one B) received 20-50 IU/kg factor twice weekly and were followed up for 6-24 months (14.5 +/- 6.6). Intermediate concentrates have been used in hemophilia A and ultrapure product for hemophilia B. The data obtained for the same group of patients before prophylaxis were used as a control group. Another control group was selected in another group of 10 hemophiliacs, mean age 12.5, and received treatment on demand. During prophylactic treatment, the episodes of bleeding were decreased (from 10.5 +/- 3.2 to 4.5 +/- 3.6). Orthopedic and radiologic joint scores were stable (from 0 to 1 and from 1.1 +/- 1.2 to 1.0 +/- 1.5). The patients spent significantly fewer days in the hospital (from 18 +/- 12 to 0.7 +/- 0.6). None of the patients was infected with hepatitis A, hepatitis B, or human immunodeficiency virus. One patient was seroconverted with anti-hepatitis C virus in the third month of prophylaxis. Mean consumption of concentrates for prophylaxis was 3489 +/- 960 IU/kg per year compared with 2073 +/- 1302 in conventional therapy. Prophylaxis was superior to treatment on demand even when given in a twice-weekly period with intermediate concentrates. In Third World countries, prophylaxis should be tried at least in selected severely hemophilic children in order to prevent disabilities.
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PMID:Prophylactic therapy for hemophilia in a developing country, Turkey. 908 43

Twenty-three total knee arthroplasties in 15 patients with severe hemophilia were performed between February 1974 and September 1988. Thirteen patients had Factor VIII deficiency and two had Factor IX deficiency. The mean followup period was 7.5 years, with a minimum of 4 years for patients who were alive (eight) at the time of this review. Seven patients had died before this report, and all were seropositive for the human immunodeficiency virus. Using the Hospital for Special Surgery knee scoring system, the result was excellent in one knee, good in three, fair in two, and poor in 17. One patient was seropositive for the human immunodeficiency virus at the time of the index procedure, and 12 were seropositive at the most recent followup; the human immunodeficiency status of three patients was unknown. There were two early and two late deep infections, all in patients who were seropositive for the human immunodeficiency virus. The most recent postoperative radiographs for all knees were reviewed using the Knee Society radiographic scoring system. Ten femoral components were well fixed, 11 were possibly loose, and two were probably loose. Eight tibial components were well fixed, 10 possibly loose, three probably loose, and two definitely loose. One knee had been revised for aseptic loosening. There are few published studies of the long term results of total knee arthroplasties in patients with hemophilia. In this series of 23 knees, there was a high rate of loosening and infection. Total knee arthroplasty may be a useful treatment for the relief of pain attributable to end stage hemophilic arthropathy, but there is a high rate of complications, especially in patients who are seropositive for the human immunodeficiency virus.
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PMID:Knee arthroplasty in hemophilic arthropathy. 1010 22

Progressive multifocal leukoencephalopathy (PML) is a demyelinating disease resulting from lytic infection of oligodendrocytes by the papovavirus JC (JCV). PML has also been recognized as an AIDS-defining illness. The incidence of PML has increased since 1987 and it occurs in up to 4% of patients with AIDS. To date, there is no treatment available for PML and it usually results in death within 3-6 months of diagnosis. However, there are some reports of remission of PML after antiretroviral therapy. We report a 12-year-old child with hemophilia B and developing AIDS with the onset of PML. With highly active antiretroviral therapy, PML subsided with an increase of CD4 count from 10 to 300/microl in spite of about 1.0 X 10(4) human immunodeficiency virus (HIV)-1-RNA copies. He has survived more than 1 year without specific therapy against JCV. Highly active antiretroviral therapy appears to have improved his prognosis in HIV-associated PML.
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PMID:Remission of progressive multifocal leukoencephalopathy following highly active antiretroviral therapy in a patient with HIV infection. 1048 77

As of summer 2000, more than 400 protocols developed for human gene therapy have been reported, and there have been recent successful applications in some diseases such as arteriosclerosis obliterance, immunodeficiency X-1 (SCID-X1) and hemophilia B. However, complications have also occurred. Successful gene therapy is dependent on the development of an effective gene delivery system. One approach is development of chimeric vector systems that combine at least two different vector systems. However, a perfect vector system has not yet been constructed. Difficulties of in vivo gene transfer appear to result from resistance of living cells to invasion by foreign materials and from interference of cellular functions. We should reevaluate what barriers in tissues affect in vivo gene transfection and how to solve these problems for gene therapy. Moreover, in Japan, there should be more extensive preparation of social systems to promote clinical trials based on basic research.
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PMID:[Gene therapy: current status and promise]. 1133 80

Highly active anti-retroviral therapy(HAART) for human immunodeficiency virus(HIV) has delayed the disease progression. The advances prompted us to undertake liver transplantation in a 41-year-old man with hemophilia B, HIV infection, and hepatitis C(HCV) end-stage liver disease. The donor was the patient's elderly brother. His right liver was implanted by the standard method. Two months after the operation, interferon alfa and ribavirin were administered for HCV infection. HCV was eradicated two weeks after the treatment. The HIV viral load is persistently negative and HAART has not been started so far. Utilizing a organ from living relatives should be one of the options to resolve the concern around the utilization of a scarce public source from cadavers for patients who may not have an equivalent survival to HIV negative patients.
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PMID:[Living donor liver transplantation in a patient with HIV]. 1196 92

Recent studies have shown that nondividing primary cells, such as hepatocytes, can be efficiently transduced in vitro by human immunodeficiency virus-based lentivirus vectors. Other studies have reported that, under certain conditions, the liver can be repopulated with transplanted hepatocytes. In the present study, we combined these procedures to develop a model system for ex vivo gene therapy by repopulating rat livers with hepatocytes and hepatoblasts transduced with a lentivirus vector expressing a reporter gene, green fluorescent protein (GFP). Long-term GFP expression in vivo (up to 4 months) was achieved when the transgene was driven by the liver-specific albumin enhancer/promoter but was silenced when the cytomegalovirus (CMV) enhancer/promoter was used. Transplanted cells were massively amplified ( approximately 10 cell doublings) under the influence of retrorsine/partial hepatectomy, and both repopulation and continued transgene expression in individual cells were documented by dual expression of a cell transplantation marker, dipeptidyl peptidase IV (DPPIV), and GFP. In this system, maintenance or expansion of the transplanted cells did not depend on expression of the transgene, establishing that positive selection is not required to maintain transgene expression following multiple divisions of transplanted, lentivirus-transduced hepatic cells. In conclusion, fetal hepatoblasts (liver stem/progenitor cells) can serve as efficient vehicles for ex vivo gene therapy and suggest that liver-based genetic disorders that do not shorten hepatocyte longevity or cause liver damage, such as phenylketonuria, hyperbilirubinemias, familial hypercholesterolemia, primary oxalosis, and factor IX deficiency, among others, might be amenable to treatment by this approach.
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PMID:Repopulation of rat liver by fetal hepatoblasts and adult hepatocytes transduced ex vivo with lentiviral vectors. 1271 80

The use of plasma-derived factor products to treat hemophilia A, hemophilia B, and von Willebrand disease (vWD) has changed since the start of the human immunodeficiency virus (HIV) epidemic. The use of plasma-derived factor concentrates for hemophilias A and B has decreased in developed countries because of the availability of recombinant products. However, in developing countries, which encompass most of the world's hemophilia community, plasma-product-based therapy remains the backbone of treatment because of economic constraints. Viral attenuation strategies have resulted in a much safer product profile. vWD product selection is less complicated than for hemophilas A and B because plasma-derived products are the only choice for patients who are unresponsive or who cannot receive pharmacologic therapy. As the majority of patients in the world with hemophilias A, B and vWD are treated with plasma-derived clotting factors, the need for these safe and efficacious therapies will continue in the future. This chapter discusses safety strategies for plasma-derived clotting factor, its availability, economics, efficacy, and inhibitor formation.
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PMID:Clinical uses of plasma and plasma fractions: plasma-derived products for hemophilias A and B, and for von Willebrand disease. 1637 40


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