Acute chest syndrome in children with sickle cell disease: Data from a national AIEOP cohort identify priority areas of intervention in a hub-and-spoke system.

Acute chest syndrome (ACS) is a frequent cause of hospitalization in sickle cell disease (SCD). Despite advances in acute care, many settings still lack knowledge about ACS best practices. After the AIEOP Guidelines were published in 2012, suggesting standardized management in Italy, a retrospective study was performed to assess the diagnostic and therapeutic pathways of ACS in children. From 2013 to 2018, 208 ACS episodes were presented by 122/583 kids in 11 centres. 73 were male, mean age 10.9 years, 85% African, 92% HbSS or Sß°. In our hub-and-spoke system, a good adherence to Guidelines was documented, but discrepancies between reference centres and general hospitals were noted. Improvement is needed for timely transfer to reference centres, use of incentive spirometry, oxygen therapy and pain management.

Contamination of drinking water sources during the rainy season in an urban post-conflict community in Guinea Bissau: implications for sanitation priority.

Since the 1998 civil warcholera outbreaks and waterborne infections have been a major cause of morbidity and mortality during the rainy season in Guinea Bidsau. Our survey aims at: (1) describing the distribution, characteristics and use of water sources and sewage facilities in a central area of the capital city of Bissau; (2) determining the microbiological quality of drinking water during the rainy season. After mapping of the Cuntum 3 study area, water sources' and latrines' location, characteristics and use were determined by visual inspection and interviews with householders. Microbiological analyses were peformed from water sources for evaluation of total Coliforms, E. coli, Enterococcus faecalis. Twelve water sources (9 wells, 3 taps) and 15 latrines were identified and used by 444 inhabitants. Water sources and latrines were at less than 5 meters distance apart. Wells were self-built, hand-dug, shallow (4-6 meters), unprotected. Taps were located outdoor. Latrines were self-built, open air, unprotected. None of the houses had a bathroom. Maintenance of wells, taps and latrines is not performed on regular basis and well's handling habits are not safe. Well and tap water showed heavy faecal contamination with more than 1000 CFU/100 ml. The contamination of drinking water in Bissau due to poor construction, maintenance and improperuse ten years after the civil war, demonstrates the need to allocate resources after conflicts in the area of water and sanitation. Both should be included as a priority in post-conflict reconstruction programs in order to reduce cholera outbreaks and diarrhoea related mortality.

Feasibility and Effectiveness of Tuberculosis Active Case-Finding among Children Living with Tuberculosis Relatives: a Cross-Sectional Study in Guinea-Bissau.

BACKGROUND AND OBJECTIVES: The World Health Organization End tuberculosis (TB) Strategy, approved in 2014, aims at a 90% reduction in TB deaths and an 80% reduction in TB incidence rate by 2030. One of the suggested interventions is the systematic screening of people with suspected TB, belonging to specific risk groups. The Hospital Raoul Follereau (HRF) in Bissau, Guinea-Bissau, is the National Reference Hospital for Tuberculosis and Lung Disease of the country. We performed an active case-finding program among pediatric age family members and cohabitants of admitted adult TB patients, from January to December 2013. METHODS: Newly admitted adult patients with a diagnosis of TB were invited to bring their family members or cohabitants in childhood age for clinical evaluation in a dedicated outpatient setting within the hospital compound. All the children brought to our attention underwent a medical examination and chest x-ray. In children with clinical and/or radiologic finding consistent with pulmonary TB, a sputum-smear was requested. RESULTS: All admitted adult patients accepted to bring their children cohabitants. In total, 287 children were examined in 2013. Forty-four patients (15%) were diagnosed with TB. The number needed to screen (NNS) to detect one case of TB was 7. 35 patients (80%) had pulmonary TB; 2 of them were sputum smear-positive. No adjunctive personnel cost was necessary for the intervention. CONCLUSIONS: A hospital-based TB active case-finding program targeted to high-risk groups like children households of severely ill admitted patients with TB can successfully be implemented in a country with limited resources.

European intergroup studies (MMT4-89 and MMT4-91) on childhood metastatic rhabdomyosarcoma: final results and analysis of prognostic factors.

PURPOSE: Final results are presented from two consecutive European studies for patients with metastatic rhabdomyosarcoma (RMS) to identify prognostic variables and determine the value of high-dose chemotherapy (HDCT) in complete remission. PATIENTS AND METHODS: A total of 174 patients aged 3 months to 18 years participated. From 1989 to 1991, patients received four cycles of intensive multiagent chemotherapy. From 1991 to 1995, patients achieving complete remission received consolidation with HDCT. All received local therapy (surgery, radiation therapy) according to response. RESULTS: At a median follow-up of 8 years, 5-year overall survival (OS) and event-free survival (EFS) for the whole group were 24% and 20%, respectively. No statistical difference was found between HDCT and standard chemotherapy (5-year OS, 36% v 27%; EFS 29% v 23%). Univariate analysis identified primary tumor in parameningeal, extremity, or other sites; age younger than 1 year and older than 10 years; bone or bone marrow metastases; multiple metastases; and multiple sites of metastases as unfavorable prognostic factors for OS and EFS. Multivariate analysis identified unfavorable site, bone or bone marrow involvement, and unfavorable age as independently unfavorable factors. Two subgroups were identified. Those with fewer than two unfavorable factors had 5-year EFS and OS of 40% and 47%, respectively. Patients with > or = two unfavorable factors had 5-year EFS and OS of 7.5% and 9%, respectively. CONCLUSION: A minority of patients with metastatic RMS have better survival than overall results for this population suggest. Those in the highest risk group have such poor survival that they are candidates for first-line novel therapies. There is no evidence that consolidation with HDCT improves outcome.

High-dose melphalan with autologous stem-cell rescue in metastatic rhabdomyosarcoma.

PURPOSE: The European Collaborative MMT4-91 trial was conducted as a prospective nonrandomized study to evaluate the potential benefit of high-dose melphalan as consolidation of first complete remission in children with stage IV rhabdomyosarcoma. PATIENTS AND METHODS: Fifty-two patients in complete remission after six courses of chemotherapy received "megatherapy": 42 received melphalan alone, whereas 10 received melphalan in combination with etoposide, carboplatin/etoposide, or thiotepa/busulfan and etoposide. The outcome of this group of patients was compared with that observed in 44 patients who were also in complete remission after six courses of identical chemotherapy (plus surgery or radiotherapy) but went on to receive a total of up to 12 courses of conventional chemotherapy (four cycles). No differences were found between the two groups regarding clinical characteristics, chemotherapy received before complete remission, or response to chemotherapy. In particular, there was no significant difference between the groups for site of primary tumor, histologic subtype, age at presentation, presence of bone or bone marrow metastases, or number of metastases. RESULTS: The 3-year event-free survival (EFS) and overall survival (OS) rates were 29.7% and 40%, respectively, for those receiving high-dose melphalan or other multiagent high-dose regimens and 19.2% and 27.7%, respectively, for those receiving standard chemotherapy. The difference was not statistically significant (P =.3 and P =.2 for EFS and OS, respectively). There was a significant prolongation in the time from the last day of high-dose chemotherapy or the end of chemotherapy cycle 4 to the time of relapse in those receiving megatherapy (168 days for patients receiving megatherapy v 104 days for those receiving standard therapy; P =.05). CONCLUSION: The addition of a high-dose alkylating agent to consolidation therapy may have prolonged progression-free survival in this poor-risk patient group, but it did not significantly improve the ultimate outcome.

Intensive chemotherapy for children and young adults with metastatic primitive neuroectodermal tumors of the soft tissue.

The MMT4 study was designed to explore an intensive chemotherapy regimen (MMT4-89) and the role of high-dose melphalan (MMT4-91) in children with metastatic soft tissue sarcoma, including extraosseous peripheral neuroectodermal tumor (PNET). Thirty-one patients with PNET were treated between 1989 and 1995 (11 according to MMT4-89 and 20 according to MMT4-91). Chemotherapy consisted of four CEVAIE cycles, each including three 3-week courses: CEV (carboplatin 500 mg/m(2), epirubicin 150 mg/m(2), vincristine 1.5 mg/m(2)), IVA ifosfamide 9 g/m(2), actinomycin 1.5 mg/m(2), vincristine 1.5 mg/m(2)), IVE (ifosfamide 9 g/m(2), etoposide 600 mg/m(2), vincristine 1.5 mg/m(2)). In MMT4-91 the fourth CEVAIE was replaced with melphalan 200 mg/m(2) with stem cell rescue. The CEV combination was evaluated as a window study. Surgery followed the second cycle. Radiotherapy was administered to post-surgical residual disease. The response rate was 55% after CEV, rising to 80% after the first CEVAIE. Twenty-five patients achieved complete remission (CR). Overall, the 5-year EFS was 22.6%: 36.4% and 15% for patients treated according to MMT4-89 and MMT4-91, respectively (P = 0.3). Local control was achieved in 77% of irradiated patients vs 45% of non-irradiated. Age >10 years was associated with significantly poorer outcome (P = 0.04). In conclusion, despite the high CR rate, intensive chemotherapy with or without high-dose melphalan appeared to have little impact on the survival of patients with metastatic extraosseus PNET.