Desmopressin (DDAVP) in the management of patients with congenital bleeding disorders

Bleeding disorders, including haemophilia, von Willebrand disease, and platelet function abnormalities pose a substantial, ongoing management challenge. Patients with these disorders not only require treatment during bleeding events but also need effective management strategies to prepare for events ranging from minor dental procedures to major surgery and childbirth. Moreover, women with bleeding disorders often require ongoing treatment to prevent menorrhagia during childbearing years. Desmopressin (DDAVP), a synthetic derivative of the antidiuretic hormone l ‐arginine vasopressin, has become a well‐established tool for the management of patients with bleeding disorders in a variety of clinical settings. However, despite the widespread use of DDAVP, the available clinical evidence on its efficacy and safety in these settings is limited, and there has not been a recent comprehensive review of its role in the clinical management of patients with bleeding disorders. As such, this article provides a review of the mechanism of action and pharmacokinetic properties of DDAVP, followed by a concise summary of the available evidence for its use in the treatment and prevention of bleeding.     

Legionnaires' disease in non-Legionnaires. A report of five cases

Seventeen patients with illness resembling Legionnaires' disease were evaluated for antibodies to the Legionnaires' bacillus. Three patients were Legionnaires who developed pneumonia after attending the July 1976 convention. All three were seropositive (titers greater than or equal to 1:128) to the Legionnaires' bacillus. None of the remaining 14 patients were Legionnaires. Five were seropositive and nine, seronegative (titers less than or equal to 1:32). The seropositive patients had illnesses best explained by Legionnaires' disease, whereas the seronegative patients had illnesses better explained by other diagnoses. This correlation between serology and clinical findings supports the specificity of the antibody test for Legionnaires' disease. Of the five non-Legionnaire seropositive cases, two occurred before, and one 3 months after, the convention. The case histories of these five patients are presented. Disease generally involved the lungs, kidneys, and the central nervous system. Although the pathogenesis of this multiple organ involvement is unclear, a possible explanation is the production of a toxin by the Legionnaires' bacillus.     

Treatment of cryptosporidiosis with paromomycin. A report of five cases.

Cryptosporidiosis continues to be one of the most devastating complications of the acquired immunodeficiency syndrome, causing severe, chronic diarrhea that is largely refractory to treatment. More than 60 drugs have been tried in the treatment of cryptosporidiosis, none of which have been consistently successful. We describe the successful treatment of cryptosporidiosis in five patients with acquired immunodeficiency syndrome with oral paromomycin at a dose of 1500 to 2000 mg/d. All five patients had resolution of symptoms and normalization of bowel movements, although one patient later relapsed while receiving paromomycin. Three of five patients cleared Cryptosporidium from the stool. Paromomycin is a promising therapy for cryptosporidiosis in acquired immunodeficiency syndrome and further prospective clinical trials are warranted.     

Malignant lymphoma in systemic rheumatic diseases. A report of five cases

We describe five patients suffering from systemic rheumatic diseases who developed a malignant lymphoma. Two patients, one with systemic lupus erythematosus and another with systemic necrotizing vasculitis, suffered from Hodgkin's disease and three other patients, one with lupus and two with Sj?gren's syndrome, developed non-Hodgkin's lymphoma. Only one of these patients had been treated with immunosuppressive drugs before the onset of the lymphoproliferative malignancy. Four patients were given combination chemotherapy and complete remission was achieved in the three patients for whom follow-up data were available. We discuss the relationships between systemic rheumatic diseases and lymphoma.     

Visceral leishmaniasis (kala-azar) in solid organ transplantation: report of five cases and review.

Visceral leishmaniasis is an infectious disease that occurs only rarely in recipients of solid organ grafts but is associated with an elevated mortality rate despite proper treatment. We report five cases diagnosed in our hospital. All the patients were men aged 30 to 60 years who had undergone kidney transplantation (3 patients), heart transplantation (1), or liver transplantation (1). Three of the patients died, one had multiple recurrences, and one developed post-kala-azar cutaneous leishmaniasis. We review the clinical features, treatments, and outcomes of 26 previously reported cases, pointing out the lower cure rate associated with human immunodeficiency virus infection.     

About five cases of acute rheumatic fever in the adult

Summary We describe five cases of acute rheumatic fever in adults (applying Jones's criteria and exclusion of other common causes of acute polyarthritis in adults). The polyarthritis was migratory in three patients and additive in two. Severe tenosynovitis was present in three patients. Only one patient had carditis and received corticoids. The others improved with aspirin. The disease subsided after a time period varying from one week to two months and did not relapse.     

Arterial manifestations of Behcet's disease. A report of five operated cases

Vascular involvement in Behcet's disease is common, especially in deep vein thrombosis. Aneurysmal or occlusive arterial disease is, however, rare. The authors report five cases of vascular Behcet's disease reviewed over a period of 4 years (1996-1999). All these patients were men and the mean age was 38 years. The arterial disease was the presenting symptom of Behcet's disease in 3 cases. The other patients had been followed up for Behcet's disease for 4 years. The average time of onset of arterial disease was 7.2 years with respect to the presenting symptom, the range being 2 to 20 years. In all, seven arteries were involved, two patients having two arterial sites at the same time. The femoral artery was involved in 3 cases. The iliac artery was involved twice. One patient had a coronary thrombosis and the last case was of a pseudoaneurysm of the infra-renal abdominal aorta which ruptured into the retroperitoneal space. Six lesions were aneurysmal. All patients underwent surgery. The histological analysis of the resected lesions showed non-specific panvasculitis. The postoperative period was marked by short and medium term complications: four prosthetic thromboses and two anastomotic pseudo-aneurysms which required several surgical procedures and which led to two deaths. These results and a review of the literature underline the need to search for Behcet's disease in all cases of aneurysmal or occlusive arterial disease in young patients, especially those born in the Mediterranean regions. Surgical treatment should not be delayed. In these inflammatory conditions with pronounced perivascular involvement, the surgery is difficult and postoperative complications are common, especially anastomotic disunion. Medical treatment has no surgical implication, but does not prevent progression of the disease.     

Shigella bacteremia in adults. A report of five cases and review of the literature

Shigella bacteremia is rare, occurring mainly in children. We describe five adult patients with Shigella bacteremia and review data on 22 cases reported in the English-language medical literature. Eighteen (67%) of 27 patients had either an underlying condition or were aged older than 65 years. Most patients had clinical signs of acute febrile gastroenteritis. However, in six patients, the organism was not isolated from stool. Species isolated from blood included Shigella flexneri in 11 patients, Shigella sonnei in eight, and Shigella boydii and Shigella dysenteriae in one patient each. Isolation of the bacterium from blood only was associated with a high mortality rate, in contrast to its isolation both from blood and stool. It is suggested that blood cultures should be obtained from elderly or immunocompromised patients with acute febrile gastroenteritis to detect shigellemia as well as bacteremia caused by other enteric pathogens, such as Salmonella or Campylobacter.     

Desmopressin (DDAVP) in bleeding disorders of childhood

As in adults, desmopressin (DDAVP) can be used in children for prophylaxis of bleeding and to stop bleeding in many hereditary and acquired bleeding disorders. DDAVP is the treatment of choice in children with mild hemophilia and type 1 von Willebrand's disease (vWD). It is effective in some variants of vWD and in many patients with platelet function defects. It reduces the bleeding diathesis of children with uremia and drug-induced bleeding complications. In any case, a test dose of DDAVP has to be given to the patient to predict the hemostatic effect before relying on this drug for treatment. The response can be measured by shortening of the bleeding time (BT) and of partial thromboplastin time (PTT), indicating a rise of Factor (F) VIII or von Willebrand factor (vWF). Side effects such as facial flushing, transient headache, increased pulse rate, and drop in systolic blood pressure are mild and transient. They can be minimized when the dose is not exceeding 0.3 microg/kg body weight, and the infusion lasts at least 20 to 30 minutes. The strong antidiuretic action of DDAVP has some potential problems that are negligible in adults and older children when water intake is restricted. In infants and small children under the age of 18 months, however, DDAVP should be used with caution and with close surveillance in order to prevent water intoxication and electrolyte imbalance. The danger is increased when the patients receive parenteral fluid substitution. The advantages of DDAVP include the reduction in the use of plasma factor concentrates, thereby minimizing the danger of immunological or infectious complications, as well as the considerable reduction of costs realized by treatment with this form of medication. Fortunately, it can be applied successfully in the most frequent hereditary bleeding disorder, namely vWD type 1.     

Lymphoproliferative disorders in paediatric rheumatic diseases. A report of two cases

Lymphoproliferative disorders (LPD) are reported with a much lower frequency in children with rheumatic diseases than in their adult counterparts. We describe 2 patients who developed a lymphoma during the course of the disease. The first is a 16-year-old girl diagnosed with systemic juvenile idiopathic arthritis 6 years before who developed a mucosa-associated lymphoid tissue (MALT) lymphoma. The second report involves a boy diagnosed with systemic lupus erythematosus at 9 years of age who developed a Hodgkin's lymphoma 9 years after the disease onset. In spite of the low frequency of LPD in children with rheumatic diseases, these processes do occur.     

Interventional EUS cholangiography: A report of five cases

BACKGROUND: ERCP may be unsuccessful because of the presence of a complex peripapillary diverticulum, prior surgery, obstructing tumor, papillary stenosis, or impacted stones. Percutaneous transhepatic cholangiography is a classic technique for accessing the bile duct and remains the primary alternative when biliary ERCP is unsuccessful. With the evolution of interventional EUS, additional options are available for management of biliary obstruction. METHODS: EUS cholangiography was performed, after which the puncture was enlarged to form an enterocholedochal fistula that was used for interventions that resulted in biliary decompression in 5 patients with obstructive jaundice. OBSERVATIONS: Cholangiography was readily performed in all 5 patients. In one patient, a guidewire could not be manipulated across the papilla via the enterocholedochal fistula, necessitating percutaneous intervention. Biliary decompression was achieved in the other 4 patients, in the last two, as a single procedure. CONCLUSIONS: Interventional EUS cholangiography is a new technique that allows drainage of a dilated biliary system when the bile duct is inaccessible by conventional ERCP.     

Paroxysmal ventricular tachycardia: A report of five cases

Paroxysmal ventricular tachycardia is more common than the number of reported cases would indicate. Five cases are described, illustrating different causes for the attacks.In two, the subjects of advanced myocardial disease with steadily progressing congestive failure, digitalis was the provoking cause of the attacks. The dose of digitalis did not exceed that calculated by the body-weight method of Eggleston. It is concluded that this disorder of rhythm depends more upon the state of cardiac nutrition than upon the degree of concentration of the drug in the tissues. It is suggested that a mechanism, similar to that which underlies the production of auricular fibrillation by digitalis in cases of heart disease with normal rhythm, may also be present in these cases where the drug has provoked ventricular tachycardia.In one case, nicotine intoxication was regarded as an important factor in the production of this arhythmia.Advanced coronary sclerosis and myocardial disease were present in one case, and in the last case no demonstrable cause could be found. Quinidine sulphate was of value in controlling the paroxysms in this case.The prognosis is briefly discussed and emphasis is placed upon closely watching the heart rate in all cases of advanced myocardial failure under treatment with digitalis.