Outcomes of UCB transplantation are comparable in FLT3+ AML: results of CIBMTR, EUROCORD and EBMT collaborative analysis.

Allogeneic hematopoietic cell transplantation (HCT) from siblings or unrelated donors (URD) during complete remission (CR) may improve leukemia-free survival (LFS) in FMS-like tyrosine kinase 3+ (FLT3+) acute myeloid leukemia (AML), which has poor prognosis because of high relapse rates. Umbilical cord blood (UCB) HCT outcomes are largely unknown in this population. We found that compared with sibling HCT, relapse risks were similar after UCB (n=126) (hazard ratio (HR) 0.86, P=0.54) and URD (n=91) (HR 0.81, P=0.43). UCB HCT was associated with statistically higher non-relapse mortality compared with sibling HCT (HR 2.32, P=0.02), but not vs URD (HR 1.72, P=0.07). All three cohorts had statistically nonsignificant 3-year LFS: 39% (95% confidence interval (CI): 30-47) after UCB, 43% (95% CI: 30-54) after sibling and 50% (95% CI: 40-60) after URD. Chronic graft-versus-host disease rates were significantly lower after UCB compared with either sibling (HR 0.59, P=0.03) or URD (HR 0.49, P=0.001). Adverse factors for LFS included high leukocyte count at diagnosis and HCT during CR2 (second CR). UCB is a suitable option for adults with FLT3+ AML in the absence of an human leukocyte antigen-matched sibling and its immediate availability may be particularly important for FLT3+ AML where early relapse is common, thus allowing HCT in CR1 (first CR) when outcomes are best.

High incidence of post-transplant cytomegalovirus reactivations in myeloma patients undergoing autologous stem cell transplantation after treatment with bortezomib-based regimens: a survey from the Rome transplant network.

The incidence of cytomegalovirus (CMV) reactivations in patients with multiple myeloma (MM) receiving autologous stem cell transplantation (ASCT) is relatively low. However, the recent increased use of novel agents, such as bortezomib and/or immunomodulators, before transplant, has led to an increasing incidence of Herpesviridae family virus infections. The aim of the study was to establish the incidence of post-engraftment symptomatic CMV reactivations in MM patients receiving ASCT, and to compare this incidence with that of patients treated with novel agents or with conventional chemotherapy before transplant. The study was a survey of 80 consecutive patients who underwent ASCT after treatment with novel agents (Group A). These patients were compared with a cohort of 89 patients treated with VAD regimen (vincristine, doxorubicin, and dexamethasone) before ASCT (Group B). Overall, 7 patients (4.1%) received an antiviral treatment for a symptomatic CMV reactivation and 1 died. The incidence of CMV reactivations was significantly higher in Group A than in Group B (7.5% vs. 1.1%; P = 0.048). When compared with Group B, the CMV reactivations observed in Group A were significantly more frequent in patients who received bortezomib, whether or not associated with immunomodulators (9.4% vs. 1.1%; P = 0.019), but not in those treated with immunomodulators only (3.7% vs. 1.1%; P = 0.396). These results suggest that MM patients treated with bortezomib-based regimens are at higher risk of developing a symptomatic CMV reactivation after ASCT.

Use of Impella Recover 2.5 left ventricular assist device in patients with cardiogenic shock or undergoing high-risk percutaneous coronary intervention procedures: experience of a high-volume center.

AIM: Percutaneous coronary interventions are increasingly applied to high-risk patients. The availability of hemodynamic support devices offers a promising option to prevent and treat low-output syndrome in these patients. The aim of this study was to evaluate the feasibility, safety and efficacy of the Impella Recover'' LP 2.5 left ventricular assist device in patients with cardiogenic shock or undergoing high-risk percutaneous coronary interventions. METHODS: Eleven patients presenting cardiogenic shock (N=6) or scheduled for high-risk percutaneous revascularization (N=5) were evaluated. The Impella pump was successfully implanted in all patients, except one. When implanted, the device was correctly positioned in the left ventricle and remained in a stable position. RESULTS: Bleedings occurred in 7 patients (5 of them presented cardiogenic shock), while renal failure and severe thrombocytopenia were observed in 4 and 1 patients respectively, all with cardiogenic shock. During high-risk procedures, the Impella pump succeeded in obtaining hemodynamic stability, while in only two patients with cardiogenic shock the device determined a significant improvement of hemodynamic variables. All elective patients and two patients with cardiogenic shock were discharged from the hospital and were still alive at 30-day follow-up. CONCLUSION: These data, although preliminary due to the limited sample size, demonstrated the feasibility, safety and efficacy of the Impella Recover LP 2.5 during high-risk percutaneous procedures, even though the benefits of prophylactic deployment of such a system have to be further investigated. The use of Impella Recover LP 2.5 in patients with cardiogenic shock is feasible and safe, however it maybe insufficient in reversing an advanced cardiogenic shock which, probably, has to be treated with more powerful left ventricular assist devices.

An open-label study of controlled-release melatonin in treatment of sleep disorders in children with autism.

Long-term effectiveness of controlled-release melatonin in 25 children, aged 2.6-9.6 years with autism without other coexistent pathologies was evaluated openly. Sleep patterns were studied using Children's Sleep Habits Questionnaire (CSHQ) and sleep diaries at baseline, after 1-3-6 months melatonin treatment and 1 month after discontinuation. Sleep diary and CSHQ showed a more problematic sleep in autistic children compared with controls. During treatment sleep patterns of all children improved. After discontinuation 16 children returned to pre-treatment score, readministration of melatonin was again effective. Treatment gains were maintained at 12 and 24-month follow-ups. No adverse side effects were reported. In conclusion, controlled-release melatonin may provide an effective and well-tolerated treatment for autistic children with chronic sleep disorders.

Markers of hypercoagulability and inflammation predict mortality in patients with heart failure.

BACKGROUND AND AIMS: Plasma levels of inflammatory markers are increased in chronic heart failure (HF) and are also subclinical indicators of future HF. Inflammation is strictly correlated with clotting activation, but the association between inflammation, hypercoagulability and prognosis in HF has not been previously reported. METHODS AND RESULTS: Markers of inflammation (interleukin-6; IL-6, and C-reactive protein; CRP) and hypercoagulability (D-dimer; DD, and thrombin-antithrombin III complex; TAT) were prospectively assessed in 214 subjects with New York Heart Association (NYHA) functional class II-IV HF. During a median follow-up of 8.5 months, 32 patients had an event: 13 died and 19 were hospitalized because of worsening of HF. IL-6, DD and TAT levels were all significantly associated with increased risk of death after adjustment for other known HF prognostic factors (age, gender, traditional cardiovascular risk factors, NYHA class, systolic left ventricular function, renal failure, hemoglobin, serum sodium) in a Cox multivariate proportional hazard model (P = 0.003, P = 0.01 and P = 0.02, respectively). When these markers were added simultaneously to the known prognostic factors in a new Cox multivariate model, only DD levels were significant predictors of mortality (hazard ratio [95% confidence interval; CI]: 11 [2.7-45.1], P = 0.001). The Kaplan-Meier curve revealed a significantly better outcome in patients with DD below 450 ng mL(-1). NT-pro-BNP was the only significant predictor of rehospitalization (HR [95% CI]: 5.3 [2.0-13.8], P < 0.001). CONCLUSION: Hypercoagulability and inflammation, as assessed by DD, TAT and IL-6 levels, are associated with an increased mortality risk in HF.

Evaluation of ovarian functionality after a dietary treatment in obese women with polycystic ovary syndrome.

In women with polycystic ovary syndrome (PCOS) weight loss is associated with an improvement in insulin sensitivity and a reduction of the insulin concentration in the plasma. It is clear, then, that the first therapeutic approach that can be considered in obese PCOS patients for restoration of the menstrual cycle should be a diet. The aim of our study was to examine the effect of long-term caloric restriction on the clinical and biochemical abnormalities in obese PCOS women. The results obtained make it clear that caloric restriction for 4 weeks causes an increase in SHBG and decreases of free testosterone and insulin, with consequent improvement of the clinical picture.

Sleep problems and daytime behavior in childhood idiopathic epilepsy.

PURPOSE: To evaluate the presence of sleep problems and their association with behavioral and adjustment problems in children with idiopathic epilepsy. METHODS: A parental questionnaire was used to assess sleep problems in 89 children with idiopathic epilepsy for comparisons with 49 siblings and 321 healthy control children, equally distributed for age and sex. Sleep problems were clustered into five factors: parasomnias, parent/child interaction during the night, sleep fragmentation, daytime drowsiness, and bedtime difficulties. Daytime behavior and psychological adjustment were assessed by means of the Child Behavior Checklist. Maternal distress and disturbance was evaluated by the Malaise Inventory. The better to identify factors associated with sleep problems in the children with epilepsy, multiple regression analysis was performed. RESULTS: Children with epilepsy showed significantly more sleep problems than did both siblings and healthy controls. Within the epileptic group, children with current seizures complained more of sleep problems than did the seizure-free children. Moreover, children with epilepsy showed more behavioral problems and maladjustment. Age, paroxysmal activity density, duration of illness, seizure frequency, and behavioral problems were significantly associated with sleep problems in the epileptic group. CONCLUSIONS: The results of this study in a highly selected sample pointed out the presence of sleep problems, and adjustment and behavioral problems in children with idiopathic epilepsy. The presence of epilepsy, although benign, in childhood is associated with adaptive problems of the child. From this point of view, the alteration of some sleep habits may be a sign of emotional maladjustment. Although parents failed to perceive them as a problem, our findings indicate that attention to sleep and behavioral problems is important in clinical management of children with idiopathic epilepsy.

Usefulness of a 12-month treatment with finasteride in idiophathic and polycystic ovary syndrome-associated hirsutism.

OBJECTIVE: Hirsutism is considered as a skin disease due to increased 5 alpha-reductase activity in the pilosebaceous unit and finasteride is a drug that inhibits this enzymatic activity. This study showed the effectiveness of a chronic treatment with a selective 5 alpha-reductase inhibitor, finasteride, in idiopathic and PCOS-associated hirsutism. METHODS: Finasteride was administered orally at a daily dose of 5 mg for a period of 12 months to 20 women with IH and 20 women with PCOS. MAIN OUTCOME MEASURES: Each group was submitted to clinical (with Ferriman-Gallwey method) and serum hormonal (FSH, LH, 17 beta-estradiol, total and free T, delta 4-androstenedione, DHEAS; dihydrotestosterone, 3 alpha-androstanediol glucuronide) studies at baseline and after 3, 6 and 12 months of treatment. RESULTS: After 3 months of finasteride treatment, a significant decrease in the average hirsutism scores was recorded both in IH (p < 0.0001) and PCOS patients (p < 0.0001). A progressive significant decrease of hirsutism score was observed in IH patients after 6 and 12 months (p < 0.002) and in PCOS patients after 6 but not 12 months. In fact, the maximal therapeutic effect on the hirsutism was obtained after 12 months in the IH and 6 months in PCOS group.

Prevalence of sleep disorders in childhood and adolescence with headache: a case-control study.

Although a relationship between headache and sleep disturbances has been reported in adults, only few data have been available in children. Accordingly, we performed a survey to determine the prevalence of sleep disturbances in children with migraine and tension-type headache. A questionnaire of history and clinical data and of sleep disturbances was given to parents of 283 headache subjects (164 with migraine and 119 with tension-type headache). Results were compared to a normative group comparable for age and sex of 893 normal healthy subjects. Migraine subjects showed a higher prevalence of sleep disturbances during infancy as well as 3-month colic. In both headache groups, more parents had sleep disturbances and there was a higher occurrence of co-sleeping and napping. A high frequency of sleep disturbances involving sleep quality, night awakenings, nocturnal symptoms and daytime sleepiness was reported in headache children. No statistical differences were found in the prevalence of sleep disturbances between migraine and tension-type headache. However, the migraine group tended to have "disturbed sleep" more often with increased prevalence of nocturnal symptoms such as sleep breathing disorders and parasomnias. Our results give further support to an association between sleep and migraine that may have a common intrinsic origin.

The Sleep Disturbance Scale for Children (SDSC). Construction and validation of an instrument to evaluate sleep disturbances in childhood and adolescence.

To attempt a categorization of sleep disorders in children, we developed a 27 item Likert-type rating scale (Sleep Disturbance Scale for Children: SDSC) and assessed the psychometric properties was developed. The scale was distributed to the mothers of 1304 children (1157 controls, mean age 9.8 y; 147 sleep disorder subjects, mean age 9.2y, composed of four clinical groups: Insomnia 39 subjects, Hypersomnia 12 subjects, Respiratory disturbances during sleep 25 subjects and Parasomnias 71 subjects). The internal consistency was high in controls (0.79) and remained at a satisfactory level in sleep disorder subjects (0.71); the test/retest reliability was adequate for the total (r = 0.71) and single item scores. The factor analysis (variance explained 44.21%) yielded six factors which represented the most common areas of sleep disorders in childhood and adolescence. Enuresis was the only item with a factor loading lower than 0.40 and with a low inter-item correlation and was therefore eliminated, resulting in a final scale of 26 items. The re-evaluation of the sample, using the factor scores, supported the validity and the discriminating capacity of the scales between controls and the four clinical groups. The correlation between factor scores corroborated the hypothesis that childhood sleep disturbances are not independent entities nor do they cluster into different groupings related to each other. The SDSC appears to be a useful tool in evaluating the sleep disturbances of school-age children in clinical and non-clinical populations.

Sleep characteristics in healthy children from birth to 6 years of age in the urban area of Rome.

The current survey is an attempt to evaluate age-specific sleep characteristics and to identify the presence of sleep problems in Italian normally developing preschool-aged children. A cross-sectional survey by parental interview on sleep behavior was carried out on 2,889 children (from birth to 6 years). Groups were formed based on age level. Results showed a developmental trend of some sleep characteristics, regarding mainly the length of sleep and rating of night wakings. Comparison with other studies showed that the children in this study had a later sleep onset time and slept less than children of the same age living in some other countries. These dissimilarities may be due to sociocultural and climate differences. Sleep problems (sleep latency longer than 30 minutes or disruptive night wakings) were found in 35% of children less than 2 years old, in 23% of 2-3-year-olds and in 14% of 4-6-year-olds. Children with sleep problems slept significantly less (on average 30-40 minutes across all age levels, required parental presence at time of sleep onset and shared their parents' bed more frequently than those without sleeping problems.

Sleep disorders in tuberous sclerosis: a polysomnographic study.

Overnight polysomnography was performed in 10 subjects with tuberous sclerosis (TS) and partial epilepsy in order to investigate the relationships between sleep organization, sleep disorders and epilepsy. Sleep architecture abnormalities were observed in 9 cases. Compared with ten healthy age-matched controls, the TS group showed a shorter total sleep time, a reduced sleep efficiency, a higher number of awakenings and stage transitions, an increased wake after sleep onset and stage 1 and a decreased REM sleep. Children with seizures showed a more disrupted sleep architecture compared with seizure-free children. Sleep disorders in TS were mainly due to sleep-related epileptic events and were more evident in children who showed large bifrontal or temporal tubers on MRI.

Short temperament questionnaire for children aged 8-12 years in the city of Rome.

The assessment of temperament is usually measured by means of parental questionnaires. Since temperament questionnaires in children aged 8-12 years do not exist in Italy we planned a study to develop an Italian questionnaire. Initially we tried to adapt Hegvik et al.'s questionnaire and delivered 389 questionnaires to the mothers of children aged 8-12 years, but most of them were given back uncompleted, essentially because they often described behavior not usually observed in Italian children. Then we prepared a new, short (30 items) questionnaire which we distributed to 431 mothers of children aged 8-12 years. This new questionnaire was completed by 98.76% of mothers and a high three week rating-re-rating reliability for the different temperamental characteristics under assessment was proved. We believe that this new questionnaire is reliable for temperament assessment in Italian children aged 8 to 12 years, living in a big city environment in Central Italy.

[Continuous localized EEG discharge during sleep in children without neuropsychological problems]. / Décharges EEG localisées continues pendant le sommeil chez des enfants sans troubles neuropsychiques.

After having reported continuous localized EEG discharge during slow sleep (CLEDS) in six children with congenital encephalopathy, we observed a similar EEG picture in six children free from both neuropsychological and neuroradiological defects. They suffered from partial idiopathic epilepsy; five presented a familial disposition towards febrile seizures. Continuous paroxysmal activity during sleep was observed from 4.8 yrs to 4.11 yrs (mean age: 4.9 yrs). Spontaneous remission of CLEDS was observed in three cases after 2-25 months, but one or more relapses occurred in two cases, and five children are still suffering from CLEDS. Seizures were controlled by drugs in all cases. Deterioration of intelligence level, although not severe, was observed in one case, after 24 months of CLEDS.