Subclinical neuropathy in children with inherited haemostasis disorders.

Motor and sensory conduction of the right peroneal and sural nerves was studied in 28 children (17 HIV seropositive) with inherited hemostasis disorders, without any symptoms of neuropathy. The amplitude ratio of the evoked muscle potential (EMP) at distal stimulation to that at proximal stimulation at the right peroneal nerve was also studied. Thirty healthy aged-matched children were used as controls. There was no statistically significant difference in the distal latency, amplitude and conduction velocity of motor and sensory nerves between patients and controls. On the contrary, a great diminution of amplitude of the EMP during proximal stimulation of nerve was observed in patients, statistically very significant, as compared to controls. This difference was independent of patients' age, severity of hemostasis defect or HIV status. In 9 patients the amplitude was within normal limits. Intraneural oozing due to trivial trauma is postulated as a possible mechanism of peroneal nerve lesion.

Seventeen years of experience with chronic idiopathic thrombocytopenic purpura in childhood. Is therapy always better?

Between 1975 and 1992 450 children with idiopathic thrombocytopenic purpura (ITP) were diagnosed, and of those 100 (22%) developed the chronic form of the disease. Approximately half the patients with chronic ITP presented with mild to moderate hemorrhagic manifestations at the onset of purpura (30 cases) and/or later during the course of the disease (25 cases). The incidence of intracranial hemorrhage was 1%, and the mortality rate due to overwhelming septicemia after splenectomy was also 1%. Overall one-third of the patients received no therapy; two-thirds of them went into spontaneous remission within 8 months to 8 years from the onset of ITP. Steroids given in conventional or high doses (51 cases) achieved a transient (if any) rise in platelet count, but in no case were steroids curative. Remission related to intravenous immune globulin (IVIG) therapy was noticed in 38.5% of the children (10 of 26) after variable courses. The response rate to splenectomy was 95.0%. Ultimately the long-term outcome in children with chronic ITP was as follows: remission, 58 cases (spontaneous, 30; after IVIG therapy, 10; after splenectomy, 18); hemostatic platelet values, 22 cases (spontaneous, 16; after IVIG, 5; after splenectomy, 1). Thirteen children were lost in follow-up, and 7 remain thrombocytopenic but asymptomatic. These data indicate that chronic ITP in childhood runs a benign course in most cases and may remit with or without therapy even several years from onset. Therefore, therapeutic intervention has to be individualized, and splenectomy, which is not always safe, should be reserved for problematic cases that fail to respond to conventional therapeutic modalities.

Diffuse splenic and visceral hemangiomas complicated by chronic consumption coagulopathy.

The case of a 7-year-old girl with a 2 year history of easy bruising associated with thrombocytopenia is reported. On admission she presented with ecchymoses, abdominal distention and splenomegaly. Hemostasis investigation revealed a consumption coagulopathy. Several radiological studies failed to confirm the diagnosis of diffuse splenic and visceral hemangiomatosis, which was eventually established by an explorative laparotomy. Platelet count and the other coagulation abnormalities progressively returned to normal after splenectomy, although the remaining hemangiomas were extensive.

Prevalence of inhibitor formation in a cohort of haemophilic children exposed to several products of various purities.

To evaluate the frequency and potency of inhibitor formation based on the product used, we retrospectively reviewed the records of 99 children with various types and severity of haemophilia (haemophilia A 82, severe 46; haemophilia B 10, severe 6; vWD 7) treated for the last 20 years. After a mean observation period of 8 years an overall of 23 patients (23.2'/0) developed an inhibitor (haernophilia A 26.8%; severe 4O%, moderate 20%, mild 3.8%). None of the haemophilia B patients presented with an inhibitor, and only one child with bevere vWD (1/7, 14.3%) showed a transient inhibitor under cryoprecipitate therapy. Inhibitor titre was low (< 5 BU) in most cases (91.3%) and in only two patients (8.75%) was 6 and 8 BU respectively. Antibodies to FVIII were transient (detected only once) in four (17.4%) and intermittent in 19 patients (82.6%). By the age of 12 years, 17/23 patients (73.9%) had demonstrated an inhibitor. The inhibitor detection seemed to be higher in the groups of patients exposed to monoclonal (3115, 20%), SID-treated (10159, 16.9%) or H/T FVIII concentrates (6/41, 14.6%), compared to groups of patients who received cryo/plasma (9.5%) or unmodified concentrates (5.1%); nevertheless the differences were not statistically significant. Surprisingly, none of the 52 patients who received a S/D + chromatography-treated factor VIIl concentrate developed an inhibitor after a mean observation period of 1.7 years (range 0.2-2 years). The overall prevalence of inhibitor formation in previously untreated haemophiliacs was 14.3% (4/28), irrespective of the product used. Our data indicate that a high proportion of our haemophilic children exposed to several products of various purities have developed a low-titre inhibitor which in most cases was transient or intermittent. However, despite the presence of the antibody, none of the patients needed a change in the mode of treatment.