Immunological reconstitution after HLA-haploidentical bone marrow transplantation for SCID in an infant with ADA deficiency.
Explore the source record for details and available documents.
Biomedical subjects
Publications and source records attributed to L Nespoli.
Explore the source record for details and available documents.
Explore the source record for details and available documents.
Explore the source record for details and available documents.
A double blind vs placebo study was carried out to study the effect of letosteine on the symptoms and clinical course of paediatric patients suffering from acute febrile bronchitis. Forty children were recruited for the research: 20 were treated with letosteine in a dose of 25 mg x 3 g/die and 20 with placebo; treatment lasted 10 days. The following parameters were assessed during the trial: body temperature, cough, thoracic objectivity, respiratory function indices. The results of the study show that in the letosteine treated group there is a statistically significant decrease in fever, a favourable evolution of thoracic objectivity and an improvement in certain respiratory function parameters (MEF 75, PEF). It is concluded that treatment with letosteine leads to a significant increase in the rate of regression of thoracic symptomatology and a faster, more substantial reduction in fever in children suffering from acute bronchitis. This is probably the result of drug action on mucus viscosity, restoring optimal mucociliary clearance, and through action fostering the penetration of antibacterial substances into the mucus.
Explore the source record for details and available documents.
Explore the source record for details and available documents.
Peripheral blood mononuclear cells (PBMC) from 16 non-institutionalized patients with Down syndrome (DS) were studied with various monoclonal antibodies and analysed for natural killer (NK), and NK-like activity. Lymphocyte proliferation and cytotoxic T-lymphocyte (CTL) cytotoxicity generated in mixed lymphocyte culture (MLC) were also evaluated in 11 DS patients. Phenotypic characterization of PBMC from DS subjects confirms our previous findings of high numbers of CD8+ lymphocytes and HNK-1+, and CD16+ cells. Lymphocyte proliferation and CTL cytotoxicity generated in MLC were low or absent in most patients. NK activity was low in almost all DS patients, while NK-like cytotoxicity generated in MLC was normal in the majority and did not correlate with NK activity from unstimulated PBMC.
We studied the cytochemical distribution of tetrahydrofolate dehydrogenase (FH4D), an enzyme involved in nucleic acid metabolism and thus in cell proliferation and differentiation processes, in bone marrow blasts from 37 cases of acute lymphoblastic leukemia (ALL), of whom 23 were pediatric patients. 26 cases were analyzed at onset, 11 in relapse. The ALL cases were immunologically classified as T (10), common (20), B (3) and null (4). In each subgroup the majority of lymphoblasts were positive, with heterogeneous positivity patterns and variable degrees of enzyme activity. Most T lymphoblasts were characterized by focal localization of FH4D, whereas in common blasts reactivity - usually less strong - was either focally localized or scattered with several fine granules. Finally, many B and null blasts showed diffuse positivity. A quantitative evaluation of FH4D activity using cytophotometric technique (Vickers M86) demonstrated higher degrees of reactivity in leukemic blasts than in normal lymphocytes. Moreover, slightly different levels of reactivity were observed in relation to immunological phenotype, age and stage of the disease. Therefore we think that FH4D is a useful additional marker for ALL characterization.
Ten children with the characteristic clinical and haematological features of haemophagocytic lymphohistiocytosis are reported. Four patients treated with a combination of drugs comprising etoposide, methotrexate, and steroids were in complete remission after 10 to 30 months. Natural cytotoxic mechanisms including natural killer cell activity, antibody dependent cell mediated cytotoxicity, lymphokine activated killer cell activity, and natural killer cell like activity were persistently absent or severely impaired in these four patients despite their clinical remission. Their parents and one healthy sibling also had impaired natural cytotoxic mechanisms. Constitutional impairment of natural cytotoxic mechanisms could be important in the pathogenesis of haemophagocytic lymphohistiocytosis.
Cell-mediated cytotoxicity is considered to play a major role in immune defense and in particular in the killing of virus-infected and neoplastic cells. It appears to have some interesting implications when considering the infectious risk of acute lymphoblastic leukemia (ALL) children during immunosuppressive chemotherapy and the role of self-defense against minimal residual disease. We have studied natural killer (NK) activity and lymphokine-activated killer (LAK) activity in children during and after treatment for ALL. We observed that peripheral blood mononuclear cells in 22 children undergoing maintenance chemotherapy displayed significantly depressed NK activity compared with normal controls even when the proportion of NK cells was normal. LAK activity was also considered in 43 ALL children during and after maintenance chemotherapy. We observed that LAK activity was persistently comparable with that of normal controls. It seems definite that NK activity impairment is transient and is completely restored in ALL children a few months after chemotherapy has been successfully completed. The evidence that LAK activity is not impaired in ALL children may have some implications in view of a possible immunomodulatory approach in the presence of refractory disease.
Anti-streptolysin O (ASO) antibodies are an expression of the frequent encounters even during the first years of life with beta hemolytic Streptococci, they are easily measured by quantitative laboratory tests and so possess the characteristics of a screening test for impaired antibody production. We have assessed ASO titers in 1955 healthy subjects of different ages (range: 1 month to 97 years). The behaviour of ASO titer is extensively described in the text. The percentage of people with less than 10 TU titers is under 5% after the age of 5 years up to 15 years; from 15 to 60 years there are no subjects with undetectable ASO titer and after this age the percentage is still under 5%. It seems therefore advisable not to define a "normal" treshold when titrating these antibodies to avoid the risk of missing low antibody producers.
Explore the source record for details and available documents.
Explore the source record for details and available documents.
Explore the source record for details and available documents.
One hundred thirty-three children with acute myelogenous leukemia (AML) entered the multicenter Pediatric Branch of the Italian Association Against Leukemia trial AIEOP/LAM 8204 between July 1982 and May 1986. Induction therapy consisted of two courses of daunomycin (DNM) plus cytosine arabinoside (Ara-C). Those patients who achieved remission were given four courses of consolidation with DNM, 6-thioguanine (6-TG) and escalated doses of Ara-C followed by six courses of sequential continuation therapy using monthly pairs: etoposide (VP-16)/Ara-C, Ara-C/6-TG, and DNM/Ara-C. Periodic intrathecal Ara-C was used for CNS prophylaxis. One hundred seven (80%) children achieved complete remission (CR). Kaplan-Meier estimates of 3-year disease-free survival (DFS) and event-free survival (EFS) are 41% and 33%, respectively. Relapses occurred in 34 patients after 5 to 97 weeks (32 marrow; 2 marrow plus CNS). Overall, 14 patients died of complications during treatment (nine during induction; five during the postremission phase), mostly from infection. Risk factor analysis showed that induction failures occurred predominantly in children with French-American-British (FAB) M5 and in those with elevated leukocyte counts; by step-up Cox analysis, only FAB subtype was predictive of remission success. None of the variables examined was significant for predicting the duration of remission. Hyperleukocytosis was predictive of a significantly worse EFS rate. These results are encouraging and further support the use of intensive chemotherapy programs for childhood AML.
Explore the source record for details and available documents.
Fourteen scans employing the adrenergic blocking agent [131I]MIBG were performed on 10 children with neuroblastoma (NB) or ganglioneuroblastoma (GNB). The scans were negative in 5 cases, and 1 further case produced doubtful results in both the MIBG and CT scan tests. In 4 cases, very positive results were obtained with clear vision of the primary tumor and its metastases. In 1 case, which demonstrated partial differentiation of the outer part of the tumor mass toward GNB, a differentiated tumor specimen did not reveal significant uptake of the tracer. Half-lives of the tracer as measured by external detection in the period 24-48 h after injection were reduced after successful therapy. MIBG scanning appears to be a feasible indicator of NB adrenergic activity, and it can assume a primary role in the staging and follow-up of NB. Higher tumor uptake of the [131 I]MIBG and low background offer new perspectives in the radiometabolic treatment of MIBG.
The aim of the study was to test the antipyretic effect and the tolerability of alpha-methyl-4-(2-thienyl-carbonyl)phenylacetic acid (suprofen, Suprol) syrup in a sample of pediatric patients with fever of various etiology. The study was designed as an open multiple dose study including 41 hospitalized children (males and females) aged 6 months to 12 years. Temperature, pulse rate, and respiratory rate were measured immediately after consumption of the syrup, after 30 min, 1 h, 1 1/2, 2, 3, 4, 5, and 6 h. The study was performed at one investigational center. At the end of the study, data were evaluated for a total of 41 patients. 27 patients were 5 years or less, 14 patients were 6 to 12 years old. The mean age of the males (23 patients) was 4.5 years; the mean age of the females (18 patients) was 3.8 years. There was no significant sex dependence of the development of temperature, the pulse rate, and the respiratory rate. Prior to treatment the mean temperature was 38.6 degrees C. Temperature decreased to 37.7 degrees C within 1 h and to 37.2 degrees C within 2 h after treatment. From then on, the temperature increased to 37.7 degrees C at the end of the observation period. The decrease in temperature (compared to baseline) was significant throughout the trial. The mean pulse rate decreased from 131.2/min to 119.9/min within 1 h, and to 114.0/min within 2 h. At the end of the trial the mean pulse rate was 120.1/min.(ABSTRACT TRUNCATED AT 250 WORDS)