Turoctocog alfa pegol (N8-GP) is a novel glycoPEGylated extended half-life recombinant factor VIII (FVIII) product developed for prophylaxis and treatment of bleeds in patients with haemophilia A, to enable higher activity levels with less frequent injections compared with standard FVIII products. This phase III (NCT01480180), multinational, open-label, non-randomised trial evaluated the safety and clinical efficacy of N8-GP when administered for treatment of bleeds and for prophylaxis, in previously treated patients aged ≥12 years with severe haemophilia A. Patients were allocated to receive N8-GP for prophylaxis or on-demand treatment for up to 1.8 years. Patients on prophylaxis were administered one dose of 50 IU/kg of N8-GP every fourth day. Bleeds were treated with doses of 20-75 IU/kg. Total exposure to N8-GP in the trial was 14,114 exposure days (159 patient-years). For the prophylaxis arm (n=175), the median annualised bleeding rate (ABR) was 1.33 (interquartile range, 0.00-4.61), the mean ABR was 3.70 (95 % confidence interval 2.94-4.66) and 70 (40 %) patients had no bleeds during the trial. Across treatment arms, 83.6 % of bleeds resolved with one injection and 95.5 % with up to two injections. N8-GP had a favourable safety profile and was well tolerated. The frequency and types of adverse events reported were as expected in this population. One patient developed inhibitory antibodies against FVIII (≥0.6 Bethesda units [BU]) after 93 N8-GP exposure days. No clinically significant safety concerns were identified and N8-GP was effective for prophylaxis and treatment of bleeds in previously treated patients.
Transient myeloproliferative disorder (TMD) in newborns with Down syndrome (DS) has been well described. We report 4 newborns, 2 with DS and 2 without DS, who developed TMD. One newborn with DS developed multiorgan failure and died despite treatment with low-dose cytarabine. In 3 newborns, the TMD resolved spontaneously. Two of these patients, 1 with and 1 without DS developed leukemia on subsequent follow-up and were treated successfully. We reviewed the clinical and laboratory data on 14 non-DS infants with TMD reported in the literature. According to limited data, these patients are more likely to develop leukemia than DS patients, however their outcome is better.
The combination of 2-chlorodeoxyadenosine (2-CDA) and cytosine arabinoside (Ara-C) has been shown to be effective in children with refractory Langerhans cell histiocytosis (LCH). We have treated 5 patients with recurrent LCH with 2-CDA/Ara-C chemotherapy and closely followed immune and hematopoietic function. These patients display a decline in the absolute CD4, CD8, and natural killer cell number, decrease in the CD4/CD8 ratio. Septic events, including pneumocystis infection were present after most of the treatment courses (15/21). These data suggest that 2-CDA /Ara-C, should be considered in resistant and relapsed pediatric patients with LCH with high-risk multiorgan involvement. Consequent profound prolonged combined immune deficiency and myelosupression should be anticipated.
Intracranial involvement of the Hodgkin disease (HD) is a rare entity. Until now, 9 cases of initial presentation of the HD as a brain tumor with appropriate morphologic and histochemical confirmation were reported. Of the 9 patients, 6 had isolated primary intracranial HD and 3 patients after further investigation were found to have extracranial involvement. Seven patients had nodular sclerosing histology, 1 had mixed cellularity, and in 1 case histology was not reported. We describe a patient with systemic nodular sclerosing HD, who initially presented with a brain mass mimicking meningioma and was found to have disseminated lymphadenopathy and bone involvement.
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