Successful intraosseous factor VIII application in a haemophilic emergency.

WHAT IS KNOWN AND OBJECTIVES: Intravenous (IV) replacement therapy with plasma derived or recombinant factor VIII (FVIII) and factor IX concentrates is the mainstay for treatment of patients with haemophilia A and B. Therefore, the current therapy is particularly dependent on the presence of a secure IV access especially in case of emergency. CASE DESCRIPTION: A life-threatening bleeding event in an 8-month-old boy is managed by intraosseous (IO) infusion of recombinant FVIII concentrate. No adverse events have been observed 6 months after the application, and complete heeling has been reported. WHAT IS NEW AND CONCLUSION: Venous application of factor concentrate remains inevitable in any haemophilic emergency. In case IV access is lacking, an IO institution of factor might be considered. To our knowledge, this represents the first reported case of IO application of recombinant FVIII concentrate in a patient with haemophilia.

Treatment of Childhood Acute Myeloid Leukemia in Bulgaria.

BACKGROUND: During the last four decades the prognosis of childhood acute myeloid leukemia (AML) has been substantially improved due to an increase in complete remission (CR) rates, event-free survival (EFS) and reduced early mortality. The relapsed AML still remains a therapeutic challenge. AIM: To report the AML treatment results of the Bulgarian pediatric oncohematological centers. MATERIALS AND METHODS: Retrospective analysis of the treatment results of children and adolescents (age from 0 to 20 years) with primary AML. Unified AML BFM- backbone type treatment protocol is used. RESULTS: This study included 97 newly diagnosed patients (44 girls and 53 boys) with AML in Bulgaria between 2003 and 2016. The median age at diagnosis was 10.2 years. The most frequent FAB-morphologic subtype was M2 followed by M4. First complete remission (CR1) was achieved in 83 patients (85.6%). The 13-year EFS was 49%, while the overall survival (OS) was 54.6%. Twenty seven (27.8%) patients relapsed, with only 5 of them being still alive towards the end of the study period. CONCLUSION: The EFS and OS for the children with AML in Bulgaria are comparable with those reported by other European groups. The prognosis of relapsed AML remains still unfavorable for the past 13 years.

Cerebellar Degeneration as a Rare Paraneoplastic Syndrome in a Child With Hodgkin Lymphoma.

We report a rare case of cerebellar degeneration as a paraneoplastic syndrome in an 8-year-old boy with Hodgkin lymphoma that presented during first-line treatment. Antibodies against Purkinje cells (anti-Tr antibodies) were detected in the serum of the patient. After successful treatment of the lymphoma, the cerebellar symptoms resolved partially. Childhood presentation of paraneoplastic cerebellar degeneration is extremely rare, with only a few reports in the literature. For this reason, the description of all such cases contributes to the enrichment of the medical knowledge and will improve the diagnosis and the treatment of this complication.

Cardiogenic shock associated with reversible dilated cardiomyopathy during therapy with regular doses of venlafaxine.

We report a cardiac complication in a patient treated with regular doses of venlafaxine. A 49-year-old man with prior normal cardiac function and stable chronic hepatitis C was treated for a major depressive disorder with usual doses of venlafaxine during an 8-month period until the occurrence of a cardiogenic shock in a context of dilated cardiomyopathy. Three months after withdrawal of the drug, the left ventricular ejection fraction returned to normal values. Cardiomyopathy is a rare complication with high doses of venlafaxine that was not previously reported in patients free of prior cardiac disease and cardiomyopathy and treated with usual doses (initially 150 mg daily; after 3 months, 75 mg daily). An objective assessment revealed that venlafaxine was probably implied in the subsequent development of cardiomyopathy when considering the Naranjo Probability Scale. Physicians who usually prescribe venlafaxine have to be briefed on such potential cardiac adverse effects even with usual doses.

Diagnosis and treatment of pleuropulmonary blastoma-single center experience.

Pleuropulmonary blastoma (PPB) is a rare and potentially aggressive intrathoracic disembryonic neoplasm typically occurring in children less than 6 years of age. We assessed the relative incidence, clinical characteristics, treatment outcome, and the prognostic factors for long-term survival in patients with PPB treated at our institution over a 25-year period, and compared these data with reports in the literature. From 1985 to 2010, 11 children (4 males and 7 females), with a median age of 5.4 years (range, 1-12 years) were treated at our hospital. Here we described the main characteristics of these patients, the diagnostic methods, and treatment modalities used. During a median follow-up period of 80, 9 months, the overall survival (OS) and disease-free survival (DFS) rates were 54, 6% and 45, 5%, respectively. Two patients survived for more than 20 years. The main prognostic factors for long-term survival were the diseases type I and II and treatment with radical surgery. Our results show that in order to improve the prognosis of patients with PPB a timely in our opinion and accurate diagnosis needs to be established and treatment should be offered according to the disease type and extend of dissemination.

Successful mobilization of peripheral blood stem cells in children with cancer using plerixafor (Mozobil) and granulocyte-colony stimulating factor.

This paper describes the successful mobilization of peripheral blood stem cells for autologous transplantation in three children with malignant diseases by using plerixafor (Mozobil; Genzyme Corporation, Cambridge, MA) and granulocyte-colony stimulating factor (G-CSF) after failed previous mobilizations. A median sixfold increase in the number of circulating CD34+ cells after plerixafor treatment as compared with the baseline level was observed. An optimal CD34+ cell count for transplantation with one or two leukapheresis sessions was achieved. Mobilization using plerixafor was found to be safe with no adverse events. Therefore, the combination of G-CSF and plerixafor in children results in effective increases in peripheral CD34+ cell counts and reduces the risk of mobilization failure.