Diagnosis and management of acquired aplastic anemia in childhood. Guidelines from the Marrow Failure Study Group of the Pediatric Haemato-Oncology Italian Association (AIEOP).

Acquired aplastic anemia (AA) is a rare heterogeneous disorder characterized by pancytopenia and hypoplastic bone marrow. The incidence is 2-3 per million population per year in the Western world, but 3 times higher in East Asia. Survival in severe aplastic anemia (SAA) has improved significantly due to advances in hematopoietic stem cell transplantation (HSCT), immunosuppressive therapy, biologic agents, and supportive care. In SAA, HSCT from a matched sibling donor (MSD) is the first-line treatment. If a MSD is not available, options include immunosuppressive therapy (IST), matched unrelated donor, or haploidentical HSCT. The purpose of this guideline is to provide health care professionals with clear guidance on the diagnosis and management of pediatric patients with AA. A preliminary evidence-based document prepared by a group of pediatric hematologists of the Bone Marrow Failure Study Group of the Italian Association of Pediatric Hemato-Oncology (AIEOP) was discussed, modified and approved during a series of consensus conferences that started online during COVID 19 and continued in the following years, according to procedures previously validated by the AIEOP Board of Directors.

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.

Endocrine and metabolic complications in children and adolescents with Sickle Cell Disease: an Italian cohort study.

BACKGROUND: Children with Sickle Cell Disease (SCD) show endocrine complications and metabolic alterations. The physiopathology of these conditions is not completely understood: iron overload due to chronic transfusions, ischemic damage, and inflammatory state related to vaso-occlusive crises may be involved. Aims of this study were to evaluate the growth pattern, endocrine complications, and metabolic alterations and to detect the relationship between these conditions and the SCD severity in affected children and adolescents. METHODS: Fifty-two children and adolescents with SCD [38 homozygous sickle hemoglobin (HbSS) and 14 heterozygous sickle hemoglobin (HbSC); age range 3-18 years] were recruited. Anthropometric [height, body mass index (BMI), arm span, sitting height, target height (TH), and pubertal status] and laboratory [blood cell counts, hemolysis indices, metabolic and nutritional status indices and hormonal blood levels] data were evaluated. The SCD severity was defined according to hematological and clinical parameters. RESULTS: Height-SDS adjusted for TH and BMI-SDS were significantly higher in HbSC children than in HbSS ones. Forty-eight out of 52 patients (92%) had at least one metabolic and/or endocrine alteration: insufficiency/deficiency of vitamin D (84.7%), insulin resistance (11.5%), growth hormone deficiency (3.8%), subclinical hypothyroidism (3.8%), and hypogonadism (1.9%). Levels of vitamin D were significantly and negatively correlated with clinical indicators of the SCD severity. Subjects with HbSS genotype show significant lower levels of both insulin-like growth factor-1 (IGF-1) and insulin-like growth factor binding protein 3 than children with HbSC. In the study population IGF-1 values were significantly and positively correlated with Hb and negatively with lactate dehydrogenase. CONCLUSIONS: Metabolic alterations and endocrine complications are very common in children and adolescents with SCD. A regular follow-up is necessary to identify subjects at risk for complications to precociously start an appropriate treatment and to improve the quality of life of SCD patients.

[Organizational appropriateness of recoveries: results of the evaluation of recoveries by AEP/PRUO at the Careggi Hospital in Florence in 1995-1998]. / Appropriatezza organizzativa dei ricoveri: risultati della valutazione con AEP/PRUO dei ricoveri dell'AO Careggi di Firenze. Anni 1995 e 1998.

The organizational appropriateness of hospital stays is an expression of a hospital's efficiency and aim of the management control. The AEP (American Version)/PRUO (Italian Version) protocol is specific for measuring the organizational appropriateness of hospital stays. The aim of this study is the comparative analysis of the organizational appropriateness of hospital stays in Careggi Hospital of Florence and in all hospital Departments in 1995 and 1998. In 1998 the AEP/PRUO protocol was applied to 2148 samples of hospital stays out of 38,968 eligible hospitalizations, and in 1995 a sample of 1989 hospital stays out of 35,108. In 1998 2,148 admissions and 15,338 days of hospitalization were tested. In 1995, 1989 admission days and 12,264 days of hospitalization were tested, 63 departments were studied. In 1995 the first edition of the protocol was applied, in 1998 the second. A sample of 218 of the 1998 hospital stays was tested with both editions to evaluate the impact of differences. In 1998, with the second edition of the protocol, the inappropriateness rate of admission days and hospitalization days and the rate of in-patients inappropriate hospital stays was respectively 38.0%, 43.8% and 18.5%. Compared with 1995 results, the rates increased +6.2%, 6% and +7.5% respectively. Instead, with the first edition of the protocol, the rates were 29.7%, 37.6% and 10.7% and, compared with 1995, they were reduced -2.1%, -0.6% and -0.3%. The longitudinal multilevel analysis has allowed the evaluation of the performance of each department.

pQCT (quantitative peripheral tomography) and data evaluation of phosphocalcic metabolism in thalassaemic patients.

pQCT is a method which allows the separate determination of cortical and trabecular bone mineral density in the peripheral skeleton. 21 thalassaemic patients (8 females, 13 males) aged from 10 to 32 years, were examined using pQCT at the ultra distal radius to evaluate SSI (Stress-Strain Index). ALP, serum calcium, hydroxyproline, magnesium, IGF-I, and body surface were determined. The results show a good correlation between cortical BMD and age, concentration of hydroxyproline in urine, serum bone Gla protein, body surface index, bone density of trabecular bone and SSI. Good correlation was found between trabecular bone density and age, IGF-I, BGP and PTH, and between SSI and cortical BMD, age and BSI. The linear relationships between age and cortical and trabecular density show an increase of cortical BMD with age and a decrease of trabecular density with age. The same results were obtained considering trabecular and cortical density versus SSI.