Danaparoid reduces transplant‐related mortality in stem cell transplantation for children

In SCT, death from transplant‐related complications is the major obstacle hindering improvement of transplant outcomes, and proper supportive care is essential to reduce TRM. The transplant outcomes of 210 pediatric patients with malignant and non‐malignant disorders who consecutively underwent SCT in our institution from 2000 to 2013 were analyzed. The transplant years were divided into three periods: A (2000‐2004), B (2005‐2008), and C (2009‐2013), and an improvement in 5‐year OS and a decrease in 5‐year TRM were observed over these time periods; that is, OS was 61.5%, 60.3%, and 79.5% (P = .062), and TRM was 19.9%, 7.9%, and 0.0% (P < .001) in periods A, B, and C, respectively. On multivariate analysis, the prognostic factor for TRM for all patients was administration of danaparoid (HR = 0.109, 95% CI = 0.033‐0.363, P < .001), and for patients with hematological malignancies in allogeneic SCT, the prognostic factors were danaparoid (HR = 0.046, 95% CI = 0.006‐0.326, P = .002) and advanced disease at SCT (HR = 4.802, 95% CI = 1.734‐13.30, P = .003). A reduction in TRM after SCT was observed over the time periods, and supportive care with danaparoid was found to be significantly effective in reducing TRM in SCT for children.     

Pediatric respiratory tract diseases: Chronological trends and perspectives

Background

The aim of this study was to describe the epidemiological profile of childhood respiratory tract diseases (RTD) in the region of Sfax, Tunisia, and to evaluate their trends over a 13 year period.

Methods

We conducted a retrospective study of all children hospitalized with RTD aged under 14 years. We collected data from the regional morbidity register of the university hospital of Sfax from 2003 to 2015.

Results

A total of 10 797 RTD patients were enrolled from 49 880 pediatric hospitalizations (21.7%). A male predominance was noted (60%). The median age was 8 months (IQR, 2–36 months). Acute bronchitis (AB) accounted for 53.8%, followed by asthma (15%), pneumonia (14%) and acute upper respiratory infection (AURI; 7.2%). The hospital incidence rate (HIR) of RTD was 34/10 000 inhabitants/year. It was 18.2; 5.07; 4.7 and 2.4/10 000 inhabitants for AB, asthma, pneumonia and AURI, respectively. We noted a significant increase in the HIR of RTD with an annual percentage change (APC) of 10.94% (P < 0.001); in the HIR of AB (APC, 5.27%; P < 0.001); and in asthma HIR (APC, 11.2%; P < 0.001). Otherwise, a significant decrease in AURI HIR was observed (APC, –8.8%; P < 0.001). AB lethality rate increased significantly, with an APC of 7.4% (P < 0.001). Projected trends analysis up to 2024 showed a significant rise in AB and in asthma, while AURI would significantly decrease.

Conclusions

RTD continues to be a serious health problem over time in terms of morbidity and mortality. Preventive and curative strategies are needed urgently.     

Educational and learning morbidity in pediatric heart transplant recipients: A pediatric heart transplant society study

Educational development is an important component of quality of life for children with heart transplant. Aims include determining prevalence of and risk factors for modified education placement in a large representative sample of pediatric heart transplant recipients. Participants included 1495 patients (age 6‐18 years) from the PHTS database. Data on education placement and clinical predictors were collected at listing and at 1 and 3 years post‐transplant. At listing, 88% of patients were in typical education placement, while 12% were in modified education. Males (P = .02), those with CHD (P < .0001), those with non‐private insurance (P < .0001), and those with longer hospital stay (P = .001) were more likely to be in a modified education placement at time of listing. Age, race, listing status, mechanical support, and waitlist time were not significantly associated with placement. The prevalence of typical education placement was similar (87% at 1‐year and 86% at 3‐year) post‐transplant. Predictors of modified education placement at 3‐year follow‐up included placement at listing (OR = 12.9 [95% CI 7.6‐21.9], P < .0001), non‐private insurance (OR = 2.0 [95% CI 1.3‐3.2], P = .001), CHD (OR = 1.8 [95% CI 1.1‐2.7, P = .01), history of post‐transplant infection (OR = 1.9 [95% CI 1.2‐2.9, P = .007), and number of post‐transplant infections (OR = 1.3 [95% CI 1.1‐1.5, P = .002). Among pediatric heart transplant recipients, males, those with non‐private insurance, those with CHD, and those who experience post‐transplant infections are at greatest risk for modified academic placement, which persists for several years post‐transplant and deserves targeted intervention.     

Early childhood development and stunting: Findings from the MAL‐ED birth cohort study in Bangladesh

Information on the association between stunting and child development is limited from low‐income settings including Bangladesh where 36% of children under‐ 5 are stunted. This study aimed to explore differences in early childhood development (ECD) between stunted (length‐for‐age z‐score [LAZ] < ?2) and nonstunted (LAZ ≥ ?2) children in Bangladesh. Children (n = 265) aged 6–24 months who participated in the MAL‐ED birth cohort study were evaluated by trained psychologists at 6, 15, and 24 months of age using the Bayley Scales of Infant and Toddler Development‐III; child length and weight were measured using standard procedures. ECD scores (z‐scores derived from cognitive, motor, language and socio‐emotional skills) were compared between stunted, underweight (weight‐for‐age z‐score < ?2), and wasted (weight‐for‐length z‐score < ?2) children, controlling for child age and sex and maternal age, education, body mass index (BMI), and depressive symptoms. Stunted children had significantly lower ECD scores than their nonstunted peers on cognitive (P = .049), motor (P < .001), language (P < .001) and social–emotional (P = .038) scales where boys had significantly lower fine motor skills compared with girls (P = .027). Mother's schooling and BMI were significant predictors of ECD. Similar to stunting, underweight children had developmental deficits in all domains (cognitive: P = .001; fine motor: P = .039, and P < .001 for both gross motor and total motor; expressive communication: P = .032; total language: P = .013; social–emotional development: P = .017). Wasted children had poor motor skills (P = .006 for the fine motor; P < .001 for both gross motor and total motor development) compared with the nonwasted peers. Early childhood stunting and underweight were associated with poor developmental outcomes in Bangladesh.     

Side effects and efficacy of renal sparing immunosuppression in pediatric liver transplantation—A single center matched cohort study

Immunosuppressive combination therapy with MMF can reduce CNI associated nephrotoxicity. We investigated effectiveness and safety of de novo MMF‐tacrolimus based immunosuppression after pLTx. Patients after pLTx receiving immunosuppression with MMF/tacrolimus (MMF/TAC) were compared to retrospectively selected age‐ and diagnosis‐matched patients with tacrolimus monotherapy (TAC) and cyclosporine/prednisolone therapy (CSA) (19 patients each, n = 57). Effectiveness, renal function and side effects were analyzed for 1 year after pLTx. Tacrolimus reduction in combination therapy (0.7 μg/L over the year) was lower than aspired (2 μg/L). Acute BPAR occurred equally in MMF/TAC and TAC groups (31.6% each), being slightly higher in CSA group (42.1%; OR = 1.5; 95% CI = 0.42‐5.44; P = .5). GFR deteriorated comparably in all 3 groups (P < .01 each) without significant differences between the groups. Septicemia was detected significantly more often in MMF/TAC (73.6%) than in TAC (31.6%) (OR 4.17; 1.07‐16.27; P = .04). EBV reactivation occurred more often in CSA patients (84.2%) than in MMF/TAC (47.4%; OR 5.16; 0.98‐27.19; P = .05) and TAC patients (52.6%; OR 8.16; 1.48‐44.89; P = .02) the same was true for other viral infections (47.4% (CSA) vs 15.8% (TAC); OR 4.21; 0.95‐18.55; P = .05). Our study does not provide additional evidence for a benefit of initial use of MMF/TAC over TAC regarding renal function, but raises concerns regarding a potentially increased risk of serious infections under MMF/TAC compared to TAC monotherapy at equivalent renal outcome; our study is, however, limited by the minor CNI reduction in combination therapy.     

Dose‐escalation is needed for gross disease in high‐risk neuroblastoma

1 Background

Locoregional failure is common after subtotal resection in high‐risk neuroblastoma. Although a dose of 21 Gy radiation therapy (RT) is standard for treatment of high‐risk neuroblastoma after gross total resection, the dose needed for local control of patients with gross residual disease at the time of RT is unknown. We sought to evaluate local control after 21–36 Gy RT in patients with high‐risk neuroblastoma undergoing subtotal resection.

2 Methods

All patients with high‐risk neuroblastoma who received RT to their primary site from 2000 to 2016 were reviewed. Of the 331 patients who received consolidative RT to their primary site, 19 (5.7%) underwent subtotal resection and were included in our analysis. Local failure (LF) was correlated with biologic prognostic factors and dose of RT.

3 Results

Median follow‐up among surviving patients was 6.0 years. Median RT dose was 25 Gy (range, 21 Gy–36 Gy). The 5‐year cumulative incidence of LF among all patients was 17.2%. LF at 5 years was 30% in those who received <30 Gy versus 0% in those who received 30–36 Gy (P = 0.12). There was a trend towards improved local control in patients with tumor size ≤10 cm at diagnosis (P = 0.12). The 5‐year event‐free and overall survival were 44.9% and 68.7%, respectively.

4 Conclusion

After subtotal resection, patients who received less than 30 Gy had poor local control. Doses of 30–36 Gy are likely needed for optimal control of gross residual disease at the time of consolidative RT in high‐risk neuroblastoma.     

Facial deformation following treatment for pediatric head and neck rhabdomyosarcoma; the difference between treatment modalities. Results of a trans-Atlantic,multicenter cross-sectional cohort study

Background

The four different local therapy strategies used for head and neck rhabdomyosarcoma (HNRMS) include proton therapy (PT), photon therapy (RT), surgery with radiotherapy (Paris-method), and surgery with brachytherapy (AMORE). Local control and survival is comparable; however, the impact of these different treatments on facial deformation is still poorly understood. This study aims to quantify facial deformation and investigates the differences in facial deformation between treatment modalities.

Methods

Across four European and North American institutions, HNRMS survivors treated between 1990 and 2017, more than 2 years post treatment, had a 3D photograph taken. Using dense surface modeling, we computed facial signatures for each survivor to show facial deformation relative to 35 age–sex–ethnicity-matched controls. Additionally, we computed individual facial asymmetry.

Findings

A total of 173 HNRMS survivors were included, survivors showed significantly reduced facial growth (p < .001) compared to healthy controls. Partitioned by tumor site, there was reduced facial growth in survivors with nonparameningeal primaries (p = .002), and parameningeal primaries (p ≤.001), but not for orbital primaries (p = .080) All patients were significantly more asymmetric than healthy controls, independent of treatment modality (p ≤ .001). There was significantly more facial deformation in orbital patients when comparing RT to AMORE (p = .046). In survivors with a parameningeal tumor, there was significantly less facial deformation in PT when compared to RT (p = .009) and Paris-method (p = .007).

Interpretation

When selecting optimal treatment, musculoskeletal facial outcomes are an expected difference between treatment options. These anticipated differences are currently based on clinicians’ bias, expertise, and experience. These data supplement clinician judgment with an objective analysis highlighting the impact of patient age and tumor site between existing treatment options.     

The efficacy of serum brain natriuretic peptide for the early detection of portopulmonary hypertension in biliary atresia patients before liver transplantation

Severe portopulmonary hypertension (POPH) is a contraindication for liver transplantation (LT) because of the high risk of postoperative heart failure. The early detection of POPH is important for patients with biliary atresia (BA). Brain natriuretic peptide (BNP) is known to be correlated with liver fibrosis in patients with liver cirrhosis. The aim of this study was to elucidate the efficacy of BNP measurement for the follow‐up of patients with BA. Thirty‐two patients with BA were identified from September 2011 to December 2016. As indices of liver fibrosis/cirrhosis, APRI (P < .0001), FIB‐4 (P < .0001), Child‐Pugh score (P < .0001), IV collagen (P = .0005), and hyaluronic acid (P = .0291) had high or moderate correlations with BNP. Patients with splenomegaly, esophageal varices, liver fibrosis, and collateral veins had significantly higher BNP levels than those without. Patients diagnosed with POPH had significantly higher BNP levels in comparison with those patients without (P = .0068). In contrast, PELD/MELD scores showed an almost negligible correlation with the BNP level. LT was successful in 3 asymptomatic BA patients with POPH who had high BNP levels despite the low PELD/MELD scores. In conclusion, routine serum BNP surveillance can be easy to predict asymptomatic POPH. This may help to identify POPH before it reaches a stage that would contraindicate LT.     

Early predictors of mortality in children with pulmonary complications after haematopoietic stem cell transplantation

PC are a main cause of death following HSCT in children. We aimed to evaluate early predictors of mortality in paediatric recipients with PCs. A retrospective observational study of 35 patients with 49 episodes of PI on chest radiography (of 124 patients) who had undergone HSCT at a tertiary university hospital between January 2011 and December 2012 was performed. During follow‐up (median 26.1 months), 15 episodes led to death (30.6%). An aetiologic diagnosis was made by non‐invasive tests in 24 episodes (49.0%) and by adding bronchoalveolar lavage and/or lung biopsy in 7 episodes with diagnostic yield (77.8%, P = .001). Thus, a specific diagnosis was obtained in 63.3% of the episodes. Aetiology identification and treatment modification after diagnosis did not decrease mortality (P = .057, P = .481). However, the number of organ dysfunctions at the beginning of PI was higher in the mortality group, compared to the survivor group (1.7 ± 1.2 vs 0.32 ± 0.59; P = .001). Hepatic dysfunction (OR, 11.145; 95% CI, 1.23 to 101.29; P = .032) and neutropaenia (OR, 10.558; 95% CI, 1.07 to 104.65; P = .044) were independently associated with risk of mortality. Therefore, hepatic dysfunction and neutropaenia are independent early predictors of mortality in HSCT recipients with PCs.     

Trends in kidney transplant outcomes in children and young adults with cystinosis

Temporal changes in kidney transplant outcomes for cystinosis are unknown. We used the SRTR to identify all kidney transplants performed for cystinosis in patients younger than 31 years between 1987 and 2017. We divided time into three equal eras (1987‐1997, 1998‐2007, and 2008‐2017) to assess changes in outcomes using Cox proportional and linear regression models. We examined 441 transplants in 362 patients. Age at ESRD progressively increased (12.1 vs 13.3 vs 13.4; P = .046). Eras 2 and 3 had lower risk of acute rejection (aHR 2 vs 1:0.45; P < .001) (aHR 3 vs 1:0.26; P < .001) and higher 5‐year mean GFR (difference 2 vs 1:9.2 mL/min/1.73 m²; P = .005) (difference 3 vs 1:12.9 mL/min/1.73 m²; P = .002) compared with era 1. Five‐year graft survival was similar across eras, but 5‐year patient survival was higher for era 2 (aHR: 0.25; P = .01). Seventy‐nine patients underwent retransplantation. Five‐year patient (94.2% vs 92.5%; P = .57) and graft survival (79.1% vs 74.1%; P = .52) were similar between primary and subsequent transplants. Age at ESRD, acute rejection, GFR at 5 years, and patient survival improved over time. Kidney retransplantation is associated with excellent outcomes in children and young adults with cystinosis.     

Associations between neutrophil-to-lymphocyte ratio and platelet-to-lymphocyte ratio and prognosis in patients with neuroblastoma

Objectives

Recent studies have shown that the neutrophil-to-lymphocyte ratio (NLR) is a new inflammatory marker that is effective in determining the prognosis of many solid tumors, chemotherapy responses, survival, and their recurrence rate. Therefore, we performed a retrospective study to investigate the effect of neutrophil-to-lymphocyte and platelet-to-lymphocyte ratio (PLR) on risk factors and prognosis in these patients.

Materials and methods

In this study, 246 pediatric patients with neuroblastoma who were diagnosed, treated, and followed up during 2000–2021 in Division of Pediatric Oncology, Çukurova University Faculty of Medicine, were included. Required information of patients was obtained from archive files, Mergentech hospital program, and E-pulse system.

Results

Median value for NLR was found to be 1.06, for PLR it was found as 92. The relationship of NLR values with age, stage, risk group, and Shimada was found to be statistically signifıcant with p < .001, vanillylmandelic acid (VMA) (p = .006) also depicted the signifıcant value. Likewise, the relationship of PLR values with age (p < .001), stage (p = .022), Shimada (p = .004), and N-Myc amplification (p = .039) was found to be statistically significant as well. Survival analysis showed that no statistically significant difference was observed among the higher and lower values of NLR. Survival rates were noticed to be higher in the lower values of NLR (10-year overall survival [OS] 55% vs. 49%, 10-year event-free survival (EFS) 54% vs. 43%), albeit nonsignificant.

Conclusion

Pretreatment evaluation of NLR and PLR values in patients with neuroblastoma may be instructive in respect of prognosis and risk group.     

Increase in atopic sensitization rate among Dutch children with symptoms of allergic disease between 1994 and 2014

Background

The prevalence of symptoms of allergic diseases has increased significantly during the last decades. However, studies into time trends of atopic sensitization among children are limited and have focused on aeroallergen sensitization. We aimed to investigate time trends in the prevalence and degree of atopic sensitization to inhalant and food allergens among children (0‐17 years) with symptoms of allergic disease.

Methods

Sensitization data of all children tested in our clinical laboratory during 1994‐2014 were analyzed. Sensitization was detected using the ImmunoCAP system and defined as a specific IgE level of ≥0.35 kU/L. Trends in sensitization rates to 5 food and 5 aeroallergens for different age categories were investigated with logistic regression, adjusted for age and sex.

Results

Sensitization data of 18 199 children were analyzed. Between 1994 and 2014, a steady and statistically significant increase in overall sensitization rate was found (from 40.5% in 1994 to 48.9% in 2014, adjusted odds ratio [aOR] 1.01 per year, 95% confidence interval [CI] 1.00 to 1.01, P = .003). This increase in sensitization rate was mainly explained by increasing aeroallergen sensitization among 4‐ to 11‐year‐old children (aOR 1.02, 95% CI 1.01 to 1.02, P < .001). We found no increase in sensitization rates to food and aeroallergens in other age categories. The degree of sensitization did not change significantly during the study period (all tests P > .15).

Conclusion

We observed a statistically significant increase in sensitization rate between 1994 and 2014 among children with symptoms of allergic disease. This was mainly explained by increasing aeroallergen sensitization among 4‐ to 11‐year‐olds.     

Disparities in household material hardship,financial toxicity,and income loss in pediatric cancer

Background

Based on previous reports of disparities in financial burden following a cancer diagnosis, this study aims to characterize mechanisms of disparities experienced by caregivers of children with cancer, including the impact of work flexibility and social support.

Methods

Cross-sectional survey (in English or Spanish) of caregivers of children with cancer that assessed household material hardship (HMH), financial toxicity, and income change.

Results

Of 156 caregivers surveyed, 32% were Hispanic and 32% were low income. Hispanic caregivers were more likely to report HMH and financial toxicity compared to non-Hispanic White and Asian (HMH: 57% vs. 21% vs. 19%, p < .001; financial toxicity: 73% vs. 52% vs. 53%, p = .07). Low- and middle-income caregivers were more likely to experience HMH and financial toxicity compared to high-income caregivers (HMH: 68% low vs. 38% middle vs. 8.7% high, p < .001; financial toxicity: 81% vs. 68% vs. 44%, p < .001). All income categories demonstrated significant increases in HMH 1 year after diagnosis. Seventeen percent reported more than 40% income loss, more of whom were low income than high income (27% vs. 12%, p = .20). Work flexibility and social support were associated with income and financial toxicity.

Conclusion

HMH, financial toxicity, and income loss are prevalent after a child's cancer diagnosis, suggesting that screening should be incorporated into routine care. This financial burden disproportionately affects low-income and Hispanic caregivers. Further research is needed to elucidate the roles of work flexibility and social support, how safety net services are utilized by families, and how best to support families with HMH.     

The effect of subclinical infantile thiamine deficiency on motor function in preschool children

We investigated the long‐term implications of infantile thiamine (vitamin B1) deficiency on motor function in preschoolers who had been fed during the first 2 years of life with a faulty milk substitute. In this retrospective cohort study, 39 children aged 5–6 years who had been exposed to a thiamine‐deficient formula during infancy were compared with 30 age‐matched healthy children with unremarkable infant nutritional history. The motor function of the participants was evaluated with The Movement Assessment Battery for Children (M‐ABC) and the Zuk Assessment. Both evaluation tools revealed statistically significant differences between the exposed and unexposed groups for gross and fine motor development (p < .001, ball skills p = .01) and grapho‐motor development (p = .004). The differences were especially noteworthy on M‐ABC testing for balance control functioning (p < .001, OR 5.4; 95% CI 3.4–7.4) and fine motor skills (p < .001, OR 3.2; 95% CI 1.8–4.6). In the exposed group, both assessments concurred on the high rate of children exhibiting motor function difficulties in comparison to unexposed group (M‐ABC: 56% vs. 10%, Zuk Assessment: 59% vs. 3%, p < .001). Thiamine deficiency in infancy has long‐term implications on gross and fine motor function and balance skills in childhood, thiamine having a crucial role in normal motor development. The study emphasizes the importance of proper infant feeding and regulatory control of breast milk substitutes.     

Outcomes of liver transplantation in pediatric recipients with cardiovascular disease

LT exerts considerable stress on the heart perioperatively. Limited data exist on impact of cardiovascular diseases on LT children. This study evaluated the outcomes of children with CVD who underwent LT and compared with pretransplant findings. From 518 LT recipients, 82 (15.8%) had CVD. Sixty patients were classified as low‐risk adjustment for congenital heart surgery 1 (RACHS 1 and 2). Five patients were classified as RACHS ≥3. The most common echocardiographic finding in the CVD patients (25/82) was ASD. CVD patients had more abnormal EKG (32.4% vs 14.5%, P < .001), abnormal chest X‐ray (11.8% vs 1.4%, P < .001), and altered echocardiography (89.7% vs 15.4%, P < .001) findings compared with the No‐CVD group pretransplant. Post‐transplant, significant differences between groups were observed related to abnormal EKG (14.7% vs 7.0%, P = .03) and echocardiography (48.5% vs 3.2%, P < .01) findings. Pretransplant ASD spontaneously closed in 22 patients. At 1 and 5 years post‐transplant, there was no difference in the survival rate between groups (P = .96). The prevalence of CVD in recipients of LT was high, and its presence was associated with significantly higher cardiac decompensation before and after LT. Minor and moderate cardiovascular disease did not impact the long‐term survival.     

Renal transplantation in children under 3 years of age: Experience from a single‐center study

RTx remains challenging in children under 3 years of age. This single‐center study reviewed the medical records of children <3 years transplanted since 1987 (N = 32, Group 1). They were matched for donor type and RTx period with children aged 3‐13 years (N = 32, Group 2) and 13‐18 years (N = 32, Group 3). There were no between‐group significant differences regarding distributions of gender, primary renal disease, proportion of dialysis before RTx, and growth (SDS). Compared to Groups 2 and 3, Group 1 had more peritoneal dialyses (P < .001), more EBV mismatches (P = .04), and longer warm ischemia times (P < .001). The risk of graft loss was not significantly different among age groups (hazard ratio, 2.4 in Group 2 and 2.0 in Group 3 vs Group 1; P = .2). Death occurred in four patients (3 in Group 1 and 1 in Group 2) and graft loss occurred in 28 patients, mainly due to chronic allograft nephropathy. In recipients <3 years of age, the outcomes of RTx are close to those obtained in older pediatric age groups. Thus, young patients may be transplanted in experienced multidisciplinary teams without additional risks provided that particular attention is paid to donor selection and prevention/early diagnosis of comorbidities and complications.     

Socioeconomic status significantly impacts childhood cancer survival in South Africa

Background and aims

Significantly discrepant survival rates have been documented in single disease childhood cancer cohorts in South Africa; those from higher socioeconomic groups were shown to have a significantly lower risk of death than those from less affluent households. This study aimed to determine the impact of socioeconomic status (SES) on childhood cancer survival using pooled South African data.

Methods

Five databases spanning January 2000 to December 2021 were interrogated. SES status was assigned based on a public sector annual household income classification. H0 households (formally unemployed) received free healthcare. H1, H2 and H3 (annual income > United States Dollar [USD] 19,000) households paid for healthcare relative to their income. The Spearman test assessed correlations between SES and disease stage in patients with solid tumours. Hazard ratios were determined using Cox regression modelling. The Kaplan–Meier procedure estimated overall survival (OS).

Results

A total of 1598 children were eligible for analysis; 1269 had a solid tumour with a negative correlation between SES and stage (Spearman rho = −.178; p < .001). Patients with solid tumours and lower SES showed proportionately higher numbers of stage III and IV disease (p < .01). This proportion decreased with higher SES categories. In the multivariate analyses adjusted for sex, age, tumour type and stage, higher SES was associated with lower mortality risk (p < .001), indicating that the impact of SES on survival was in excess of any effect that could be explained by lower stage disease alone. There was a strong positive correlation between race and SES (Fisher's exact tests, p < .001) across all groups and all SES strata. Five-year OS was 85.3% in children from H3 households versus 46.3% in children from H0 households (p < .001).

Conclusion

SES significantly impacts childhood cancer survival for children with solid tumours in South Africa. SES is a robust surrogate for race in South Africa as a prognostic metric of disease outcome in childhood cancer. Advocacy to increase social support for impoverished patients is essential to achieve equitable improvements in outcomes treated with standardised national treatment guidelines.     

First liver transplantation for biliary atresia in children: The hidden effects of non‐centralization

The aim of our study was to determine the impact of initial orientation for medical and surgical care of children with BA on procedures and outcomes of the first LT. We retrospectively analyzed charts of children with BA who underwent first LT between 2006 and 2015. Patients were divided into two groups for comparison: a single‐center management group (from diagnosis to transplantation) and a secondarily referred group (children referred after failure of KP). We focused analysis on disease severity at transplantation, blood transfusion, and overall survival. One hundred and eighty‐five children were included. The median delay between pretransplant check‐up and transplantation was shorter in patients secondarily referred. A severe undernutrition was observed in 23.7% of children secondarily referred compared to 11.1% in children with a single‐center management (P = .024). At transplantation, INR and factor V level were higher in single‐center group patients (respectively, 67% vs 55%, P < .001 and 61% vs 49%, P = .002). The total of red blood cell and fresh frozen plasma administrated during procedure was two times higher in patients secondarily referred. Finally, patients with a single‐center management had a higher overall 12 months of survival rate (92.1% vs 83.1%, P = .033). In a country without low‐density population issues, the authors advocate an early referring to transplant center to further improving LT outcomes in children with BA.     

Nocturnal hypertension and left ventricular hypertrophy in pediatric renal transplant recipients in South India

HTN after renal transplantation is associated with cardiovascular morbidity. ABPM allows diagnosis of masked HTN and isolated nocturnal HTN. Longitudinal ABPM data in children post‐transplant are limited. ABPM was performed in children post‐transplant and repeated in 6‐12 months. BP indices were used to determine the prevalence of masked HTN, masked uncontrolled HTN (masked HTN in patients on antihypertensive medications), and isolated nocturnal HTN. Linear regression determined the association between LVMI and ABPM indices. Thirty children underwent a baseline ABPM. Ambulatory HTN was present in 25 (83%). Masked HTN was present in 18 (60%) and isolated nocturnal HTN in 13 (43%). Nocturnal ambulatory BP was higher than corresponding daytime BPs (P < .001 for systolic and diastolic) and 25 (83%) had a blunted nocturnal dip. Prednisone dose predicted nocturnal DBP index and DBP load (r² = .40, P = .024 and r² = .178, P = .02). ABPM was repeated in 18 patients within 11 (±3) months. BP indices decreased with time, but nocturnal BPs remained higher than daytime (P < .001 for SBP and DBP). Blunted nocturnal dip did not improve. LVH was present in 12 (57%). LVMI was directly related to the nocturnal SBP index (r² = .377, P = .003) and nocturnal DBP index (r² = .493, P < .001). We found no association between LVMI and daytime BP indices. The prevalence of masked HTN, isolated nocturnal HTN, and blunted nocturnal dip was high in children with kidney transplants. Nocturnal BP predicted LVMI. Ambulatory BP improved on longitudinal follow‐up, but the pattern of isolated nocturnal HTN persisted.     

Hydroxyurea therapy in UK children with sickle cell anaemia: A single‐centre experience

1 Introduction

Despite the demonstrated efficacy of hydroxyurea therapy, children with sickle cell anaemia in the UK are preferentially managed with supportive care or transfusion. Hydroxyurea is reserved for children with severe disease phenotype. This is in contrast to North America and other countries where hydroxyurea is widely used for children of all clinical phenotypes. The conservative UK practice may in part be due to concerns about toxicity, in particular marrow suppression with high doses, and growth in children.

2 Methods and results

We monitored 37 paediatric patients with sickle cell anaemia who were treated with hydroxyurea at a single UK treatment centre. Therapy was well tolerated and mild transient cytopenias were the only toxicity observed. Comparative analysis of patients receiving ≥26 mg/kg/day versus <26 mg/kg/day demonstrates increasing dose has a significant positive effect on foetal haemoglobin (Hb; 29.2% vs. 20.4%, P = 0.0151), mean cell volume (94.4 vs. 86.5, P = 0.0183) and reticulocyte count (99.66 × 10⁹/l vs. 164.3 × 10⁹/l, P = 0.0059). Marrow suppression was not a clinical problem with high‐dose treatment, Hb 92.25 g/l versus 91.81 g/l (ns), neutrophil count 3.3 × 10⁹/l versus 4.8 × 10⁹/l (ns) and platelet count 232.4 × 10⁹/l versus 302.2 × 10⁹/l (ns). Normal growth rates were maintained in all children. Good adherence to therapy was a significant factor in reducing hospitalisations.

3 Conclusion

This study demonstrates the effectiveness and safety in practice of high‐dose hydroxyurea as a disease‐modifying therapy, which we advocate for all children with sickle cell anaemia.