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Biomedical subjects

R M Simon

Publications and source records attributed to R M Simon.

At least 19 recordsLinked to original sources

Comparison of in vitro anticancer-drug-screening data generated with a tetrazolium assay versus a protein assay against a diverse panel of human tumor cell lines.

The National Cancer Institute (NCI) is implementing a large-scale in vitro drug-screening program that requires a very efficient automated assay of drug effects on tumor cell viability or growth. Many laboratories worldwide have adopted a microculture assay based on metabolic reduction of 3-(4,5-dimethylthiazol-2-yl)-2,5-diphenyltetrazolium bromide (MTT). However, because of certain technical advantages to use of the protein-binding dye sulforhodamine B (SRB) in a large-scale screening application, a detailed comparison of data generated by each type of assay was undertaken. The MTT and SRB assays were each used to test 197 compounds, on simultaneous days, against up to 38 human tumor cell lines representing seven major tumor categories. On subsequent days, 38 compounds were retested with the SRB assay and 25 compounds were retested with the MTT assay. For each of these three comparisons, we tabulated the differences between the two assays in the ratios of test group values to control values (T/C) for cell survival; calculated correlation coefficients for various T/C ratios; and estimated the bivariate distribution of the values for IC50 (concentration of drug resulting in T/C values of 50%, or 50% growth inhibition) for the two assays. The results indicate that under the experimental conditions used and within the limits of the data analyses, the assays perform similarly. Because the SRB assay has practical advantages for large-scale screening, however, it has been adopted for routine use in the NCI in vitro antitumor screen.

Antineoplastic Agents

A method for the evaluation of dose-toxicity relationships in clinical trials.

This paper considers a proportional hazards model that describes the relationship between time-dependent cumulative dose of drug and development of toxicity. We estimate probabilities of developing toxicity in both the presence and the absence of competing risks and provide variances for the latter case. Mitoxantrone data collected in Southwest Oncology Group studies illustrate the methods.

Adult

Cardiotoxicity in patients treated with mitoxantrone: Southwest Oncology Group phase II studies.

A model describing the development of cardiotoxicity as a function of cumulative dose of a drug and other covariates is presented. Methods are given for testing and illustrating a cumulative dose effect. Also, equations for estimation of the probability of developing cardiotoxicity in the presence or absence of competing risks are given. The methods are illustrated for mitoxantrone by means of data obtained from Southwest Oncology Group studies.

Dose-Response Relationship, Drug

The pathologic and clinical heterogeneity of lymphocyte-depleted Hodgkin's disease.

Patients with Hodgkin's disease of the lymphocyte-depleted subtype (LDHD) have been said to have a poor prognosis. However, reports of this subtype are complicated by the fact that the histologic diagnosis of LDHD is often not straightforward, and its distinction from aggressive non-Hodgkin's lymphomas (NHL) can be difficult. We have reviewed our patients with LDHD at the National Cancer Institute (NCI) in light of an additional decade of experience with neoplastic and non-neoplastic conditions mimicking Hodgkin's disease. Of 198 patients who received MOPP (mechlorethamine, vincristine, procarbazine, prednisone) treatment at the NCI for Hodgkin's disease between 1964 and 1976, 43 (22%) were originally classified as LDHD. The initial diagnostic biopsies from 39 of these patients were reviewed and revealed ten with NHL, nine with LDHD, and 13 with nodular sclerosing Hodgkin's disease of the lymphocyte-depleted subtype (NSLD). The other seven patients had Hodgkin's disease without a lymphocyte-depleted component. The NHL patients were further subclassified as diffuse, large-cell (two cases) and large-cell, immunoblastic (eight cases). The pathologic review was done without knowledge of clinical features which were examined after review in the three major subgroups. Of ten patients with NHL, only three had a complete remission (CR), and median survival was 7 months. Nine of the NHL patients presented with features that are unusual for patients with Hodgkin's disease, such as bulky abdominal disease, epitrochlear lymphade-nopathy, or hypercalcemia. CRs were attained by 67% and 85% of patients in the LDHD and NSLD groups, respectively: median survival had not been reached in either group with a median of 14 years of follow-up. Lymphocyte-depleted Hodgkin's disease, adequately treated, is in our limited group of patients no worse than other histopathologic subtypes of Hodgkin's disease. The erroneous inclusion of patients with high-grade NHLs into this subtype of Hodgkin's disease may be one reason for earlier literature reports of its more aggressive nature. The diagnosis of LDHD should be made cautiously, particularly in patients with clinical features that are unusual for Hodgkin's disease at presentation.

Adolescent

Efficiency of antitumor screening relative to activity criteria.

Well over 100,000 compounds have been screened by the National Cancer Institute (NCI) in mouse leukemia P388 in the search for new leads toward antineoplastic agents. The protocol for screening requires confirmation testing of compounds showing initial activity. By activity criteria being varied for initial and confirmation testing, it is possible to improve the efficiency of screening. The experimental variability of P388 screening was first determined by analysis of outcomes from 90 tests on each of 21 compounds of varied activity. By application of these results to NCI P388 data for 31,428 compounds adequately tested on a daily times five schedule, the consequences of using different activity thresholds were estimated. The results here may apply to those of other similar large-scale screening programs.

Animals

Antiemetic studies: a methodological discussion.

Methodological issues encountered in reviewing the antiemetic literature are discussed. Variables affecting antiemetic assessment include those relating to the patient, the emetic stimulus, and the differences in action of the antiemetic drugs. Study design questions including the desirability of randomization, double blinding, use of controls, and stratifications for factors such as previous exposure to chemotherapy and strength of the emetic stimulus are discussed. From a statistical viewpoint a parallel subject design with adequate sample size is recommended. The problems of standardizing the evaluation of nausea and vomiting are considered and simple ordinal scales grading severity are suggested. The patient's overall assessment of tolerance of therapy is of primary importance.

Antiemetics

Application of a human tumor colony-forming assay to new drug screening.

The applicability of a human tumor colony-forming assay to drug screening was investigated in terms of feasibility, validity, and potential for discovering new antitumor drugs. Feasibility was addressed in a pilot study during which basic methods, appropriate assay quality controls, and a standardized protocol for screening were developed. Considerable variability was noted in the availability and colony growth of different tumor types. The majority of the evaluable experiments utilized breast, colorectal, kidney, lung, melanoma, or ovarian tumors. For many tumor types, little evidence of growth was observed, or only rare specimens formed colonies. Colony-forming efficiencies ranged from 0.05 to 0.11% for the six most useful tumors listed above. A set of quality control measures was developed to address technical problems inherent in the assay. Testing of standard agents in the pilot study established that most of these agents could be detected as active. However, it also identified three assay limitations: compounds requiring systemic metabolic activation are inactive; medium constituents may block the activity of certain antimetabolites; and compounds without therapeutic efficacy may be positive in the assay. The assay categorized nontoxic clinically ineffective agents as true negatives with 97% accuracy. Of 79 compounds which were negative in the current National Cancer Institute prescreen (leukemia P388), 14 were active in the assay. Several demonstrated outstanding in vitro activity and are structurally unrelated to compounds already in development or in clinical trials. A subset of these active compounds were found to lack activity in a P388 in vitro colony-forming assay. This indication of differential cytotoxicity to human tumor cells makes this subset of compounds particularly interesting as antitumor drug leads. The demonstrated sensitivity to most standard agents, discrimination of nontoxic compounds, reproducibility of survival values within assays and between laboratories, and evidence of ability to identify active compounds which were negative in the in vivo prescreen suggest that the human tumor colony-forming assay may be a valuable tool for antitumor drug screening. However, because of technical limitations inherent in the current assay methodology, this must be confined to selected tumor types and limited to screening on a moderate scale.

Antineoplastic Agents

Cryptococcal meningitis and internal ophthalmoplegia.

A 17-year old girl received prednisone and azathioprine for the treatment of systemic lupus erythematosus. She developed a fever and hallucinations 18 months later; cryptococcal meningitis was diagnosed. An internal ophthalmoplegia with loss of accommodation and dilation of the pupils developed together with bilateral lateral rectus palsy. Treatment with intravenous amphotericin resulted in disappearance of papilledema, muscle palsy, and internal ophthalmoplegia. We believe that the internal ophthalmoplegia was secondary to involvement of the accommodative and pupillary fibers of both third nerves at the base of the brain.

Adolescent

Delata-9-tetrahydrocannabinol as an antiemetic in cancer patients receiving high-dose methotrexate. A prospective, randomized evaluation.

Fifteen patients with osteogenic sarcoma receiving high-dose methotrexate chemotherapy were studied in a randomized, double-blind, placebo-controlled trial of oral and smoked delta-9-tetrahydrocannabinol (THC) as an antiemetic. Each patient served as his or her own control. Fourteen of 15 patients had a reduction in nausea and vomiting on THC as compared to placebo. Delta-9-tetrahydrocannabinol was significantly more effective than placebo in reducing the number of vomiting and retching episodes, degree of nausea, duration of nausea, and volume of emesis (P less than 0.001). There was a 72% incidence of nausea and vomiting on placebo. When plasma THC concentrations measured less than 5.0 ng/mL, 5.0 to 10.0 ng/mL, and greater than 10.0 ng/mL, the incidences of nausea and vomiting were 44%, 21%, and 6%, respectively. Delta-9-tetrahydrocannabinol appears to have significant antiemetic properties when compared with placebo in patients receiving high-dose methotrexate.

Adolescent

Histologic grade in advanced ovarian cancer.

The histologic grade of epithelial ovarian tumors was found to be a major prognostic factor for survival in patients with advanced (stage III-IV) disease. In addition, a grading system based on cytologic detail (modified Broders' grades I-IV) identified groups with a differential response to chemotherapy. The overall improvement in survival observed with combination chemotherapy was related primarily to an increased survival of patients with grade II and III lesions. Although the survival of patients with grade I lesions was markedly better than for patients with grade IV lesions, in neither case was it influenced by the choice of single-agent or combination chemotherapy. In prospective clinical trials in advanced disease, modified Broders' grades should be included as a separate stratification factor.

Altretamine

Treatment of osteogenic sarcoma. II. Aggressive resection of pulmonary metastases.

Thirty-nine patients with osteogenic sarcoma were treated in a prospective protocol involving the use of adjuvant high-dose methotrexate, frequent screening for pulmonary metastases, and aggressive resection of all metastatic disease whenever possible. Twenty-two of these 39 patients have had recurrence and in 20 patients evidence of metastatic disease was confined to the lungs. Eighteen of the patients had thoracotomy, and in 11 patients all known disease was resected. Although four of these patients have required further thoracotomies, all 11 patients have no evidence of disease. Thus, of the original 39 patients, 30 (76.9%) are now alive and 28 (71.8%) have no evidence of disease, with a median followup of 27 months. Survival is significantly improved compared to historical control patients (P less than 0.001; one-sided Kruskal-Wallis test).

Antineoplastic Agents

Treatment of osteogenic sarcoma. I. Effect of adjuvant high-dose methotrexate after amputation.

Thirty-nine patients with extremity osteogenic sarcoma and no discernible metastases were treated with amputation and postoperative adjuvant high-dose methotrexate with leucovorin rescue. One half of the patients were also randomized to receive bacillus Calmette-Guérin by a multiple-puncture technique. Results have been analyzed with a minimum followup of 10 months and a median followup of 27 months. Actuarial analysis estimates that 38% of current protocol patients remain continuously free of disease for 24 months compared to only 17.4% of historical control patients (P = 0.029; one-sided generalized Kruskal-Wallis test). Bacillus Calmette-Guérin administered by a multiple-puncture technique had no effect on disease-free interval. Minor differences between current protocol and historical control patients with regard to race, age, histologic type, and site and size of primary tumors do not affect the difference in disease-free interval between these two patient groups. However, current patients had somewhat lower grade lesions and consideration of the patients with grade III and IV lesions only, lessens the difference between current and historical control patients (P = 0.11; one-sided generalized Kruskal-Wallis test). High-dose methotrexate was administered with virtually no morbidity and no deaths. The small differences observed in this study between protocol patients treated with surgery plus high-dose methotrexate and historical control patients treated with surgery alone point to the need for a prospective randomized study to establish the role of high-dose methotrexate in the adjuvant treatment of patients with osteogenic sarcoma.

Adolescent

Advanced ovarian adenocarcinoma. A prospective clinical trial of melphalan (L-PAM) versus combination chemotherapy.

Eighty patients with advanced ovarian adenocarcinoma were treated in a prospective, randomized trial comparing a four-drug combination--hexamethylmelamine, cyclophosphamide, methotrexate and 5-fluorouracil--with the oral alkylating agent, melphalan. Treatment with the four-drug combination was associated with a significantly increased overall response rate (75 vs. 54 per cent) (P less than 0.05), more complete remissions (33 vs. 16 per cent) and longer median survival (29 vs. 17 months) (P less than 0.02) but more severe toxicity than occurred with melphalan. Patients with minimal residual disease had a significantly higher overall response rate than patients with extensive residual disease (84 vs. 53 per cent) (P less than 0.05). Patients with advanced disease who achieved a complete remission documented by peritoneoscopy or laparotomy (or both) have a median survival that will exceed three years. The four-drug regimen is more effective than melphalan in the management of advanced ovarian adenocarcinoma.

Adenocarcinoma

Increasing incidence of Gram-positive sepsis in cancer patients.

A review of the agents causing septicemia in cancer patients during a nine-year period (1968--76) revealed that Gram-positive organisms (especially S aureus) have become the most frequent isolates during the last two years. This unanticipated increase (P less than 0.001) in the number of Gram-positive isolates could not be related to horizontal transmission nor to changes in patient characteristics or therapy.

Adolescent

Failure of immunotherapy with neuraminidase-treated tumor cell vaccine in mice bearing established 3-methylcholanthrene-induced sarcomas.

C3H/HeJ mice bearing MC-80 fibrosarcomas were given immunotherapy consisting of multiple injections of a Vibrio cholerae neuraminidase (VCN)-treated tumor cell vaccine at a site remote from the established tumor. In five separate experiments we were unable to show either partial or complete tumor regression or prolongation of survival for vaccine-treated mice compared to appropriate controls. Further, the use of BCG in addition to VCN-treated tumor cells failed to show any therapeutic efficacy. We could not confirm the successful immunotherapy results reported by others despite multiple efforts of reproduce the immunotherapy model as carefully and precisely as possible.

Animals