Subcutaneous bolus injection of deferoxamine is an alternative method to subcutaneous continuous infusion

The objective of this study was to compare the short- and long-term efficacy of deferoxamine (DFO) given by subcutaneous (SC) continuous infusion over 10 hours via a pump (n = 10) versus a twice-daily subcutaneous bolus injection of the same overall dose (n = 10) in 20 thalassemic children. Urinary iron excretion was measured in 24-hour urine samples after DFO treatment in the 20 patients. The patients were randomized to two groups: 10 patients continued SC continuous infusion with a pump and the remaining 10 received the same overall dose of DFO by twice-daily SC bolus injection for a year. Serum ferritin levels and T1-weighted spin-echo and T2-weighted fast spin-echo signal intensities of liver and paraspinal muscle were determined at initiation and 1 year after initiation of the therapy. In 12 patients, six from each group, liver biopsies were performed and hepatic iron concentration was determined at initiation of therapy and 1 year after treatment. A similar and significant decrease in ferritin levels and improvement in signal intensities of the liver were observed in response to chelation therapy with DFO in both groups (P < 0.01, within each group). Hepatic iron concentration decreased in all patients in the SC bolus injection group (P < 0.05) and in four patients in the SC continuous infusion group (P > 0.05). Hepatic iron concentration was noted to be slightly increased in two patients in the SC continuous infusion group, which may be due to poor compliance. Based on these results, twice-daily SC bolus injection of DFO is as effective as administration via SC continuous infusion using a pump. Subcutaneous bolus injection, being more convenient for the patient, may be a more preferable method of DFO administration.     

Emollients improve treatment results with topical corticosteroids in childhood atopic dermatitis: a randomized comparative study

The aim of the study was to investigate whether adding emollients to the standard topical corticosteroid therapy influences the outcome of children with atopic dermatitis. Fifty‐two children aged between 2 and 12 yr were divided randomly in two subgroups consisting of 26 children each. Both groups applied 0.1% methylprednisolone aceponate cream on lesional atopic skin once daily for 2 wk and were observed for another 4 wk after treatment discontinuation. Group B used additionally emollients for the whole study period. Patients were evaluated at days 0 (baseline), 7, 14 (end of therapy), 28 and 42 (follow‐up). Both groups demonstrated significant improvement of disease severity according to EASI (Eczema Area and Severity Index) scale (group A: 6.8 ± 3.59 before and 0.87 ± 1.25 after therapy, p < 0.001; group B: 9.6 ± 8.39 before and 1.11 ± 2.37 after therapy, p < 0.001). Xerosis improved significantly better in group B compared to group A, both clinically (group A: 1.38 ± 0.57 scores before and 1.5 ± 0.58 scores after therapy, p = 0.11; group B: 1.62 ± 0.64 scores before and 0.12 ± 0.33 scores after therapy, p < 0.001), and by corneometry assessment (group A: 41.7 ± 9.1 units before and 51.3 ± 11.3 units after therapy, p < 0.001; group B: 38.9 ± 12.9 units before and 58.2 ± 13.5 units after therapy, p < 0.001). A trend towards faster resolving of pruritus in group B (group A: 5.44 ± 2.6 scores before and 3.22 ± 2.31 scores after therapy, p = 0.001; group B: 5.87 ± 2.79 scores before and 2.24 ± 1.59 scores after therapy, p < 0.001) was also observed. In group B, the improvement was maintained for couple of weeks after treatment discontinuation, while in group A recurrence of the disease was noted (EASI at day 42 in group A vs. group B: 5.29 ± 5.6 vs. 1.25 ± 1.4, p = 0.01). Similar results were also observed for xerosis (p < 0.001) and pruritus (p = 0.002). Concomitant usage of emollients significantly improves xerosis and pruritus during corticosteroid treatment of atopic dermatitis and enables to maintain clinical improvement after therapy discontinuation.     

Self-reported level of and factors influencing the compliance to desferrioxamine therapy in multitransfused thalassaemias

Aim: To analyse the self‐reported degree of and factors influencing the compliance to desferrioxamine (DFO) therapy in children with transfusion‐dependent thalassaemia major in Malaysia. Methods: A cross‐sectional study was conducted on all children with thalassaemia major on DFO attending Likas Hospital, Sabah, in September 2008. Patients or carer‐providers were interviewed to report on the degree of compliance as either highly (administering >90% of DFO), moderately (51–90%), poorly (1%–50%) or non‐compliant (0%) to DFO in the preceding month. The latest serum ferritin levels were noted. Results: The median (range) age at first blood transfusion of these 139 (73 males, median (range) age at interview: 9.0 years (2–16 years)) patients were 1.0 year (2 months to 10 years). The median (range) duration of regular DFO therapy was 2 years (2 months to 10 years). Forty‐three (31%) of the patients reported themselves to be highly compliant, 70 (50%) moderately and 26 (19%) poorly or non‐compliant. Multivariate analysis showed that a lower family income negatively affected the degree of compliance, while family support positively affected the degree of compliance to DFO. No correlation existed between self‐reported degree of compliance and latest serum ferritin level (6444 ± µmol/L; P = 0.186). Conclusion: The self‐reported compliance to DFO therapy was moderate in this cohort of patients with thalassaemia major in Malaysia. The serum ferritin level was high, possibly because of the relatively short duration of DFO therapy and compliance. Oral iron chelator should be considered to improve the compliance to iron chelation.     

51������β�鵰�������ϰ���ƶѪ���� ������Ѫȥ�������������صĹ�ϵ�о�

目的探讨重型β珠蛋白生成障碍性贫血(beta-thalassaemia,简称β-TM)患儿长期输血、去铁治疗与铁过载的关系。方法深圳市第二人民医院2001年成立"地贫之友"与"地贫服务队",对β-TM患儿进行规范性的长期输血和去铁治疗。每3个月监测血清铁蛋白浓度(SF)、肝肾功能、心肌酶谱、心功能、心脏和肝脾B超、血糖和尿糖。2001年2月至2010年6月对其中51例患儿进行核磁共振检测心脏T2*、左心室射血分数(LVEF)、肝脏T2*、胰腺T2*和垂体T2*。根据治疗方法分为足疗程去铁胺+去铁酮(DFO+DFP)联合去铁治疗组(足疗程联合组)10例、不足疗程DFO+DFP联合去铁治疗组(不足疗程联合组)31例、单用足疗程地拉罗司(DFX)去铁治疗组(单用DFX组)10例。根据SF质量浓度分为SF≤2000μg/L组(A组)12例、SF~3000μg/L组(B组)17例、SF>3000μg/L组(C组)22例。结果各组LVEF、心脏T2*、垂体T2*值差异无统计学意义(P>0.05);足疗程联合组肝脏T2*高于不足疗程联合组(P<0.05),单用DFX组肝脏T2*、胰腺T2*、垂体T2*均高于足疗程联合组和不足疗程联合组(P<0.05)。足疗程联合组SF低于不足疗程联合组,单用DFX组SF低于足疗程联合组和不足疗程联合组,差异均具有统计学意义(P<0.05)。C组肝脏T2*和胰腺T2*明显低于A组和B组,差异具有统计学意义(P<0.05)。心肌铁过载11例(21.6%),肝脏铁过载43例(84.3%)。SF与心脏T2*无相关性(r=0.254,P>0.05),与肝脏T2*呈中度负相关(r=0.558,P<0.01)。结论足疗程DFO+DFP联合去铁治疗和单用足疗程DFX去铁治疗均能有效降低血清铁蛋白浓度,动员肝脏组织铁,效果优于不足疗程DFO+DFP联合去铁治疗。不同去铁方式均能减轻心脏铁过载。     

Intensification of chelating-therapy in patients with thalassemia major

With the introduction of "hypertransfusion" regimens the extent of disease- and therapy-related hemosiderosis has become the survival limiting factor for patients with beta-thalassemia major as iron transferred with transfusions cannot be excreted by physiological means. Subsequent introduction of deferoxamine therapy for iron elimination and prophylaxis of hemosiderosis has improved prognosis and life quality of these patients considerably. We report our experience with seven adolescent patients with beta-thalassemia and ineffective subcutaneous therapy and severe hemosiderosis-related organ complications. For that reason they received i. v. intensified chelate therapy. The patients were given 70 to 120 mg/kg DFO 7 days a week continuously via a Port-a-cath or Hickman central venous line. Under high-dose i. v. DFO therapy, serum ferritin levels significantly decreased in all patients. Target serum ferritin levels of 3 000 ng/ml were reached after 12 to 20 months of treatment. In 3 of the 5 patients that were treated for longer than 43 months serum ferritin levels even dropped below 2 000 ng/ml. Serum ferritin levels also correlated well with SQUID examinations. Therefore, monitoring of serum ferritin may be useful to monitor patient's compliance and control intensified DFO therapy. Continuous administration of the intensified DFO therapy induced normalization of liver function and left ventricular cardiac function in all patients who are still alive. Two patients died due to cardiac decompensation. In five patients 19 episodes of central catheter-related infections were observed (1.5 infections per 1 000 catheter days). No DFO-associated allergic reactions nor irreversible organ dysfunction were observed. Our results indicate that intensified i. v. DFO therapy is an effective and safe method for treatment of severe organ dysfunction in patients with thalassemia major. The most severe problems are catheter-related infections and inconsistent long-term compliance.     

Zinc concentration in amniotic fluid of zinc-deficient rats and its relation to fetal weight

Zinc concentration in amniotic fluid and its relation to fetal weight were investigated in three groups of pregnant rats: group A received a zinc-adequate diet, and the rats of groups B and C were fed a zinc-deficient diet. Group C also received zinc supplementation in water. The daily food consumption, weight and plasma zinc levels on days 1, 10 and 20, zinc concentration in amniotic fluid, the number of implantation sites, the number of resorptions, the number of live fetuses and fetal weight were determined. Plasma zinc concentrations were significantly different at the end of gestation between group B (Zn = 167.6 +/- 26.6 micrograms/dl) and the other two groups (group A = 199 +/- 18.6 micrograms/dl; group C = 204 +/- 13.7 micrograms/dl). The number of resorptions was significantly higher in group B and the number of live fetuses was significantly lower in this same group (p less than 0.025). Fetal weight was significantly lower in group B (p less than 0.001). The zinc concentrations of the amniotic fluid were significantly lower in group B (14 +/- 4.7 micrograms/dl) as compared to group A (83 +/- 11.4 micrograms/dl) and C (82 +/- 21 micrograms/dl; p less than 0.001). There was a positive linear correlation between zinc concentrations in amniotic fluid and fetal weight, being r: 0.7379 (p less than 0.001).     

Comparison of treatments of chronic hepatitis B in children with lamivudine and α-interferon combination and α-interferon alone

BACKGROUND: The aim of this study was to compare the efficacy of the alpha-interferon treatment with treatment using alpha-interferon and lamivudine in combination for cases of childhood chronic hepatitis B infection. METHODS: Patients were evaluated in two groups retrospectively. In group 1, 27 patients were simultaneously given alpha-interferon 2b 10 MU/m2, 3 days a week by s.c. injection plus lamivudine 4 mg/kg a day (maximum 100 mg) for 12 months. In group 2, there were 13 patients who only received the same dosage of alpha-interferon and no lamivudine over the same period of time. RESULTS: In group 1 the initial mean value of alanine aminotransferase (ALT) was 121 +/- 66 IU/L and decreased to 27.8 +/- 11.5 IU/L; in group 2, initial mean values of ALT was 129 +/- 46 IU/L and decreased to 60 +/- 6 IU/L at the end of the twelfth month of the therapy (P < 0.05). Hepatitis B virus DNA (HBV-DNA) clearance was obtained in all group 1 patients and six of 13 patients in group 2 at the end of the therapy (P < 0.001). The rates of hepatitis B early (HBe) antigen clearance and anti-HBe seroconversion were 59 and 37% in group 1 and 46 and 30.7% in group 2 (P > 0.05). The number of patients with complete response was found to be 10 out of 27 (37%) in group 1 and four out of 13 cases (30.7%) in group 2, 6 months after the end of the therapy. There was no statistically significant difference between both groups (P > 0.05). CONCLUSION: alpha-Interferon and lamivudine combination therapy had a more beneficial effect than alpha-interferon monotherapy in normalization of ALT and clearance of HBV-DNA; however, the complete response rate at 6 months after the end of the therapy was not statistically significantly different between both groups.     

Efficacy, tolerance, and pharmacokinetics of once daily tobramycin for pseudomonas exacerbations in cystic fibrosis

OBJECTIVE: To compare once daily with thrice daily tobramycin for treatment of Pseudomonas aeruginosa infection in patients with cystic fibrosis. DESIGN: 22 patients with cystic fibrosis, mean (SD) age 11 (3.4) years (range 5.6-19.3), with pulmonary pseudomonas exacerbations were randomly assigned to receive a 14 day course of tobramycin (15 mg/kg/day) either in three infusions (group A) (n = 10) or a single daily infusion (group B) (n = 12), combined with ceftazidime (200 mg/kg/day as three intravenous injections). Efficacy was assessed by comparison of pulmonary, nutritional, and inflammatory indices on days 1 and 14. Cochlear and renal tolerance were assessed on days 1 and 14. Tobramycin concentration was measured in serum and sputum 1, 2, 3, 4, 8, and 24 hours after the start of the infusion. Analysis was by non-parametric Wilcoxon test. RESULTS: Variables improving (p < 0.05) in both groups A and B were, respectively: weight/height (+4% and +3.1%), plasma prealbumin (+66 and +63 mg/l), forced vital capacity (FVC) (+14% and +11%), forced expiratory volume in one second (+15% and +14%), and forced expiratory flow between 25% and 75% of FVC (+13% and +21%). Improvement was not significantly different between groups. Renal and cochlear indices remained within the normal range. Serum peak concentration of tobramycin on day 1 was 13.2 (7.1) mg/l in group A and 42.5 (11.2) mg/l in group B (p < 0.001); serum trough was 1.1 (0.8) mg/l in group A and 0.3 (0.2) mg/l in group B (p < 0.01). Tobramycin concentrations in sputum were two to three times higher in group B than group A. CONCLUSIONS: Once daily tobramycin combined with three injections of ceftazidime is safe and effective for the treatment of pseudomonas exacerbations in cystic fibrosis patients.     

Studies of variations of subcutaneously infused desferrioxamine and iron movements in thalassaemia children

Desferrioxamine (DFO) plus ferrioxamine (FO) variations and alterations in plasma and urinary iron (Fe) levels were investigated in eight children with thalassaemia major during a 12-h s.c. DFO infusion at a dose of 40 mg/kg body weight. During the infusion, blood samples were regularly taken and total urine was also regularly collected in all patients. In the plasma, a mean DFO plus FO plateau level of 9 mg/l was reached after 4 h and remained steady in the subsequent 8 h. At 30 h the mean DFO plus FO concentration in plasma was still 4.5 mg/l. At 30 h, the urinary excretion ranged from 15% to 70% of the infused dose (mean: 42%). In plasma, the Fe concentration increased on average by 20% (range 10%–30%), remained steady during the DFO infusion, then returned to the basal level after 16–24 h. Urinary Fe excretion started early and still persisted 18 h after the infusion. The amount excreted at 30 h ranged from 5 to 27 mg (mean: 13.6 mg). This study emphasizes the delay in obtaining a DFO plus FO plateau in plasma, the important interindividual variations and the slow decrease of the drug after the end of the infusion. It points out the delay and the values of the urinary Fe excretion compared to the plasmatic and urinary drug variations.Abbreviations Fe iron - FO ferrioxamine - DFO desferrioxamine - s.c. subcutaneous     

Alternate day corticosteroid therapy and growth in renal transplant children

Corticosteroid therapy is probably the main factor inducing stunted growth in pediatric renal transplant recipients. Results of a randomized study in 35 children who received their kidney between 1981 and 1984 are reported. All patients had normal renal function and were taking azathioprine and prednisone to ensure immunosuppression. Eighteen months after transplantation, patients with normal renal biopsy results were randomized to receive further daily corticosteroid therapy (group A) for an additional year or to alternate-day corticosteroid therapy (group B). Chronologic age, bone age, renal function, and previous growth retardation were strictly comparable, in the two groups. During the first year, only prepubertal children in group B exhibited catch-up growth. In children undergoing puberty, annual statural gain was greater in group B (5.6 cm versus 3.2 cm in group A: p less than 0.001). Group A children were switched to alternate-day corticosteroid therapy one year after initiation of the study and exhibited improved growth after this change. No patient had renal function deterioration under alternate-day corticosteroid therapy, throughout the study period. Alternate-day prednisone should be offered to pediatric renal transplant recipients with satisfactory renal function as a mean for protecting growth potential.     

Renal outcome of children with one functioning kidney from birth. A study of 99 patients and a review of the literature

In patients with a single functioning kidney, renal function was assessed at regular intervals over a period of 10 years. Serum creatinine, glomerular filtration rate (GFR), blood pressure, and urinary protein-creatinine ratio were assessed at the age of 2, 5 and 10 years. Between January 1980 and December 2005, 99 such patients were diagnosed in the first year of life. They were divided into three groups: A, patients with multicystic kidney disease and a normal contralateral kidney (n = 36); B, patients with a normal solitary kidney without uropathy (n = 20); and C, patients with obstructive uropathy and one nonfunctioning kidney (n = 43). Serum creatinine levels increased significantly with increasing age in every group. In group C, serum creatinine was significantly elevated compared with group A in all age categories (p = 0.043, p = 0.019, p = 0.001 respectively). Median figures of GFR remained within normal limits over the 10-year period. GFR was significantly lower in group C compared with group A (p = 0.001, p = 0.009, p = 0.019 respectively) and B in all age categories (p = 0.013, p = 0.002, p = 0.016 respectively). There were no changes in blood pressure over time and no differences among the three groups were observed. At the age of 10 years, the patients in group C had a significantly higher median urinary protein-creatinine ratio (p = 0.022) than those in groups A and B. There was also an increasing level of proteinuria with increasing age in group C (p = 0.002). In conclusion, renal function was stable over time in all patients, but children with obstructive uropathy have a lower median GFR and higher serum creatinine level for the whole study period. Hypertension was exceptionally observed in group C, with obstructive uropathy, as was an elevated urinary protein-creatinine ratio.     

Effects of growth hormone therapy in prepubertal children with short stature secondary to intrauterine growth retardation

A total of 130 short children were included in a French multicentre study and randomized between a control group (group A) and two groups treated with daily subcutaneous injections of GH at doses of 0.7 IU/kg/week (group B) and 1.4 IU/kg/week (group C) for 2 years. Height velocity was significantly increased ( p <0.0005) in groups B and C, with a greater increase in group C than in group B ( p < 0.001). The benefit after 2 years compared with controls was 4.3 cm in group B and 5.9 cm in group C. The rate of bone maturation was not affected by GH therapy. These results led to the conclusion that 2 years of treatment with GH improves final height prognosis in children with short stature secondary to IUGR, and that this effect is dose dependent. The effect on final height has still to be demonstrated.     

Randomised controlled trial of effect of terbutaline before elective caesarean section on postnatal respiration and glucose homeostasis.

AIM: To determine if terbutaline given to mothers before elective caesarean section facilitates neonatal respiration and metabolism. METHODS: A randomised controlled trial of 25 full term infants delivered by elective caesarean section was conducted. The mothers received a continuous infusion of terbutaline or saline 120-0 minutes before birth. Umbilical artery blood was collected at birth and analysed for blood gases and catecholamines. The lung function of each infant was assessed two hours after birth, and blood pressure, heart rate, blood glucose and body temperature were monitored until 24 hours of age. RESULTS: The infants of the treated mothers (n = 13) had significantly higher dynamic lung compliance (p < 0.001), lower airway resistance (p < 0.001), and respiratory frequency than control infants (n = 12). Blood glucose and adrenaline concentrations were significantly higher in the treated group (p = 0.0014 and p < 0.01). None of these infants had any clinical respiratory difficulties; there were two cases of transient tachypnoea in the control group. No negative side effects due to the terbutaline treatment were seen among the infants. The mothers felt no discomfort caused by the terbutaline infusion, although they bled more during surgery (p = 0.03). CONCLUSION: Stimulation of the beta adrenoceptors in utero with terbutaline infusion to the mothers promotes neonatal respiratory and metabolic adaptation after elective caesarean section.     

Myocardial dysfunction in group B streptococcal shock

A rabbit model of group B Streptococcal (GBS) shock was used to determine if myocardial dysfunction contributes to GBS shock and, if so, to ascertain if prostaglandins modulate this dysfunction. The infusion of heat-killed GBS (group I) produced a dramatic decrease in the first derivative of left ventricular pressure with respect to time (LVdP/dt) from baseline values (p less than 0.05). LVdP/dt remained stable in rabbits pretreated with indomethacin (group II) and in saline-infused control rabbits (group III), and was significantly different at 30 min from LVdP/dt in group I (p less than 0.05). Values for group I mean arterial pressure, cardiac output, pulmonary vascular resistance, and heart rate and for pH and pO2 after GBS infusion were all significantly different from baseline values and from postinfusion values for groups II and III (p less than 0.05). Systemic vascular resistance and left ventricular end diastolic pressure did not change significantly in any group at any time interval. These results indicate a primary role for myocardial dysfunction in the pathogenesis of GBS shock, and suggest strongly that prostaglandins modulate GBS-induced myocardial dysfunction.     

The role of endothelin converting enzyme inhibition during group B streptococcus-induced pulmonary hypertension in newborn piglets

An endothelin-converting enzyme mediates the conversion from low-potency pro-endothelin to potent endothelin-1 (ET-1). Increased ET-1 levels have been observed in pulmonary hypertension of various etiologies in infants. We hypothesized that increased ET-1 levels induce pulmonary hypertension during group B Streptococcus (GBS) infusion, and this can be attenuated by the administration of an endothelin-converting enzyme inhibitor (ECEI). Twenty-two unanesthetized, chronically instrumented newborn piglets received a continuous infusion of GBS (3.5 x 10(8) colony-forming units/kg/min) while exposed to 100% O2. They were randomly assigned to receive a placebo (PL) or an ECEI (phosphoramidon, 30 mg/kg i.v.) 15 min after sustained pulmonary hypertension. Comparison of hemodynamic measurements and arterial blood gases at baseline and over the first 210 min from the onset of pulmonary hypertension was performed between groups. GBS infusion caused significant increases in mean pulmonary artery pressure, pulmonary vascular resistance (PVR), systemic vascular resistance (SVR), and PVR/SVR, and significant decreases in cardiac output, pH, and base excess. After the administration of ECEI, a significant reduction in pulmonary artery pressure (p < 0.0001), PVR (p < 0.001), and PVR/SVR (p < 0.01) and an improvement in cardiac output (p < 0.01) were observed during GBS infusion. The decrease in pH (p < 0.001) and base excess (p < 0.001) during GBS infusion was less marked after the administration of ECEI compared with the PL. Plasma ET-1 levels were obtained in 20 additional piglets; levels were significantly lower in the ECEI compared with PL after 3 h of GBS infusion (p < 0.02). All animals in the ECEI group survived the study period as opposed to 25% survival in the PL group (p < 0.001). These data suggest that the increased circulating ET-1 levels mediate, in part, the GBS-induced pulmonary hypertension.     

Protective effect of pentoxifylline on growth plate in neonatal rats following long-term phototherapy

We demonstrated previously that receiving long-term phototherapy was associated with early impairment of growth plate structure in neonatal rats, and oxidative stress may be the main risk factor for growth plate injury. The purpose of this study was to examine the histomorphometric effects of pentoxifylline treatment on the growth plate. Sixty weanling Sprague-Dawley rats were randomly separated into three equal groups. Group A, the control group, did not receive phototherapy and pentoxifylline. Groups B and C were exposed to phototherapy for 7 d. In addition to phototherapy, group C was also given pentoxifylline during the study period. Compared with zonal lengths on d 7 after initiation of phototherapy, group B had significantly lower values than group A for all zonal lengths (p < 0.001). Zonal lengths of growth plate were increased significantly with pentoxifylline treatment in group C for 7 d compared with group B (p < 0.001). After phototherapy, group B had significantly higher values than groups A and C for plasma malondialdehyde levels (p < 0.001). The pentoxifylline was found here to have some potential to reduce the effects of phototherapy on growth plate in neonatal rats at a relatively low dose.     

Psychosocial implications of Thalassemia Major

BACKGROUND: Many causes including the chronicity of disease, burden of treatment modalities, morbidities, and the expectation of early death resulting from the disease complications, may lead to psychosocial burden in Thalassemia Major (TM) patients. METHODS: A total of 38 patients with TM and their mothers were recruited to evaluate the psychosocial burden as well as to disclose whether the psychological status of the patients contribute to the compliance with the therapy or to the contrary. Demographic and disease variables were obtained. Child Behavior Check-list (CBCL) was completed by the mothers of the patients. A detailed psychiatric interview based on the 4th edition of the Diagnostic and Statistical Manual diagnostic criteria was performed for each patient. Symptom Distress Checklist 90 (SCL-90) scale was given to all mothers for evaluating their psychopathology. RESULTS: Although CBCL scores remained between the normal ranges, desferrioxamine mesylate (DFO)-compliant patients and the patients with lower ferritin values had significantly higher scores. A total of 24% of the patients had a psychiatric diagnosis including major depression, anxiety disorder, tic disorder, and enuresis nocturnal. The psychiatric diagnosis was significantly higher in the patients who were compliant with desferrioxamine compared with the non-compliant group (P = 0.007). The SCL-90 scores indicated that the mothers who had a child with good adherence to DFO had higher scale scores than the mothers with a poor adherent child. CONCLUSIONS: The increase risk of psychosocial and behavioral problems in thalassemics and their parents indicated the importance of a lifelong psychosocial support for the prevention of mental health issues. The patients and their parents, who were more conscious of the illness, were more worried but more compliant with the therapy and need stronger psychiatric support.     

唤醒疗法与去氨加压素治疗儿童原发性遗尿症的非随机对照试验

摘要 目的 比较人工唤醒、闹钟、报警器3种唤醒治疗方式与去氨加压素治疗原发性遗尿症（PNE）患儿的疗效和依从性。方法 纳入首都医科大学附属北京儿童医院2012年4月至2013年8月就诊的6~14岁PNE患儿,根据就诊先后顺序分为人工唤醒、闹钟、报警器和去氨加压素组。4组均在基础治疗上给予相应干预,记录遗尿日记。于治疗1、3、6个月随访时,评估疗效、依从性和安全性,并行意向性分析。疗效以遗尿症状好转为评价指标。采用logistic回归分析依从性、遗尿家族史、膀胱容量等因素与疗效的相关性。结果 120例患儿符合纳入和排除标准进入研究,每组各30例。①去氨加压素、报警器、闹钟和人工唤醒组的治疗6个月的总有效率为76.7%、93.3%、56.7%和76.7%,报警器组显著高于闹钟组（P<0.0125）;去氨加压素组和报警器组、人工唤醒组总有效率差异无统计学意义(P≥0.0125)。②报警器组27/30例（90.0%）依从性较好,其次为去氨加压素（24/30,80.0%）、人工唤醒（18/30,60.0%）和闹钟组（16/30,53.3%）; 报警器组优于人工唤醒组（P=0.007）和闹钟组（P=0.002）,报警器组和去氨加压素组差异无统计学意义。③Logistic回归分析结果显示,存在PNE家族史是影响治愈的危险因素（P=0.007,OR=0.204,95%CI: 0.064~0.652）。④报警器组1例出现湿疹,去氨加压素组观察到鼻出血1例,低钠血症2例。结论 报警器、去氨加压素治疗PNE的疗效相近,且依从性和安全性均较好,可作为PNE患儿的首选治疗。     

Estimating the final outcome of brace treatment for idiopathic thoracic scoliosis at 6-month follow-up

OBJECTIVE: The study was conducted on the possibility of predicting the final outcome of bracing for idiopathic scoliosis at a follow-up period of 6 months. METHODS: In a retrospective study, 62 adolescent female patients with right thoracic scoliosis (20-40 degrees Cobb angle) treated with a brace were examined. A new compliance score was developed. The sample was divided into four groups based on compliance (compliance score) and initial correction (half-year after start bracing): group A, good compliance/high initial correction; group B, good compliance/low initial correction; group C, bad compliance/high initial correction; group D, bad compliance/low initial correction. The final outcome (1 year after weaning) was defined as successful if a curve correction of at least 5 degrees was achieved. The influence of factors on final outcome was analysed by ANOVA. Differences between continuous data were analysed by a two-sample Wilcoxon test. RESULTS: The overall final outcome was not successful (thoracic curve -3 degrees). However, the average outcome of the compliant group was successful (-5 degrees), while no success was achieved without good compliance (+5 degrees). High initial correction of more than 40% (p < 0.002) and good compliance (p< 0.004) were of significant impact for the outcome. Patients showing good compliance and high initial correction presented a successful outcome of 7 degrees Cobb angle. CONCLUSION: Compliant patients with a high initial correction can expect a final correction of around 7 degrees, while compliant patients with low initial correction may maintain the curve extent. Bad compliance is always associated with curve progression.     

Colostomy type in anorectal malformations: 10-years experience

AIM: The aim of this study was to evaluate the influence of colostomy type on morbidity during the treatment of anorectal malformations. METHODS: Sixty-eight infants (male: female ratio 1.3:1) with anorectal malformations that required colostomy were treated in our clinics during the period 1991-2001. Of these patients, 26 had received a loop colostomy: 14 of these underwent posterior sagittal anorectoplasty (PSARP) at the age of 9-12 months (Group A), and 12 underwent PSARP at the age of 2-4 months (Group B). Forty-two infants received a separated-stomas colostomy and underwent PSARP at the age of 9-12 months (Group C). The incidence of complications among groups was compared using the 2 sided Fisher's exact test. RESULTS: Eight cases from group A were complicated with prolapse of the stomas, perianal wound infection, pull-through dehiscence, and anal fibrotic stricture. The only complication observed in groups B and C was perianal wound infection, which occurred in 1 case from each group. A statistically significant difference was observed in the incidence of complication between groups A and C (p<0.001) and between groups A and B (p=0.014). The results from groups B and C did not differ significantly (p=0.398). When the cases complicated with colostomy prolapse were removed from the statistical analysis, groups A and C still differed significantly (p=0.001) but groups A and B did not (p=0.069). CONCLUSIONS: As the incidence of complications increases with time after a loop colostomy, we encourage either an early corrective procedure or the modification into separated-stomas colostomy (SSC) before PSARP is performed for those cases that would involve definitive surgery in late infancy.