Survival of thalassemic patients referred to the Boo Ali Sina Teaching Hospital, Sari, Iran

Beta-thalassemia major (TM) is the most prevalent genetic disease in Mazandaran Province. Currently, about 2,700 TM patients have been registered and are under treatment in the province. This study was undertaken to evaluate the survival of patients attending a dedicated clinic at the Boo Ali Sina Teaching Hospital, Sari, Iran, which was established in 1986. This survival analysis was conducted from July 2004 to September 2006. New deaths were updated in September 2006. A total of 1,010 medical records were reviewed. Place of residence, date of birth, first transfusion, initiation date of chelation, diagnosis of cardiac complications, diabetes and death were recorded. Compliance to treatment assessed by calculating the percentage of actual usage to prescribed iron chelator medicine and also by asking the attending nurse who was constantly present throughout the last 15 years. Validity of the opinion of the nurse was ascertained in a previous study. Kaplan-Meier statistics were used for analysis. The odds ratio (OR) and its 95% confidence interval (CI) for some risk factors of death were calculated. The survival rate of patients born before and after 1986 was also compared. Survival of both genders for birth cohort before and after 1986 was not statistically significant although the cohort of patients born after 1986 was better at 30 years old (68 vs. 80%). The survival of TM patients is improving but the prevalence of complications is high.     

Cardiac related death in thalassaemia major: time trend and risk factors in a large Greek Unit

Background: Cardiac complications are the leading cause of death in thalassaemic patients (TM) worldwide. Improved management protocols including new chelators and imaging have reduced cardiac-related deaths but also require more advanced analytical methods to reflect temporal fluctuations in mortality risk. Objectives: To evaluate time patterns of risk of cardiac death in TM patients according to birth cohort, gender and degree of haemosiderosis and to apply new flexible methods of analysis that may allow assessment of trends in risks as affected by innovations in management. Design and methods: Cardiac-free survival and time trend of the risk of cardiac-related death were assessed in 648 transfusion-depended thalassaemic patients (48.3% females) managed in a single unit located in Athens, Greece. Patients were classified according to birth cohort (prior or after 1/1/1975) and according to the severity of haemosiderosis (mild, moderate and severe) as estimated by the mean ferritin levels during the last 5 yr of follow-up. Results: The median time of observation was 27.8 yr and 84 cardiac deaths were recorded. A parametric analysis predicted a non-monotonous time pattern for the cardiac risk with an initial increasing phase until the fourth decade of life followed by a drop off in deaths. Women have 23% longer life expectancy than men (P = 0.010) while patients born after 1975 die of heart complications at an older age compared to those born prior to 1975 (time ratio = 1.34, P = 0.003). Patients with mild and moderate haemosiderosis lived 89% (P < 0.001) and 43% (P < 0.001) longer, respectively, compared with patients with severe haemosiderosis. Conclusions: These results on risk of death in thalassaemia, accord with expectations. This type of analysis will be useful in the future when the expected impact of cardiac MRI with appropriate tailoring of chelation therapy using all current and future options will modify the pattern of risk of cardiac death observed to date.     

Trends in the seroprevalence of HTLV-1 in Japanese blood donors in Nagasaki Prefecture, 2000–2006

Human T cell leukemia virus type-1 (HTLV-1) is the established cause of adult T cell leukemia/lymphoma. Monitoring time trends in HTLV-1 seroprevalence in blood donors is important to assess the safety of the blood supply in the viral endemic area. We analyzed changes in HTLV-1 seroprevalence in 48415 first-time blood donors who donated blood from 2000 to 2006 in Nagasaki prefecture, an endemic area in Japan. The donors were divided into 10-year birth cohorts: before 1950, 1951–1960, 1961–1970, 1971–1980, and 1981–1990. Among the first-time blood donors, 622 were tested positive for HTLV-1 [overall seroprevalence: 1.28%, (95% CI 1.19–1.39)]. Seroprevalence was significantly high in the birth cohort of before 1950 (6.22%) and declined with birth-year. The time trend of the birth-cohort-specific seroprevalence showed almost no change within each birth cohort, except for the birth cohort of 1981–1990 that showed a significantly declining trend (P for trend = 0.006). Among the birth cohort of 1981–1990, the seroprevalence was stable among those born during 1981–1986 (0.66–0.83%), but was lower among those born during 1987–1990 (0–0.38%). Detail analyses showed that HTLV-1 seroprevalence among blood donors clearly declined in those born after 1987.     

Color flow Doppler in the diagnosis of double-chambered right ventricle: a demographic and echocardiographic study

The purpose of this study was to evaluate the demographic and echocardiographic data of patients diagnosed with double-chambered right ventricle and attempt to explain a perceived rise in the incidence. Definition: Double-chambered right ventricle (DCRV) is a division of the right ventricle into two chambers by a hypertrophied muscle bundle. Methods: The medical records of patients diagnosed with DCRV were reviewed, and demographic, echocardiographic, and catheterization data were tabulated. Annual incidence of DCRV, based on year of birth, was compared to yearly detection rate, based on year of DCRV diagnosis. To evaluate the influence of color flow Doppler on the frequency of diagnosis of DCRV, demographics of patients born prior to September 1986 (when utilization of color Doppler began in our institution) were compared to those born after that date. Results: Despite an unchanged annual incidence of DCRV, yearly detection rate of this lesion rose significantly following the introduction of color flow Doppler to our institution (September 1986). DCRV was diagnosed earlier and was accompanied by earlier catheterization, which also showed lower right ventricular body gradients after September 1986. Associated anomalies, both cardiac and noncardiac, in our population differed from those reported in previous series. Conclusion: This study infers that the advent of color flow Doppler significantly enhanced the diagnosis of DCRV in our pediatric patients and led to a perceived rise in incidence.     

The era of comparable life expectancy between thalassaemia major and intermedia: Is it time to revisit the major‐intermedia dichotomy?

In the last few decades, the life expectancy of regularly transfused β‐thalassaemia major (TM ) patients has dramatically improved following the introduction of safe transfusion practices, iron chelation therapy, aggressive treatment of infections and improved management of cardiac complications. How such changes, especially those attributed to the introduction of iron chelation therapy, improved the survival of TM patients to approach those with β‐thalassaemia intermedia (TI ) remains unknown. Three hundred and seventy‐nine patients with TM (n  = 284, dead 40) and TI (n  = 95, dead 13) were followed retrospectively since birth until 30 June 2015 or death. Kaplan‐Meir curves showed statistically significant differences in TM and TI survival (P  <  0·0001) before the introduction of iron chelation in 1965, which were no longer apparent after that date (P  =  0·086), reducing the Hazard Ratio of death in TM compared to TI from 6·8 [95% confidence interval (CI ) 2·6–17·5] before 1965 to 2·8 (95% CI 0·8–9·2). These findings suggest that, in the era of iron chelation therapy and improved survival for TM , the major‐intermedia dichotomy needs to be revisited alongside future directions in general management and prevention for both conditions.     

Confounding by contraindication in a nationwide cohort study of risk for death in patients taking ibopamine

BACKGROUND: Outcomes may differ in treated and untreated patients because of a contraindication for treatment in the latter that is independently associated with the outcome of interest. OBJECTIVE: To evaluate the effects of confounding by contraindication on risk factors for death in patients taking ibopamine after its use was restricted in early September 1995. DESIGN: Retrospective cohort study. SETTING: The Netherlands. PATIENTS: 1146 patients with congestive heart failure who were prescribed ibopamine at least once and for whom medication history and medical data were available. MEASUREMENTS: Cardiovascular risk factors, clinical characteristics, and medication use. Each patient was assigned an index date (the date of death, or a random date for patients still alive at the end of the study). RESULTS: In univariate analyses comparing patients with an index date before and those with an index date after 8 September 1995, the relative risk for death associated with current use of ibopamine was 3.02 (95% CI, 2.12 to 4.30) compared with 0.71 (CI, 0.53 to 0.96), respectively. In multivariate analyses, the risk for death was 2.62 (CI, 1.76 to 3.90) and 0.93 (CI, 0.84 to 1.02), respectively. CONCLUSION: The marked inversion of the relative risk estimate can be considered a practical example of confounding by contraindication.     

Management of patients with biliary atresia in France: results of a decentralized policy 1986-2002

This study analyzed the results of the decentralized management of biliary atresia (BA) in France, where an improved collaboration between centers has been promoted since 1997. Results were compared to those obtained in England and Wales, where BA patients have been centralized in three designated centers since 1999. According to their birth dates, BA patients were divided into two cohorts: cohort A, with patients born between 1986 and 1996, had 472 patients; and cohort B, with patients born between 1997 and 2002, had 271 patients. Survival rates were calculated according to the Kaplan-Meier method and compared by using the log rank test and the Cox model. Four-year overall BA patient survival was 73.6% (95% CI 69.5%-77.7%) and 87.1% (CI 82.6%-91.6%) in cohorts A and B, respectively (P < .001). Median age at time of the Kasai operation was 61 and 57 days in cohorts A and B, respectively (NS). Four-year survival with native liver after the Kasai operation was 40.1% and 42.7% in cohorts A and B, respectively (NS): 33.9% (cohort A) and 33.4% (cohort B) in the centers with two or fewer caseloads a year, 30.9% (cohort A) and 44.5% (cohort B) in the centers with 3-5 cases/year, 47.8% (cohort A) and 47.7% (cohort B) in the center with more than 20 caseloads a year. In cohorts A and B, 74 (15.7%) and 19 (7%) patients, respectively, died without liver transplantation (LT). Four-year survival after LT was 75.1% and 88.8% in cohorts A and B, respectively (P = .006). In conclusion, BA patients currently have the same chance of survival in France as in England and Wales. The early success rate of the Kasai operation remains inferior in the centers with limited caseloads in France, leading to a greater need for LTs in infancy and early childhood.     

Current results of management in transposition of the great arteries, with special emphasis on patients with associated ventricular septal defect

Two hundred forty-five patients less than 15 days of age with transposition of the great arteries with or without a ventricular septal defect or pulmonary stenosis were entered into an ongoing 20 institution treatment study between January 1, 1985 and June 1, 1986. Complete follow-up is available on all patients. The ventricular septal defect narrowed in only 1 of 36 patients with combined transposition of the great arteries and ventricular septal defect; pulmonary stenosis developed or worsened in 3 of these 36 patients and in 3 of the 187 patients with simple transposition. Twelve month overall survival among the 245 patients was 80%. No morphologic feature of transposition was a risk factor for death but major associated cardiac and noncardiac anomalies (more common in patients with combined transposition and ventricular septal defect) and low birth weight were risk factors. Neither arterial switch repair (n = 86), atrial switch (Mustard) repair (n = 21) nor atrial switch (Senning) repair (n = 39) was a risk factor for death, but results in all surgical groups were better in the last part of the experience. Death before repair was less frequent late in the study. Possibly, in low birth weight infants, survival was better with the arterial than with the atrial switch repair. These data suggest that survival at 1 year is similar with either the arterial or the atrial switch repair. The early results of repair of combined transposition of the great arteries and ventricular septal defect are as good as those of simple transposition. Special institutional efforts are required to attain good results with the arterial switch repair and to prevent death before repair.     

Effects of the infant stool color card screening program on 5-year outcome of biliary atresia in Taiwan

In Taiwan, a screening system using an infant stool color card to promote the early diagnosis of biliary atresia (BA) was established in 2002. This study aimed to investigate the 5-year outcome of BA before and after using the screening program. BA patients were divided into three cohorts according to their birth dates. The patients in cohort A (n = 89) were born before the stool card screening program (1990-2000); those in cohort B (n = 28) were screened by the stool card regional screening program (2002-2003); and those in cohort C (n = 74) were screened by the stool card universal screening program (2004-2005). The relative odds ratios were computed using logistic regression to compare the different factors affecting survival time. The rate of age at Kasai operation <60 days was 49.4% and 65.7% in cohorts A and B+C, respectively (P = 0.02). The jaundice-free (total serum bilirubin <2.0 mg/dL) rate 3 months after surgery was 34.8% and 60.8% in cohorts A and B+C, respectively (P < 0.001). The 3-year jaundice-free survival rate with native liver was 31.5% in cohort A and 56.9% in cohort B+C (P < 0.001), whereas the 3-year overall survival rates were 64.0% and 89.2%, respectively (P < 0.001). The 5-year jaundice-free survival rate with native liver was 27.3% in cohort A and 64.3% in cohort B (P < 0.001), and the 5-year overall survival rates were 55.7% and 89.3%, respectively (P < 0.001). CONCLUSION: The stool color card screening program for BA allows for earlier Kasai operation, which increases the jaundice-free rate at 3 months postsurgery. With higher surgical success rates, the 3- and 5-year outcome of BA patients in Taiwan improves remarkably.     

Birth prevalence and survival in cystic fibrosis: a national cohort study in the Netherlands

BACKGROUND: Birth prevalence and survival in patients with cystic fibrosis (CF) in the Netherlands were last investigated > 30 years ago. However, since then the birth prevalence may have decreased because of genetic counseling and an increased number of newborns of non-European descent. Although survival of CF patients has increased worldwide, a significantly lower median age at death was recently reported in the Netherlands compared with data from the United States. OBJECTIVES: To analyze birth prevalence and survival in CF patients in the Netherlands, and to compare this survival data with US CF data. DESIGN: Survey of all CF patients living in the Netherlands, and analysis of Dutch CF mortality statistics using data from the Dutch central statistics office, Statistics Netherlands (Voorburg, the Netherlands), and a comparison with Cystic Fibrosis Foundation (Bethesda, MD) patient registry data. SETTING: All CF centers in the Netherlands and the United States. PARTICIPANTS: All CF patients treated in the Netherlands on January 1, 2001, and all persons who died of CF between 1974 and 2000, and an equivalent US population. MEASUREMENTS: Birth prevalence and birth cohort-specific survival. RESULTS: The overall birth prevalence of CF for 1974 to 1994 was 1 in 4,750 live births, which is a considerable decrease compared with 1961 to 1965 (1 in 3,600 live births). Estimated survival to 30 years increased from 6% in the 1950-to-1954 cohort, to 36% in the 1970-to-1973 cohort. Exact survival could be calculated from 1974 onwards. Survival to 15 years increased from 72% from the 1974-to-1979 cohort, to 91% in the 1985-to-1989 cohort. Survival in the United States in the 1980-to-1984 cohort was better compared to the Netherlands, but this difference has disappeared over subsequent cohorts. CONCLUSIONS: The actual birth prevalence of CF in the Netherlands is clearly lower than it was 30 years ago. Survival in CF has dramatically improved. The difference in survival between the Netherlands and the United States, as observed in the cohorts born > 20 years ago, has disappeared.     

A phase III comparative trial of m-BACOD vs m-BNCOD in the treatment of stage II-IV diffuse non-Hodgkin's lymphomas

From September, 1984 to July, 1986 seventy previously untreated adult patients with advanced non-Hodgkin's lymphoma of intermediate or high grade histology were enrolled in an open phase III multicenter, comparative chemotherapeutic trial. The objectives were to compare, by means of response rate, duration of response and survival time, the efficacy and safety of substituting mitoxantrone for doxorubicin in the combination chemotherapy regimen m-BACOD (methotrexate, bleomycin, doxorubicin, cyclophosphamide, vincristine and dexamethasone). Thirty-five patients were randomly assigned to receive m-B-NOVANTRONE TM (mitoxantrone)-COD (m-BNCOD) and 35 m-B-ADRIAMYCIN TM (doxorubicin)-COD. The complete response rate was 57% for both treatment groups, and no significant disease-free survival and survival differences were found between the two groups of patients. Patients treated with m-BACOD experienced severe alopecia more frequently (p less than 0.001) and, after 10 cycles, six adverse cardiac event of WHO grade greater than 1, as opposed to none among those who received m-BNCOD. The mitoxantrone-containing regimen (m-BNCOD) has equivalent efficacy and reduced toxicity compared to the standard doxorubicin-containing regimen (m-BACOD) in patients with poor prognosis NHL.     

Survival in Duchenne muscular dystrophy

Objective:

To determine the survival in a population of German patients with Duchenne muscular dystrophy.

Patients and methods:

Information about 94 patients born between 1970 and 1980 was obtained by telephone interviews and questionnaires. In addition to age of death or actual age during the investigation, data concerning clinical course and medical interventions were collected.

Results:

67 patients with molecularly confirmed diagnoses had a median survival of 24.0 years. Patients without molecular confirmation (clinical diagnosis only) had a chance of 67 % to reach that age. Grouping of our patient cohort according to the year of death (before and after 2000), ventilation was recognized as main intervention affecting survival with ventilated reaching a median survival of 27.0 years. For those without ventilation it was 19.0 years.

Conclusion and clinical relevance:

our study provides survival data for a cohort of DMD patients in Germany stratified by year of death. Median survival was 24.0 years in patients confirmed by molecular testing. Ventilated patients had a median survival of 27 years. We consider this piece of information helpful in the medical care of DMD patients.Key words: duchenne muscular dystrophy, survival, ventilation     

The prevalence of hepatitis C in England and Wales

OBJECTIVES: To estimate the background population prevalence of hepatitis C in England and Wales, observe the prevalence over time and assess the extent of infection outside of known risk groups. METHODS: Sera from residual specimens from adult patients submitted to laboratories in England and Wales were tested for anti-HCV. Testing was carried out using a cost-effective pooling strategy. RESULTS: Although the prevalence of anti-HCV was highest in 1986 (1.07%), in the multivariable analysis, prevalence did not vary significantly between the 3 periods 1986, 1991 and 1996 (P=0.14). The prevalence of infection was higher in males than in females (P=0.0013). An age-period-cohort analysis revealed a cohort effect due to a lower HCV prevalence in the most recent birth cohorts, that is, those born between the calendar years 1971-1975 and 1976-1980. CONCLUSIONS: The majority of HCV infections in England and Wales were probably acquired before 1986. Infections in younger males identified in 1996 may signify more recent acquisition by injecting drug use.     

Mother-child transmission of HIV-1 and infant survival in Brazzaville, Congo

The aim of this study was to compare the probability of survival of infants born to anti-HIV-1-positive and anti-HIV-1-negative mothers. One thousand, eight hundred and thirty-three pregnant women, recruited sequentially in two mother-child clinics in Brazzaville, were screened for anti-HIV-1 (by enzyme-linked immunosorbent assay with confirmation by Western blot). Each seropositive mother (71 out of 1833, 3.9%) was matched for age, presumed date of delivery and place of residence with two seronegative mothers. Sixty-four babies born to anti-HIV-1-positive mothers and 130 control babies born to anti-HIV-1-negative mothers were followed up for 12-22 months (mean, 18 months). The probabilities of survival were estimated by the Kaplan-Meier method. At birth, the two groups of babies did not differ with regard to rate of stillbirths, gestational age, sex ratio and weight. Among babies born to seropositive mothers, the probability of survival was 0.87 (s.d. 0.04) at 3 months, 0.71 (s.d. 0.06) at 6 months, 0.68 (s.d. 0.06) at 9 months and 0.61 (s.d. 0.06) at 12.5 months. In the controls the probability of survival was 0.98 (s.d. 0.01) at 3 months and 0.97 (s.d. 0.02) at 12 months. The excess of mortality in the babies born to anti-HIV-1-positive mothers is highly significant (P less than 0.001). The deaths occurred more frequently and earlier than in similar cohort studies performed in developed countries.     

Incidence of melanoma and other malignancies among rheumatoid arthritis patients treated with methotrexate

OBJECTIVE: To determine cancer risk in a cohort of 459 rheumatoid arthritis (RA) patients treated with methotrexate in community practice. METHODS: All RA patients who started methotrexate prior to June 1986 and were attending 1 of 6 rheumatologists were studied. Demographic data were matched to the State Cancer Registry to identify all malignancies (except nonmelanoma skin cancer) for 1983-1998, and to the National Death Index to identify all deaths to the end of 1999. Followup started on the date when methotrexate was started and ended either on the last confirmed date on which the patient was seen by the rheumatologist or at death. Standardized incidence ratios (SIRs) were calculated using state population cancer rates stratified by sex, age (in 5-year groups), and calendar year. RESULTS: There were 4,145 person-years of followup (average 9.3 years). Eighty-seven malignancies were identified (14 before, 64 during, and 9 after the followup period). There was an estimated 50% excess risk of malignancy among methotrexate-exposed RA patients relative to the general population (SIR 1.5, 95% confidence interval [95% CI] 1.2-1.9), with a 3-fold increase in melanoma (SIR 3.0, 95% CI 1.2-6.2), a 5-fold increase in non-Hodgkin's lymphoma (SIR 5.1, 95% CI 2.2-10.0), and an almost 3-fold increase in lung cancer (SIR 2.9, 95% CI 1.6-4.8). CONCLUSION: Compared with the general population, methotrexate-treated RA patients have an increased incidence of melanoma, non-Hodgkin's lymphoma, and lung cancer. There may be a role for regular skin cancer screening for all RA patients, particularly those receiving immunosuppressive therapy.     

Outcome and resource utilization of infants born with hypoplastic left heart syndrome in the intermountain west

The objective of the present study was to characterize the outcomes and resource utilization of all infants born with hypoplastic left heart syndrome (HLHS) in the Intermountain West. This was a retrospective cohort study of all infants born with HLHS in the Intermountain West from January 1995 and January 2010. The cohort was divided into 3 eras: era 1, 1995 to 1999; era 2, 2000 to 2004; and era 3, 2005 to 2010. Cox proportional hazards regression analysis was performed to assess mortality. The lifetime hospitalization days and charges were also determined. Of the 245 infants identified, 65% were male infants and 172 (70%) underwent Stage 1 palliation. The transplant-free survival rate for the entire cohort was 33% at 14 years. The 1-year transplant-free survival rate for the surgical cohort was 60% in era 3. The infants whose initial presentation included shock, restrictive or intact atrial septum, chromosomal defects, or multiorgan dysfunction had an increased risk of death. A recent era of birth, greater birthweight, and older gestational age were associated with improved survival. The factors associated with mortality after stage 1 included surgical procedure type (Blalock-Taussig vs Sano shunt, hazard ratio 2.1), requirement for postoperative extracorporeal membrane oxygenation (hazard ratio 4.2), postoperative renal dysfunction (hazard ratio 3.0), anomalous pulmonary venous return (hazard ratio 2.9), and moderate or greater tricuspid valve regurgitation at any point (hazard ratio 2.0). For patients who had undergone stage 1, 2, or 3 palliation, the median cumulative lifetime hospitalization was 32, 48, and 65 days, and the median cumulative lifetime charges for hospitalization were $201,812, $253,183, and $296,213, respectively. In conclusion, although hospital-based studies of HLHS have shown significantly improved survival after surgical palliation, population-based studies have shown that HLHS continues to have a high mortality and high resource utilization.     

Truncating mutations in Peutz-Jeghers syndrome are associated with more polyps, surgical interventions and cancers

Background and Goals

Peutz-Jeghers syndrome (PJS) is a rare autosomal dominant polyposis syndrome caused by STK11 germline mutations. PJS is associated with an increased risk of cancer. In our cohort, clinical and phenotypic parameters were correlated with genotypic findings and patients were prospectively followed by surveillance.

Study

Thirty-one patients treated between 2000 and 2006, were evaluated. STK11 genotyping was performed and phenotypes of patients with truncating (TM) and nontruncating mutations (NTM) were compared.

Results

Median age at first symptoms was 11 years and complications occurred before the age of ten in 42% of patients. STK11 mutations were detected in 16 of 22 families (12 TM; four NTM). Patients with TM had more surgical gastrointestinal (GI) interventions (p = 0.021), and female patients in the TM group had an increased risk of undergoing gynecological surgery (p = 0.016). Also, there was a trend towards a higher polyp count (p = 0.11) and earlier age at first polypectomy (p = 0.13) in the TM group. Ten carcinomas were detected in six patients resulting in a cancer risk of 65% up to the age of 65 years. Patients with TM tended to develop more cancers (p = 0.10). Importantly, our surveillance strategy used detected 50% of cancers (n = 5) at an early potentially curable stage.

Conclusions

Our study shows that almost half of PJ patients have complications early in life independent of mutational status. Patients with TM require more surgical GI interventions and tend to develop more polyps and cancers. Furthermore, close surveillance detects early stage cancers in patients. We propose that surveillance should be started as early as 8 years in all patients to avoid complications. Moreover, patients with TM may benefit from surveillance at shorter intervals.     

β-Thalassemia in childhood: Current state of health in a high-income country

β-thalassemia is an haemoglobinopathy characterized by a defective synthesis of the β-globin chain. To assess the current state of health of paediatric patients with β-thalassemia, data from the French national registry regarding children born between 2005 and 2020 with β-thalassemia intermedia (TI) or major (TM) were collected. A total of 237 patients (median age 7.1 years at last visit) were analysed, of whom 156 (65.8%) were born in France and 162 (68.4%) had a TM phenotype. The probability of survival for children with TM born in France was 98.3% at 15 years. Fifty-four (22.8%) children received a haematopoietic stem cell transplant with a success rate of 88.8%. Hepatic and cardiac iron overload monitoring in non-transplanted patients showed moderate overload in 15.7% (18/115) and 7.1% (7/99) of cases, respectively, while clinical complications were found in only 4 patients with TM (hepatic in 3 cases). At last visit, mean ferritinemia was 1293 ng/ml (±759). Overall, less than 10% of children underwent splenectomy. No significant impact of the disease on growth or academic achievement was observed. Deferasirox was the main first-line chelator, prescribed in 78.2% of cases, with side effects reported in 11.7% of instances.     

Disease progression and early viral dynamics in human immunodeficiency virus-infected children exposed to zidovudine during prenatal and perinatal periods

Zidovudine (Zdv) is widely used to reduce maternal-infant human immunodeficiency virus transmission (HIV), but its consequences for disease progression among children infected despite Zdv exposure remain unknown. In a multicenter observational cohort study of 325 HIV-infected children born during 1986-1997, clinical progression was compared among infected children exposed or unexposed to Zdv during prenatal and perinatal periods. Zdv exposure was associated with 1.8-fold (95% confidence interval, 1.02-3.11) increased risk of progressing to AIDS or death after adjusting for year of birth, maternal CD4 cell count, maternal AIDS diagnosis, and subsequent antiretroviral therapy of the child. Mean log(10) viral copies at 7-12 weeks were higher among Zdv-exposed children (P=.004). No infected child treated early with multidrug therapy progressed to AIDS or died by 1 year, regardless of early Zdv exposure. More rapid disease progression was observed among infected children exposed during pregnancy or birth to Zdv if effective multidrug therapy was not initiated.     

Reduced prevalence of hepatitis B surface antigen positivity among pregnant women born after the national implementation of immunoprophylaxis for babies born to hepatitis B virus‐carrier mothers in Japan

Aim

We aimed to estimate hepatitis B surface antigen (HBsAg) positivity among birth year‐stratified pregnant women in Hiroshima, Japan, and compare prevalence rates between women born before and after implementation of a national immunoprophylactic vaccination program for babies born to hepatitis B virus (HBV) carrier mothers in Japan.

Methods

Pregnant women who gave birth at all delivery hospitals/clinics in Hiroshima prefecture between 1 April 2010 and 31 March 2011 were eligible. Lists collected from each institution included survey items such as age (pregnant woman's birth year) and HBsAg and hepatitis C virus (HCV) antibody (anti‐HCV) test results, which were posted anonymously and non‐consolidated from medical records. We calculated the HBsAg and anti‐HCV prevalence in our cohort according to the mothers' birth year.

Results

In 41 of 58 hospitals/clinics, 15 233 and 15 035 pregnant women underwent HBsAg and anti‐HCV testing, corresponding to 59.6% and 58.9% of 25 546 births in the 2010 fiscal year, respectively. The overall HBsAg positive rate was 0.51% (95% confidence interval [CI], 0.40–0.63%), and an extremely low prevalence (0.10%; 95% CI, 0.00–0.25%) was observed among pregnant women born after 1986. However, the prevalence in this study was slightly higher than the nationwide value (0.31%) and the Chugoku region‐specific value (0.46%) among first‐time blood donors at Japanese Red Cross blood centers between 2001 and 2006. No significant difference in anti‐HCV positivity was observed.

Conclusion

Only two pregnant women born after the preventive program implementation were HBsAg‐positive. Perinatal HBV transmission is estimated to be almost completely inhibited in the next generation.