Potential uses of recombinant human granulocyte-macrophage colony-stimulating factor in children.

Granulocyte-macrophage colony-stimulating factor (GM-CSF) is a glycoprotein necessary for the growth, differentiation, and function of cells of the granulocyte-macrophage lineage. This agent has been molecularly cloned and expressed in bacteria, yeast, and mammalian cell lines, providing a limitless source of the pure human protein for use in children. Here, we briefly review the discovery, biologic characterization, gene identification and cloning, and clinical experience to date with this new agent, focusing where possible on the use of recombinant GM-CSF in children.     

Vitamin K status in cystic fibrosis

Appearance of PIVKA-II (protein induced by vitamin K absence-II) in serum is a biochemical sign of insufficient vitamin K-dependent carboxylation of prothrombin. Plasma concentrations of PIVKA-II and vitamin K1 were determined in 24 children with cystic fibrosis. Eight were supplemented with vitamin K1. The purpose of the study was to determine the occurrence of vitamin K deficiency in cystic fibrosis and to evaluate the effect of vitamin K supplementation. PIVKA-II was detectable in only one unsupplemented child. In this patient, the concentration of vitamin K1 was below the limit of detection of 60 ng/l. Vitamin K1 levels in the other unsupplemented children were normal (mean 476 ng/l = 1 mmol/l). The supplemented patients showed extremely high levels of vitamin K1 (mean 22445 ng/l = 50 nmol/l). In conclusion, vitamin K deficiency occurs infrequently in cystic fibrosis. Checking the coagulation system is advised, but routine vitamin K supplementation is not recommended. If additional vitamin K is needed, the starting dose should not exceed 1 mg daily.     

Exenteration in benign orbital pathology: report of a case

PURPOSE, MATERIAL AND METHOD: Orbitary exenteration is an indicated treatment for malignant and benign processes where special incidence of extension, uncontrollable pain, unrecoverable visual loss, cosmetic disfiguration, tendency to diffuse or persistent infiltration and malignancy potential have been observed. We report a case of a 22 year-old male from Senegal, presenting an isolated neurofibroma of the orbit of long evolution and great size. RESULTS/CONCLUSIONS: Treatment of this tumor is complete local resection. In this case an orbitary exenteration was performed due to size of the tumor and visual loss. We emphasize the need to treat these patients as soon as possible.     

Psychiatric morbidity and impact on hospital length of stay among hematologic cancer patients receiving stem-cell transplantation.

PURPOSE: To determine the prevalence of psychiatric disorders during hospitalization for hematopoietic stem-cell transplantation (SCT) and to estimate their impact on hospital length of stay (LOS). PATIENTS AND METHODS: In a prospective inpatient study conducted from July 1994 to August 1997, 220 patients aged 16 to 65 years received SCT for hematologic cancer at a single institution. Patients received a psychiatric assessment at hospital admission and weekly during hospitalization until discharge or death, yielding a total of 1,062 psychiatric interviews performed. Psychiatric disorders were determined on the basis of the Diagnostic and Statistical Manual of Mental Disorders, Fourth Edition. Univariate and multivariate linear regression analyses were used to identify variables associated with LOS. RESULTS: Overall psychiatric disorder prevalence was 44.1%; an adjustment disorder was diagnosed in 22.7% of patients, a mood disorder in 14.1%, an anxiety disorder in 8.2%, and delirium in 7.3%. After adjusting for admission and in-hospital risk factors, diagnosis of any mood, anxiety, or adjustment disorder (P =.022), chronic myelogenous leukemia (P =.003), Karnofsky performance score less than 90 at hospital admission (P =.025), and higher regimen-related toxicity (P <.001) were associated with a longer LOS. Acute lymphoblastic leukemia (P =.009), non-Hodgkin's lymphoma (P =.04), use of peripheral-blood stem cells (P <.001), second year of study (P <.001), and third year of study (P <.001) were associated with a shorter LOS. CONCLUSION: Our data indicate high psychiatric morbidity and an association with longer LOS, underscoring the need for early recognition and effective treatment.     

Sexual precocity: sex incidence and aetiology

The aetiology of 197 girls and 16 boys presenting with sexual precocity was reviewed. Ninety one girls and four boys had central precocious puberty (M:F 23:1); a cause was identified in all the boys but in only six girls. All boys with precocious puberty need detailed investigation; in girls investigation should be based on clinical findings, particularly the consonance of puberty.     

Thymic cysts in mediastinal Hodgkin disease

Three cases of proved thymic cysts associated with mediastinal Hodgkin disease are presented. Two illustrate regression of lymphoma with chemotherapy but persistence of thymic cysts. The third case demonstrates a thymic cyst in untreated Hodgkin disease. These cases suggest that such cysts are probably neither coincidental with nor a consequence of therapy but are probably related to initial thymic involvement by Hodgkin disease.     

Granulopoiesis in chronic myeloproliferative disorders in children.

Four children with chronic myeloproliferative disorders (three with Philadelphia [Ph1] chromosome-positive chronic myelogenous leukemia [CML] were studied with soft agar culture at diagnosis (before therapy) in an attempt to define abnormalities in granulopoiesis. The three patients with CML had elevated peripheral blood golony-forming cells (CFCs) and/or normal or decreased bone marrow CFCs (in those studied). Colony-stimulating activity (CSA) was markedly decreased or absent at diagnosis in all three. Maturation of myeloid cells eithin the colonies in agar was normal, indicating that no block in myeloid maturation was present. These findings are in general agreement with results previously reported in untreated adults with Ph1 chromosome-positive CML and further define the similarity with the adult form of the disease. One Ph1 chromosome-negative patient with a clinically similar chronic myeloproliferative disorder was studied and had similarly elevated peripheral blood CFCs. She had normal CSA with a similarly high WBC count. This finding was unexpected and suggests that, unlike the patients with CML, her monocytes were capable of elaboration CSA. This difference might prove helpful in the classification of this type of disorder in cases where the Ph1 chromosome abnormality is not present.