High-cost drugs for rare diseases: their expenditure and value based on a regional area-based study.

Background: in the field of rare diseases (RDs) most of the European studies on budget impact analysis of drugs that have been conducted often lay on theoretical assumptions and focus only on Orphan drugs (ODs). Objectives: we aimed to estimate the budget impact of specific drugs for non-oncological RDs, both ODs and non-ODs, using real-world data about patients residing in Veneto Region (Italy) and to describe its expenditure structure and dynamics. Methods: a population-based multi-source observational study was conducted using data from Regional administrative databases; an ad-hoc drugs' list specific for RDs including both ODs and non-ODs and classifying them by ATC codes has been created. Results: In 2019, the total expenditure for drugs specific for RDs was EUR 97.2 million (6.6% of the total Regional budget). The RD drug list included 58 ATC codes, of which 15 ATC had an annual budget impact over EUR 1 million ("blockbuster drugs"). The most expensive treatment was a non-OD drug (Coagulation factor VIII). The two most represented therapeutical areas were the metabolic and the hematological ones. Conclusions: Cost analyses on RD high-cost drugs expenditure should consider any specific RD drug, not only ODs. Expenditure dynamics for RD drugs are peculiar showing "blockbuster drugs". Some therapeutical areas seem to be lacking in the drug research field.

Estimating mortality in rare diseases using a population-based registry, 2002 through 2019.

BACKGROUND: Rare diseases (RD) are a heterogeneous group of diseases, sharing aspects of complexity. Prognosis is variable, even in individuals with the same disease. Real-world data on RD as a whole are scarce. The aim of this study is to provide data on mortality and survival for a substantial group of RD deriving from a population-based registry, which covers the Veneto region in Italy (4.9 million inhabitants). RESULTS: During the study period, 3367 deaths occurred, mainly in males (53.9%), elderly patients (63.5%) and patients with diseases having a reported prevalence of 1-9/100000 (65.6%). When standardizing by age, the mortality ratio was higher in RD patients than in the general population, SMR = 1.93 (95% CI 1.84-2.11), with an observed gender difference, 2.01 (95% CI 1.88-2.29) in females and 1.86 (95% CI 1.73-2.10) in males. The lowest survival rates are experienced by patients with rare neurologic diseases, rare skin diseases and rare systemic or rheumatologic diseases, 58%, 68% and 81%, respectively, after a 15-year observation period. It should be noted that only 18% of patients diagnosed with motor neuron diseases were alive after 15 years from diagnosis. CONCLUSIONS: Despite progress in diagnosis, treatment and care in recent years, RD patients globally have higher mortality rates and reduced survival compared to the general population, with specific variations according to gender, age and disease group.

ORPHAcodes use for the coding of rare diseases: comparison of the accuracy and cross country comparability.

BACKGROUND: Estimates of rare disease (RD) population impact in terms of number of affected patients and accurate disease definition is hampered by their under-representation in current coding systems. This study tested the use of a specific RD codification system (ORPHAcodes) in five European countries/regions (Czech Republic, Malta, Romania, Spain, Veneto region-Italy) across different data sources over the period January 2019-September 2021. RESULTS: Overall, 3133 ORPHAcodes were used to describe RD diagnoses, mainly corresponding to the disease/subtype of disease aggregation level of the Orphanet classification (82.2%). More than half of the ORPHAcodes (53.6%) described diseases having a very low prevalence (< 1 case per million), and most commonly captured rare developmental defects during embryogenesis (31.3%) and rare neurological diseases (17.6%). ORPHAcodes described disease entities more precisely than corresponding ICD-10 codes in 83.4% of cases. CONCLUSIONS: ORPHAcodes were found to be a versatile resource for the coding of RD, able to assure easiness of use and inter-country comparability across population and hospital databases. Future research on the impact of ORPHAcoding as to the impact of numbers of RD patients with improved coding in health information systems is needed to inform on the real magnitude of this public health issue.

Real-world use of orphan medicinal products (OMPs) in rare disease (RD) patients: A population-based registry study.

Background: Despite calls for the use of real-world data, the rare diseases (RD) treatment landscape suffers from a scarcity of data referred to orphan medicinal products (OMP) use at the population level. Objectives: We aim to describe the characteristics and patterns of utilization of OMP in a sizable group of RD patients globally monitored by an area-based rare diseases registry located in the Veneto region, Italy, during a 3-year period (1 January 2019 to 31 December 2021). Methods: A list of OMP (n = 60) was assembled for study purposes, according to extensive criteria with regard to the status of orphan designation and of national reimbursement decisions. Results: OMP prescriptions involved 1,010 patients, corresponding to the 2.3% of all the patients monitored by the RD registry. Nearly one out of five (22.8%) was a pediatric patient at the time of the first prescription. OMP use interested a limited proportion (17.5%) of diseases approaching the rarity threshold, having a prevalence of less than five per 10,000, while individuals affected by these more common rare conditions represented 49% of all the patients receiving an OMP prescription. A clustering effect of OMP use was observed in selected groups of diseases, mainly, neurological, hematological, and hereditary metabolic ones. Medication plans including an OMP show in the 45.9% of the cases a high level of complexity, both in terms of nature and number of co-prescribed treatments. Off-label use interested 15.3% (n = 155) of all the RD patients with at least an OMP prescription during the study period. Conclusion: Data collected in a real-world setting through population-based registries globally monitoring RD patients, including related medication plans, have the potential to identify which diseases, and thus patients, have less benefit from the advent of OMP so far. Furthermore, in the rapidly evolving RD therapeutic landscape, they can help understand which therapeutic areas are most in need of investment to address existing unmet care needs.

[The impact of the use of treatments not included in the reimbursement classes in the care of rare patients: a real world study.] / L'impatto dell'uso dei trattamenti non compresi nelle classi di rimborsabilità nella presa in carico dei malati rari: uno studio in campo reale.

INTRODUCTION: Rare disease (RD) patients present complex therapeutic needs. When there are therapeutic options available, orphan drugs (OD) represent only a limited proportion of prescribed treatments. This study aims at investigating the real-world use of treatments considered not replaceable and essential for the care of RD patients, besides their reimbursement status, using data from a RD population-based registry. METHODS: The study is based on data derived from the Veneto region RD registry. For the period 2019-2020, we have analyzed the prescriptions of treatments defined as essential and not replaceable, besides their reimbursement status, included in therapeutic plans issued by RD expert Centres for patients resident in the Veneto region (north-east of Italy, 4.9 million inh.). The correspondent pharmaceutical costs have been estimated as well. RESULTS: In the study period there have been 22.186 prescriptions, included in 9,197 therapeutic plans issued for RD patients resident in the monitored area. The plans present a high level of complexity in terms of number and type of prescribed treatments, with 11% of the plans containing 5 or more prescriptions. 3,041 medicinal products have been prescribed in the study period, of whom 41% are drugs. Although these prescriptions are distributed among all the groups of RD patients, only a limited proportion of products (n=10) is responsible of the 50% of all the costs attributable to these treatments. Overall, the annual cost attributable to essential treatments not directly reimbursed by the national health system is quantifiable in 1 million euros per million inhabitants. CONCLUSIONS: This real-world study offers a snapshot of the complexity of treatments defined as essential, besides their reimbursement status, in therapeutic plans issued by RD expert Centres for a consistent group of RD patients monitored by a population-based registry. It highlights the complexity of the therapeutic approaches put in place for the care of RD patients, including drugs and a variety of other treatments. Population-based registries collecting data on prescribed treatments can contribute to understand the therapeutic needs of RD patients, treatments' accessibility and the impact of prescriptions on the global pharmaceutical costs.

Organ donation from patients with a rare disease is often safe: the italian guidelines.

Although a disease is defined as rare when it has a prevalence of less than 1:2000, the overall prevalence of rare diseases in the population is greater than 1%. Among potential organ donors, a similar frequency is observed. To date, guidelines have not been established, and operational decisions have been made empirically, case- by-case, based on the experience and expertise of clinicians. For this reason, the Italian Superior Health Council (CSS) has appointed a working Group to address "patients with a rare disease as potential organ donors," with the aim of devising recommendations for the management of transplant cases in which the donors have a rare disease. This group evaluated 493 diseases (10% of all rare diseases, including over 95% of patients with a rare disease) to deliver a technical report dealing with the suitability of organ donation and transplantation, with a focus on the organs most frequently used, including kidney, liver, heart, lung, and pancreas. This work has made it clear that a rare disease "per se" does not contraindicate organ donation at all. Indeed, in donors affected by a rare disease, almost 80% of the organs are suitable for transplantation, approximately 7% are unsuitable, and approximately 14% are suitable as non-standard with an acceptable risk.

Is Cannabis Legalization Eliciting Abusive Behaviors in Parents? A Case Report.

The interest in cannabis, cannabis-based compounds, and treatments is rapidly growing along with the legalization of marijuana in many countries and widespread use of cannabis derivatives in medical products. A growing body of literature is warning about possible unintentional intoxication in children because of unregulated and unsupervised use of cannabinoids by parents; to our knowledge, very rarely have parental self-prescription and self-administration to their children (affected by neurologic or other disorders or no disorders at all) been reported. We report a 4-year-old child, suffering from an anti-N-methyl-D-aspartate receptor encephalitis, who was found unpredictably positive for cannabis and other illicit substances after drug testing was performed in order to investigate the child's treatment-resistant behavioral disturbances. Toxicologic analyses were also extended to the child's parents, who finally disclosed that they had deliberately administered a cannabis-derived product (cannabidiol extract) as a home remedy for managing their child's behavior. Careless with regard to the possible adverse effects and certain that the product was legal, they presumed there was no need for them to inform the physicians in charge of treating the child of this practice.

El 31 de diciembre de 2019 las autoridades de la República Popular China comunicaron a la OMS varios casos de neumonía de etiología desconocida en Wuhan, una ciudad situada en la provincia China de Hubei. [Editorial] / On December 31, 2019, the authorities of the People's Republic of China reported to the WHO several cases of pneumonia of unknown etiology in Wuhan, a city located in the Chinese province of Hubei. [Editorial]

Resumen El 31 de diciembre de 2019 las autoridades de la República Popular China comunicaron a la OMS varios casos de neumonía de etiología desconocida en Wuhan, una ciudad situada en la provincia China de Hubei. Una semana más tarde confirmaron que se trataba de un nuevo coronavirus que ha sido denominado SARS-CoV-2 (coronavirus de tipo 2 causante del síndrome respiratorio agudo severo, del inglés, Severe acute respiratory syndrome coronavirus. Este nuevo virus causa diversos cuadros clínicos englobados bajo el término COVID-19 (enfermedad por coronavirus 2019, del inglés, coronavirus disease 2019), e incluyen desde infección asintomática, a cuadros respiratorios leves con tos, malestar general y fiebre hasta cuadros de neumonía grave con síndrome de distrés respiratorio agudo, shock séptico y fallo multi-orgánico y muerte. La mayoría de los casos de COVID-19 notificados hasta el momento cursan con cuadros leves; no obstante, tanto el pronóstico de la enfermedad como el conocimiento actual, basado en los datos más ecientes publicados y los pacientes ya tratados, indican que un porcentaje significativo de pacientes cursaran con insuficiencia respiratoria aguda, neumonía intersticial e hipoxemia grave que requieren ventilación mecánica prolongada y presentan una mortalidad elevada. La expansión del virus a todo el planeta, su capacidad de difusión y su infectividad, el elevado número de afectados en crecimiento exponencial, una mortalidad creciente con la edad y la comorbilidad de los afectados, así como su impacto sobre la vida de las personas y el sistema de asistencia sanitaria han provocado una emergencia sanitaria mundial de grandes proporciones. La Coordinación General de Docencia, conjuntamente con los diferentes servicios de esta casa de salud, participó de una manera activa y actualizada con información referente al COVID-19, destacamos los siguientes temas: Imagenología en tiempos de pandemia; embarazo y covid-19; miocardiopatía y COVID-19; planificación de una unidad de reanimación ante una pandemia y experiencia clínica; utilidad del ultrasonido en unidades críticas COVID-19; recomendaciones y experiencias para ecocardiografía y eco pulmonar en tiempos de COVID-19; cirugía plástica y quemaduras durante la pandemia; Kawasaki-like y COVID-19; VIH y COVID-19 historia natural; hemodiálisis en tiempos de COVID-19; plasma convaleciente e inmunidad en pacientes COVID-19; prevención y vacuna en COVID-19; abordaje del recién nacido con mal formación cardiaca: tratamiento sin cirugía por cateterismo; soporte nutricional en el paciente COVID-19 crítico; eventos trombo-embolicos en pacientes COVID-19, rescate vascular cerebral en tiempos COVID-19; desafíos actuales en el manejo de infarto agudo de miocardio en tiempos de pandemia; salud mental en tiempos de COVID-19; desde la fisiopatología y Medicina Basada en Evidencias (MBE) a la práctica clínica; mensajes clave para el manejo integral del paciente grave con Insuficiencia Renal Aguda (IRA) por COVID-19; experiencias y manejo del malestar emocional durante la pandemia COVID-19; Hipertensión Arterial: análisis de las últimas Felicitamos a todos los participantes y expresamos nuestro profundo reconocimiento por su vocación de servicio y entrega para enfrentar la crisis sanitaria, asumir su rol social y constituirse en sujeto social y político, que interpreta adecuadamente la realidad y plantea soluciones que contribuyen significativamente a la resolución de los problemas sociales; cada detalle cuenta para buscar una forma de contribuir como empresa cuya misión es la Salud. La Pandemia por COVID-19 ha ocasionado grandes impactos en nuestra sociedad y nos ha entristecido en lo más profundo la partida de innumerables colegas que nos dejan huella de bondad, lealtad, solidaridad y amistad, nuestro sentido pésame a sus distinguidas familias. (au)

Exposure to PFAS and small for gestational age new-borns: A birth records study in Veneto Region (Italy).

BACKGROUND: Perfluoroalkylated substances (PFAS) in serum are inversely associated with fetal growth. Small for gestational age (SGA) is a measure based on birth weight and gestational age at birth and represents a good indicator of fetal growth but it has been used only in a small number of studies. We examined the association between PFAS exposure and the risk of severe SGA in a PFAS contaminated area in the Veneto Region (North-East of Italy). METHODS: A retrospective cohort study has been developed including all singleton live births reported in the Veneto Region Birth Registry between 2003 and 2018 to mothers living in the contaminated and in a control area. We estimated the association between mothers' area of residence and severe SGA using crude RR (and 95% CI) and stepwise logistic regression, including all the maternal characteristics. RESULTS: The study included 105,114 singleton live births. The occurence of severe SGA was 3.44% in the contaminated area and 2.67% in the control area. The multivariate analysis confirmed that living in the contaminated area significantly increased the odds of severe SGA (adjusted OR 1.27 (95% C.I. 1.16, 1.39)). CONCLUSIONS: The findings suggest that living in a contaminated area by PFAS plays a role in affecting fetal growth and support the hypothesis that PFAS exposure is a risk factor for SGA. Individual data on exposure are needed to confirm the direct association.

Pocas acciones en medicina son tan gratificantes como el trasplante exitoso de un órgano de una persona a otra. [Editorial] / Few actions in medicine are as rewarding as the successful transplant of an organ from one person to another. [Editorial]

Pocas acciones en medicina son tan gratificantes como el trasplante exitoso de un órgano de una persona a otra. El rápido desarrollo de esta terapia para enfermedades terminales depende del coraje de los receptores, la generosidad de los donantes, del extraordinario esfuerzo de cirujanos, coordinadores y demás profesionales de la salud. Esta forma de tratamiento es inseparable del acto humano de donación. En efecto, el trasplante presupone una previa, explícita, libre y consciente decisión por parte del donante. Es la decisión de ofrecer sin recompensa, una parte del propio cuerpo para dar salud y bienestar a otra persona. El futuro de este sistema terapéutico realmente espectacular, será posible gracias a la donación de órganos y tejidos. El concepto de generosidad en la donación, sin ninguna clase de compensación, se ha impuesto en nuestra civilización occidental. El objetivo y compromiso del Instituto Nacional de Donación y Trasplante de Órganos Tejidos y Células (INDOT), entidad adscrita al Ministerio de Salud Pública y el Hospital José Carrasco Arteaga (IESS) con la sociedad ecuatoriana, es lograr el consentimiento efectivo de las personas a la donación de órganos y tejidos, disminuir la morbilidad de los pacientes que requieren este tratamiento, mejorar la calidad de vida y gestionar con transparencia el proceso. Hoy en día los trasplantes se han convertido en una práctica médica muy extendida con múltiples beneficios para los pacientes que de otra forma, no tendrían una segunda oportunidad. Cuando hay un trasplante no existen palabras de agradecimiento ante el gesto desinteresado y bondadoso que esto supone. Por tanto el objetivo de este editorial es destacar una actividad que salva vidas, brindando un homenaje a los que hacen posibles este milagro.

El Día Mundial de la Diabetes (DMD) es la campaña de concienciación de mayor importancia en el mundo. [Editorial] / World Diabetes Day (WDD) is the most important awareness campaign in the world. [Editorial]

El Día Mundial de la Diabetes (DMD) es la campaña de concienciación de mayor importancia en el mundo. Fue instaurado por la Federación Internacional de Diabetes (FID) y la Organización Mundial de la Salud (OMS) en 1991, como respuesta al alarmante aumento de los casos de diabetes en el mundo. En 2007, las Naciones Unidas celebraron por primera vez este día tras la aprobación de la Resolución en diciembre de 2006, lo que convirtió al ya existente Día Mundial de la Diabetes en un día oficial de la salud de la Organización de las Naciones Unidas (ONU). El (DMD) es una campaña que presenta cada año un tema elegido por la Federación Internacional de Diabetes para centrarse en distintos aspectos relacionados con la comunidad internacional de la diabetes. Los temas tratados en el pasado incluyen la diabetes y los derechos humanos, diabetes y estilo de vida, costes de la diabetes, diabetes y obesidad, diabetes y el cuidado del pie, diabetes y las personas desfavorecidas, diabetes en niños y adolescentes, diabetes educación y prevención, entre otras. (AU)

Pattern of domestic violence from 2011 to 2015 in Beira, Mozambique.

BACKGROUND: Violence against women represents a violation of a fundamental human right and is a significant cause of death and disability worldwide. In developing countries, this issue is particularly dramatic and in sub-Saharan Africa were reached 65% of women reporting domestic violence. OBJECTIVE: In this study, we assessed the burden and pattern of domestic violence registered at Beira Central Hospital, Mozambique from 2011 to 2015. METHODS: We performed a descriptive analysis of data collected at the CHB Legal Medicine Service. RESULTS: In five years, are recorded a total amount of 1,491 admissions for domestic violence of which 1307 were females. About 80% of all female cases are represented by the 11-40 age range and, in almost 90% the aggressor was the current or past partner. More than 75% were cases of repeated violence and in more than 60% there were minors attending the phenomenon. CONCLUSION: It is crucial to act immediately and with a multi-disciplinary approach in order to fight domestic violence, especially against women due to its dramatic consequences as isolation, inability to work, loss of wages, lack of participation in regular activities and limited ability to care for themselves and their children.

The Epidemiology of Transition into Adulthood of Rare Diseases Patients: Results from a Population-Based Registry.

Background: Despite the fact that a considerable number of patients diagnosed with childhood-onset rare diseases (RD) survive into adulthood, limited information is available on the epidemiology of this phenomenon, which has a considerable impact both on patients' care and on the health services. This study describes the epidemiology of transition in a population of RD patients, using data from the Veneto Region Rare Diseases Registry (VRRDR), a web-based registry monitoring since 2002 a consistent number of RD in a defined area (4.9 million inhabitants). Methods: Longitudinal cohorts of patients born in the years 1988 to 1998 and enrolled in the VRRDR in their paediatric age were identified. Data referred to this group of patients, experiencing transition from paediatric to adult age during the years 2006⁻2016, are presented. Results: 2153 RD patients (44.1% females and 55.9% males) passed from childhood to adulthood in the study period, corresponding to a 3-fold increase from 2006 to 2016. The majority of these patients was affected by congenital anomalies (32.0%), by hematologic diseases (15.9%), eye disorders (12.1%) and neoplasms (7.9%). RD patients who experienced transition from paediatric age to adulthood represent the 9.2% of adult patients enrolled in the Registry at 31 December 2016. Conclusions: We described a subset of RD young adults experiencing transition into adulthood. The data reported can be considered as minimum values for estimating the size of this increasing population presenting specific transition needs. These figures are valuable for clinicians, patients and health planners. Public policy interventions are needed in order to promote dedicated care transition pathways in the broader framework of health policies devoted to RD.

Genital trauma and vaginal bleeding: is it a lapse of time issue? A case report of a prepubertal girl and review of the literature.

Child victims of sexual abuse may present with physical findings whose interpretation requires the most exhaustive evaluation and an accurate collection of a detailed history. Genital bleeding is usually considered as an acute sign, related to a trauma that occurred shortly before its appearance. We report a case of a 34-month-old child who was referred to the emergency room with a significant vaginal hemorrhage, originating from a wide laceration of the posterior fourchette, and a negative history for accidental trauma. The characteristics of the lesion, compared to the temporal evolution of the healing process, and the witnesses' depositions led us to assume that the time elapsed between the abusive event and the physical examination was longer in respect to what had appeared at the first sight. The judicial reconstruction of the events confirmed our assumption, allowing the charge of the right abuse perpetrator. As the literature regarding this eventuality is very poor, we report this case to stress the importance for physicians to consider that an active bleeding may be the manifestation of a trauma that occurred very long before.

Diagnosis of pervasive developmental disorders: when and how? An area-based study about health care providers.

BACKGROUND: Pervasive developmental disorders (PDDs) can be very difficult to diagnose in children and to communicate such a diagnosis to their parents. Families of children with PDD learn of their child's diagnosis long after the first symptoms are noted in the child's behavior. METHODS: An area-based survey was conducted to assess all social and health care providers taking care of patients with PDDs in the Veneto Region (North-East Italy). RESULTS: Only 28% of health care providers arrived at a definite diagnosis when the child was in his/her first year of age, 51% when the child was 2-3 years old and 21% from age of 4 years and up. On average, the latency between the time of the diagnosis and its communication to the family was 6.9 months. However, a number of families did not ever have a diagnosis communicated to them. Sometimes, 68% of the providers did not communicate a PDDs diagnosis to patient's families, and 4% of them quite commonly. CONCLUSION: The well-known delay in making a diagnosis of PDDs has two distinct components: one relating to the difficulty of confirming a diagnosis of PDDs, the other, hitherto unrecognized, relating to the family being notified.

A population-based registry as a source of health indicators for rare diseases: the ten-year experience of the Veneto Region's rare diseases registry.

BACKGROUND: Although rare diseases have become a major public health issue, there is a paucity of population-based data on rare diseases. The aim of this epidemiological study was to provide descriptive figures referring to a sizable group of unrelated rare diseases. METHODS: Data from the rare diseases registry established in the Veneto Region of north-east Italy (population 4,900,000), referring to the years from 2002 to 2012, were analyzed. The registry is based on a web-based system accessed by different users. Cases are enrolled by two different sources: clinicians working at Centers of expertise officially designated to diagnose and care patients with rare diseases and health professionals working in the local health districts. Deaths of patients are monitored by Death Registry. RESULTS: So far, 19,547 patients with rare diseases have been registered, and 23% of them are pediatric cases. The overall raw prevalence of the rare diseases monitored in the population under study is 33.09 per 10,000 inhabitants (95% CI 32.56-33.62), whilst the overall incidence is 3.85 per 10,000 inhabitants (95% CI 3.67-4.03). The most commonly-recorded diagnoses belong to the following nosological groups: congenital malformations (Prevalence: 5.45/10,000), hematological diseases (4.83/10,000), ocular disorders (4.47/10,000), diseases of the nervous system (3.51/10,000), and metabolic disorders (2,95/10,000). Most of the deaths in the study population occur among pediatric patients with congenital malformations, and among adult cases with neurological diseases. Rare diseases of the central nervous system carry the highest fatality rate (71.36/1,000). Rare diseases explain 4.2% of general population Years of Life Lost (YLLs), comparing to 1.2% attributable to infectious diseases and 2.6% to diabetes mellitus. CONCLUSIONS: Our estimates of the burden of rare diseases at population level confirm that these conditions are a relevant public health issue. Our snapshot of their epidemiology is important for public health planning purposes, going to show that population-based registries are useful tools for generating health indicators relating to a considerable number of rare diseases, rather than to specific conditions.

Visual, motor, and psychomotor development in small-for-gestational-age preterm infants.

PURPOSE: To study causal links between the visual, cognitive, and psychomotor outcomes of premature babies who are small for gestational age (SGA). METHODS: A cohort study of 17 SGA cases and 34 controls who were appropriate for gestational age (AGA) was carried out. The cases were all premature babies without any other pathology. All subjects underwent a visual, mental, and psychomotor evaluation at 1 year of age. RESULTS: Of the SGA cases, 41% had a "below normal" visual acuity versus 17.7% of the AGA controls. At 1 year of age the SGA babies showed an odds ratio of 18.73 for low visual acuity, 9.09 for low mental performance, and no significant risk for a decreased psychomotor performance. CONCLUSIONS: In this small cohort of premature infants, the SGA babies were more prone to developing low visual performance and abnormal cognitive development.

Unimanual and bimanual intensive training in children with hemiplegic cerebral palsy and persistence in time of hand function improvement: 6-month follow-up results of a multisite clinical trial.

This study aims to compare in hemiplegic children the effectiveness of intensive training (unimanual and bimanual) versus standard treatment in improving hand function, assessing the persistence after 6 months. A multicenter, prospective, cluster-randomized controlled clinical trial was designed comparing 2 groups of children with hemiplegic cerebral palsy, treated for 10 weeks (3 h/d 7 d/wk; first with unimanual constraint-induced movement therapy, second with intensive bimanual training) with a standard treatment group. Children were assessed before and after treatment and at 3 and 6 months postintervention using Quality of Upper Extremity Skills Test (QUEST) and Besta Scales. One hundred five children were recruited (39 constraint-induced movement therapy, 33 intensive bimanual training, 33 standard treatment). Constraint-induced movement therapy and intensive bimanual training groups had significantly improved hand function, showing constant increase in time. Grasp improved immediately and significantly with constraint-induced movement therapy, and with bimanual training grasp improved gradually, reaching the same result. In both, spontaneous hand use increased in long-term assessment.