Fiocruz lança Estratégia para Terapias Avançadas no tratamento de câncer e doenças raras

Como parte da Estratégia e em parceria com o Ministério da Saúde (MS), a Fundação assinou um acordo de colaboração com a organização norte-americana sem fins lucrativos Caring Cross, que prevê a transferência de tecnologia, pelo Instituto de Tecnologia em Imunobiológicos (Bio-Manguinhos/Fiocruz), para a produção de células CAR-T e vetores lentivirais.

Epidemiologic Research of Rare Cancers: Trends, Resources, and Challenges.

BACKGROUND: The goals of this project were to assess the status of NCI's rare cancer-focused population science research managed by the Division of Cancer Control and Population Sciences (DCCPS), to develop a framework for evaluation of rare cancer research activities, and to review available resources to study rare cancers. METHODS: Cancer types with an overall age-adjusted incidence rate of less than 20 cases per 100,000 individuals were identified using NCI Surveillance, Epidemiology and End Results (SEER) Program data. SEER data were utilized to develop a framework based on statistical commonalities. A portfolio analysis of DCCPS-supported active grants and a review of three genomic databases were conducted. RESULTS: For the 45 rare cancer types included in the analysis, 123 active DCCPS-supported rare cancer-focused grants were identified, of which the highest percentage (18.7%) focused on ovarian cancer. The developed framework revealed five clusters of rare cancer types. The cluster with the highest number of grants (n = 43) and grants per cancer type (10.8) was the cluster that included cancer types of higher incidence, average to better survival, and high prevalence (in comparison with other rare cancers). Resource review revealed rare cancers are represented in available genomic resources, but to a lesser extent compared with more common cancers. CONCLUSIONS: This article provides an overview of the rare cancer-focused population sciences research landscape as well as information on gaps and opportunities. IMPACT: The findings of this article can be used to develop efficient and comprehensive strategies to accelerate rare cancer research.See related commentary by James V. Lacey Jr, p. 1300.

Pharmacological management of rare coagulation factor deficiencies besides hemophilia.

INTRODUCTION: Rare coagulation factor deficiencies are less-known disorders with variable effects on the patient's life. Management of such patients is a challenge due to the paucity of evidence-based data, more so when patients with these rare disorders encounter a more rare, related condition, like inhibitor development or thrombosis. AREA COVERED: A comprehensive literature search related to RCFDs and management was performed in PubMed in order to discuss therapeutic options and challenges, prophylaxis, management of minor and major surgeries, obstetric and gynecological complications, inhibitor development, and thrombosis. EXPERT OPINION: Although significant changes have occurred in the management of RCFDs in recent years, more evidence-based studies besides expert opinion are needed for optimal management.

Osteogénesis imperfecta de tipo III en niño ecuatoriano / Osteogenesis imperfecta type III in an Ecuadorian child

Introducción: la osteogénesis imperfecta es una rara enfermedad genética hereditaria caracterizada por su heterogeneidad causada por defectos del tejido conectivo con el rasgo de fragilidad ósea determinante de múltiples fracturas, incluso prenatales; deformidades de huesos largos y columna vertebral y otros síntomas extra-esqueléticos, como escleróticas de color azul, dentinogénesis imperfecta, trastorno de audición y afectación cardiovascular. Objetivo: Presentar un paciente con las características clínicas e imagenológicas de osteogénesis imperfecta de tipo III. Presentación del caso: Niño ecuatoriano de 4 años de edad de baja talla con antecedente de fracturas múltiples desde los 8 meses de nacido, con deformidad en columna vertebral demostrada por radiología por cifoescoliosis en forma de "S" y fracturas vertebrales, con deformidad progresiva en huesos largos; ha sufrido 16 fracturas, no deambula, sensorio presente, orientado en tiempo y espacio, desarrollo cognitivo normal para la edad. La fragilidad ósea del niño según el fenotipo clasifica al diagnóstico de tipo III de osteogénesis imperfecta, el cual es progresivo e invalidante por las deformidades óseas y múltiples fracturas demostradas en exámenes imagenológicos, sin modificaciones en el color de escleróticas, de herencia presumiblemente dominante. Conclusiones: La descripción clínica y radiológica de osteogénesis imperfecta, afección poco conocida, correspondiente al fenotipo III de la enfermedad, reportada en niño ecuatoriano de 4 años de edad, con talla baja que no deambula, expresión de la severidad de su afección genética, con severas alteraciones óseas por su fragilidad con fracturas múltiples en huesos largos y vértebras(AU)

Introduction: Osteogenesis imperfecta is a rare hereditary genetic disease characterized by its heterogeneity caused by connective tissue defects with the feature of bone fragility determining multiple fractures, even prenatal ones; also deformities of long bones and spine, and other extra-skeletal symptoms, such as blue sclerotic, dentinogenesis imperfecta, hearing disorder and cardiovascular affectations. Objective: To present a patient with clinical and radiological findings of osteogenesis imperfect type III. Case presentation: Ecuadorean male child of 4 years old, with a short height, history of multiple fractures from 8 months of age, with spinal deformity demonstrated by radiology due to "S" shaped kyphoscoliosis and vertebral fractures, with progressive deformity in long bones. The boy has suffered 16 fractures, he does not wander, and he is sensory present, oriented in time and space, with normal cognitive development for his age. The bone fragility of the child according to the phenotype classifies in the type III diagnosis of osteogenesis imperfecta, which is progressive and invalidating due to bone deformities and multiple fractures evidenced in imaging tests, without changes in the color of sclerotics and of presumably dominant inheritance. Conclusions: The clinical and radiological description of osteogenesis imperfecta, which is little-known pathology, corresponding to type III phenotype is reported in a 4-year-old boy who, due to his involvement, has a short height and does not wander as a consequence of the severity of bone affectations with fractures in long bones and vertebrae, mainly produced by the fragility of the bones due to his genetic disease(AU)

Prenatal education for congenital toxoplasmosis.

BACKGROUND: Congenital toxoplasmosis is considered a rare but potentially severe infection. Prenatal education about congenital toxoplasmosis could be the most efficient and least harmful intervention, yet its effectiveness is uncertain. OBJECTIVES: To assess the effects of prenatal education for preventing congenital toxoplasmosis. SEARCH METHODS: We searched the Cochrane Pregnancy and Childbirth Group's Trials Register (31 May 2015), and reference lists of relevant papers, reviews and websites. SELECTION CRITERIA: Randomized and quasi-randomized controlled trials of all types of prenatal education on toxoplasmosis infection during pregnancy. Cluster-randomized trials were eligible for inclusion. DATA COLLECTION AND ANALYSIS: Two review authors independently assessed trials for inclusion and risk of bias, extracted data and checked them for accuracy. MAIN RESULTS: Two cluster-randomized controlled trials (RCTs) (involving a total of 5455 women) met the inclusion criteria. The two included trials measured the effectiveness of the intervention in different ways, which meant that meta-analysis of the results was not possible. The overall quality of the two studies, as assessed using the GRADE approach, was low, with high risk of detection and attrition bias in both included trials.One trial (432 women enrolled) conducted in Canada was judged of low methodological quality. This trial did not report on any of the review's pre-specified primary outcomes and the secondary outcomes reported results only as P values. Moreover, losses to follow-up were high (34%, 147 out of 432 women initially enrolled). The authors concluded that prenatal education can effectively change pregnant women's behavior as it increased pet, personal and food hygiene. The second trial conducted in France was also judged of low methodological quality. Losses to follow-up were also high (44.5%, 2233 out of 5023 women initially enrolled) and differential (40% in the intervention group and 52% in the control group). The authors concluded that prenatal education for congenital toxoplasmoses has a significant effect on improving women's knowledge, whereas it has no effect on changing women's behavior. In this trial 17/3949 pregnant women seroconverted for toxoplasmosis: 13/2591 (0.5%) in the intervention group and 4/1358 (0.3%) in the control group. The rate of seroconversion detected during the study did not differ between groups (risk ratio (RR) 1.70, 95% confidence interval (CI) 0.56 to 5.21; participants = 3949; studies = one, low quality evidence). The number of events was too small to reach conclusions about the effect of prenatal education on seroconversion rate during pregnancy.No other randomized trials on the effect of prenatal education on congenital toxoplasmosis rate, or toxoplasmosis seroconversion rate during pregnancy were detected. AUTHORS' CONCLUSIONS: Even though primary prevention of congenital toxoplasmosis is considered a desirable intervention, given the lack of related risks compared to secondary and tertiary prevention, its effectiveness has not been adequately evaluated. There is very little evidence from RCTs that prenatal education is effective in reducing congenital toxoplasmosis even though evidence from observational studies suggests it is. Given the lack of good evidence supporting prenatal education for congenital toxoplasmosis prevention, further RCTs are needed to confirm any potential benefits and to further quantify the impact of different sets of educational intervention.

Polish activity within Orphanet Europe--state of art of database and services.

Orphanet is an international project aiming to help in improvement the diagnostic process, care and treatment of patients with rare diseases, and to provide information on development in research and new therapy. Orphanet is currently represented in 38 countries. The infrastructure and coordination activities are jointly funded by Inserm, the French Directorate General for Health, and the European Commission. Moreover, certain services are specially funded by other partners. Orphanet's activities in each country of the network are partially financed by national institutions and(or) specific contracts. In this paper we present the Orphanet portal as well as the Polish national activity within this project.

Organizing national responses for rare blood disorders: the Italian experience with sickle cell disease in childhood.

BACKGROUND: Sickle cell disease (SCD) is the most frequent hemoglobinopathy worldwide but remains a rare blood disorder in most western countries. Recommendations for standard of care have been produced in the United States, the United Kingdom and France, where this disease is relatively frequent because of earlier immigration from Africa. These recommendations have changed the clinical course of SCD but can be difficult to apply in other contexts. The Italian Association of Pediatric Hematology Oncology (AIEOP) decided to develop a common national response to the rising number of SCD patients in Italy with the following objectives: 1) to create a national working group focused on pediatric SCD, and 2) to develop tailored guidelines for the management of SCD that could be accessed and practiced by those involved in the care of children with SCD in Italy. METHODS: Guidelines, adapted to the Italian social context and health system, were developed by 22 pediatric hematologists representing 54 AIEOP centers across Italy. The group met five times for a total of 128 hours in 22 months; documents and opinions were circulated via web. RESULTS: Recommendations regarding the prevention and treatment of the most relevant complications of SCD in childhood adapted to the Italian context and health system were produced. CONCLUSIONS: Creating a network of physicians involved in the day-to-day care of children with SCD is feasible in a country where it remains rare. Providing hematologists, primary and secondary care physicians, and caregivers across the country with web-based guidelines for the management of SCD tailored to the Italian context is the first step in building a sustainable response to a rare but emerging childhood blood disorder and in implementing the World Health Organization's suggestion "to design (and) implement … comprehensive national integrated programs for the prevention and management of SCD".

Prenatal education for congenital toxoplasmosis.

BACKGROUND: Congenital toxoplasmosis is considered a rare but potentially severe infection. Prenatal education about congenital toxoplasmosis could be the most efficient and least harmful intervention, yet its effectiveness is uncertain. OBJECTIVES: To assess the effects of prenatal education for preventing congenital toxoplasmosis. SEARCH METHODS: We searched the Cochrane Pregnancy and Childbirth Group's Trials Register (15 January 2012), PubMed (1966 to 15 January 2012), EMBASE (1980 to 15 January 2012), CINAHL (1982 to 15 January 2012), LILACS (1982 to 15 January 2012), IMEMR (1984 to 15 January 2012), and reference lists of relevant papers, reviews and websites. SELECTION CRITERIA: Randomized and quasi-randomized controlled trials (RCTs) of all types of prenatal education on toxoplasmosis infection during pregnancy. Cluster-randomized trials were included. DATA COLLECTION AND ANALYSIS: Two review authors independently assessed trials for inclusion and study quality. Two review authors extracted data. Data were checked for accuracy. MAIN RESULTS: Two cluster-randomized controlled trials (involving a total of 5455 women) met the inclusion criteria. The two included trials measured the effectiveness of the intervention in different ways which meant that meta-analysis of the results was not possible One trial (432 women enrolled) conducted in Canada was judged of low methodological quality. The authors did not report measure of association but only provided P values (P less than 0.05) for all outcomes. Moreover, losses to follow-up were high (34%, 147 out of 432 women initially enrolled). The authors concluded that prenatal education can effectively change pregnant women's behavior as it increased pet, personal and food hygiene. The second trial conducted in France was also judged of low methodological quality. Losses to follow-up were high (44.5%, 2233 out of 5023 women initially enrolled) and differential (40% in the intervention group and 52% in the control group). The authors concluded that prenatal education for congenital toxoplasmoses has a significant effect on improving women's knowledge whereas it has no effect on changing women's behavior. In this trial 17/3949 pregnant women seroconverted for toxoplasmosis: 13/2591 (0.5%) in the intervention group and 4/1358 (0.3%) in the control group. The number of events was too small to reach conclusions about the effect of prenatal education on seroconversion rate during pregnancy.No other randomized trials on the effect of prenatal education on congenital toxoplasmosis rate, or toxoplasmosis seroconversion rate during pregnancy were detected. AUTHORS' CONCLUSIONS: Even though primary prevention of congenital toxoplasmosis is considered a desirable intervention, given the lack of related risks compared to secondary and tertiary prevention, its effectiveness has not been adequately evaluated. There is very little evidence from RCTs that prenatal education is effective in reducing congenital toxoplasmosis even though evidence from observational studies suggests it is. Given the lack of good evidence supporting prenatal education for congenital toxoplasmosis prevention, further RCTs are needed to confirm any potential benefits and to further quantify the impact of different sets of educational intervention.

Thrombosis in patients with hemorrhagic disorders.

Venous thromboembolism is common in the general population with increasing age as one of the most important risk factors. The care of hemophilia and von Willebrand disease has improved in recent decades, resulting in the expectation of a growing population of aging people with these disorders. Thrombosis seems rare in hemorrhagic disorders but studies documenting the true epidemiology are virtually lacking. Events have been reported, however, primarily catheter-related thrombophlebitis in hemophilia, but also deep vein thrombosis and pulmonary embolism have been described in von Willebrand disease, usually in conjunction with major surgery and prolonged replacement therapy with high factor levels. Thromboprophylaxis is likely not warranted in most cases. Instead, well-designed therapy and careful monitoring are important measures to prevent risk. In von Willebrand disease particularly, the variation of the phenotype and products used for replacement are challenges, as infusion of von Willebrand factor will increase the endogenous level of factor VIII. Long-term replacement therapy into old age is becoming more common in hemophilia but will not increase occurrence of thromboembolic disease, as factor levels still will be low and have a preventive effect.

EUROPLAN: a project to support the development of national plans on rare diseases in Europe.

BACKGROUND/AIMS: National Plans for Rare Diseases (RDs) are the common denominator of current public health policy concerns on RDs across the EU. With the aim of a better distribution of the available resources, they conjugate the European objective that aims at ensuring that patients with RDs have access to high-quality care - including diagnostics, treatment and rehabilitation - with the national priorities of selecting specific measures for adoption and implementation. METHODS: The European Project for Rare Diseases National Plans Development (EUROPLAN, www.europlanproject.eu) is cofunded by the EU Commission (DG-SANCO) and is coordinated by the Italian National Center for Rare Diseases of the Istituto Superiore di Sanità (ISS). The EUROPLAN goal is to promote the implementation of National Plans or Strategies to tackle RDs and share relevant experiences within countries, linking national efforts, through a common strategy at a European level. In order to fulfill these objectives, EUROPLAN involved health authorities, clinicians, scientists, the European Organisation for Rare Diseases (EURORDIS), and many other patient groups as associated and collaborating partners from several European countries. RESULTS: The project was launched in 2008 and foresaw 2 implementation phases: phase 1 (2008-2011) to build the consensus definition of operational tools (recommendations and indicators), and the ongoing phase 2 (2012-2015), mainly aimed at capacity building with the proactive involvement of multilevel stakeholders. EUROPLAN is facilitating and accelerating the implementation of National Plans in almost all EU and several non-EU Countries. CONCLUSIONS: EUROPLAN is a European and an international process more than a project, and it could be defined as a 'litmus test' demonstrating how the collaboration between institutions and patients' associations can accelerate the process of awareness and development of policies and actions.

Cuidado integral de personas con enfermedades raras. enfermedades raras

Se conoce como Enfermedades Raras (ER) a un grupo depatologías de baja prevalencia (5 o menos de cada 10.000personas), la mayoría crónicas, que generan gran impactosanitario, social y económico para el paciente, para sufamilia y su entorno. En la provincia de Buenos Aires, laproyección de datos de la suma de prevalencias representaun total de 1 167 000 habitantes que las padecen.La Ley Provincial Nº 14239/10, declara de “InterésProvincial el diagnóstico, seguimiento e investigación de lasdenominadas enfermedades raras”, y crea el CentroProvincial de Referencia, Seguimiento y Divulgación deEnfermedades Raras.El Centro, dependiente del Ministerio de Salud de laProvincia de Buenos Aires, establece sus basesconceptuales en este documento, constituyendo sus ejesde trabajo en:1. Difusión comunitaria para la concientización y elmás amplio conocimiento de las Enfermedades Raras en elámbito social de la Provincia; 2. Capacitación Continua y Progresiva del Equipo deSalud, con foco especial en el equipo de Atención Primaria;3. Creación, mantenimiento y articulación de una Redde Atención de ER con todos los niveles del SistemaSanitario,Para ello se proponen las siguientes líneas de acción:1. Creación de un Sistema de Registro de Pacientes conER, compatible con registros Internacionales;2. Diseño e implementación de estrategias de difusióncomunitaria, unificando actividades en Salud, Educación yDesarrollo Social, principales actores provincialesinvolucrados.3. Diseño y aplicación del Programa de capacitacióndel Equipo de APS y de los otros niveles de la red deatención.4. Conformación del presente documento comoherramienta estratégica de base para la actividadconcerniente a ER, para la construcción participativa de lared de atención de pacientes con ER con las RegionesSanitarias y con los Sistemas Locales de Salud.

Xeroderma pigmentosum family support group: Helping families and promoting clinical initiatives.

The past two decades of research into Xeroderma pigmentosum (XP), an autosomal recessive disease, has been marked by significant progress in understanding the molecular basis of this rare disease. More importantly, especially from the perspective of the affected families, is that this knowledge has been applied to diagnose the condition both in utero as well as in the very early days of life. The eight known XP genes and their different phenotypes present a number of challenges that the XP Workshop sponsored by the NIH in 2010 has highlighted. There is little current treatment specifically designed for any of the XP types other than standard dermatological and neurological evaluations and care. The Xeroderma Pigmentosum Family Support Group (XPFSG) is dedicated to serving families with children and adults with all forms of XP and to help them better understand the condition, to identify practical measures which can be taken by the XP patients and their families to mitigate the effects of the disease, and to serve as patient advocates to help families discuss issues with their physicians. We summarize our efforts in terms of outreach within the US and abroad to affected families and discuss XPFSG-sponsored clinical initiatives that include molecular diagnoses, treatment, and initial proof-of-concept studies that can, if successful, improve the lives of XP patients in the near term.

Creating a European Union framework for actions in the field of rare diseases.

Rare diseases, including those of genetic origin, are defined by the European Union as life-threatening or chronically debilitating diseases which are of such low prevalence (less than 5 per 10,000). The specificities of rare diseases - limited number of patients and scarcity of relevant knowledge and expertise - single them out as a unique domain of very high European added-value. The legal instruments at the disposal of the European Union, in terms of the Article 152 of the Treaties of the European Union, are very limited. However a combination of instruments using the research and the pharmaceutical legal basis and an intensive and creative use of funding from the First Public Health Programme 2003-2008 and from the Second Health Programme 2008-2013 has permitted to create a solid basis that Member States have considered enough to put rare diseases in a privileged position in the health agenda. The adoption of the Commission Communication, in November 2008, and of the Council Recommendation, in June 2009, and the future adoption of the Directive on Cross-border healthcare, maybe during 2010, have created an operational framework to act in the field of rare disease with European coordination in several areas (classification and codification, European Reference Networks, orphan drugs, European Committee of Experts, etc.). In conclusion, Rare diseases is an area with enormous and practical potentialities for the European cooperation.

National plans and strategies on rare diseases in Europe.

This analysis of national plans and strategies on RD in Europe shows that a few countries have already set up national plans. Existing national plans show a good consistency, but also a quite different stage of progress, depending on start date as well as on resource allocation. Several other EU countries have launched actions on RD, often with a considerable strategic effort; however, such initiatives are yet not integrated in a consistent national strategy taking into account the EC recommendations. The project EUROPLAN represents a major initiative to support the development of a shared strategy on RD at EU and Member State level; critical steps include the comparative evaluation of existing plans and actions, identification of gaps and achievements, the development of consensus indicators, as well as the integration of successful national achievements within the EU strategy.

Neglected diseases caused by bacterial infections.

Bacterial infections represent a major health problem, especially in third world countries. In endemic regions, large populations of people are greatly affected, but the medical care is very limited. In this review, the neglected diseases buruli ulcer and trachoma are elucidated. Buruli ulcer is caused by Mycobacterium ulcerans which produces an outstanding immunosuppressive toxin mycolactone that induces an ulcerative, necrotic skin disease. Until today, only the combination of rifampin/streptomycin is used to treat buruli ulcer. However, this therapy is ineffective and expensive. Here, we report new findings that suggest pharmaceutical formulations such as rifapentine, in combination with clarithromycin or moxifloxacin that have shown promising results in mice footpad trials. Moreover, alternative treatment options such as heat therapy, nitric oxide cremes and French clay show bactericidal effects. The genotyping of M. ulcerans also promises new ways of finding drug targets and vaccines. Trachoma, induced by the bacterium Chlamydia trachomatis, is the primary infectious cause of blindness worldwide. Recurrent infections lead to chronic inflammation of the upper tarsal conjunctiva. As a consequence, scarring and distortion of the eye lids occur, eventually resulting in blindness. First-line medications for trachoma treatment are bacteriostatic agents such as topically applied tetracylines and systematically administered azithromycin. Surgery, environmental improvements and personal hygiene are further crucial factors in controlling trachoma. Moreover, efforts are being undertaken towards the development of vaccine systems, with the major outer membrane protein and the polymorphic membrane protein acting as attractive candidates.

[A rare coagulation disorder. Diagnostics and management in cases of hereditary dysfibrinogenemia]. / Seltene Ursache einer hämorrhagischen Diathese. Diagnostik und perioperatives Management bei hereditärer Dysfibrinogenämie.

Before elective surgery, it is mandatory that a precise history be taken to detect increased hemorrhagic diathesis and that thrombocytes, Quick/INR, and aPTT be determined. If pathological levels are found, further laboratory tests are necessary after frequent causes (e.g., liver cirrhosis) have been excluded. Single-factor analysis for the von Willebrand's factor antigen and if necessary further tests to check for von Willebrand's syndrome (multimeric analysis) as well as platelet function tests should be performed.Dysfibrinogenemia is a rare coagulation disorder, which causes elevated INR. It shows a wide spectrum of clinical manifestations including thrombophilia, excessive bleeding, and even asymptomatic cases. We present a 72-year-old patient with asymptomatic dysfibrinogenemia who needed hip replacement due to arthrosis. Lowered fibrinogen levels were substituted prior to operation and the clinical course afterwards was uneventful under additional prophylactic anticoagulation in order to prevent thrombosis. The case report illustrates the interdisciplinary teamwork which is very important in the management of patients with coagulation disorders.

A rare case of skin cancer above a subcutaneously implanted pacemaker: implications for future implants.

BACKGROUND: The occurrence of a skin neoplasm close to the position of an implanted pacemaker or cardioverter-defibrillator device is not very common. CASE REPORT: We report on an 82-year-old patient who developed a basal cell carcinoma in the skin directly above a subcutaneously implanted pacemaker generator. The patient presented with a history of recurrent basalioma at various locations. The pacemaker (Kappa KDR 731; Medtronic) was implanted 17 months before and represented a series that was recalled because of problems occurring after submuscular implantation. CONCLUSION: Primary submuscular implantation of pacemaker devices should be carefully considered in elderly patients with a history of previous skin tumors.

Meeting the ongoing needs of survivors of rarer cancer.

With more treatment options for people with cancer long-term survivorship is increasing. Physical and psycho-social needs have been identified in survivors of common cancers but very little has been written about the needs of patients with rarer cancers. Patients treated for rarer cancer are discharged to the primary health care team (PHCT), yet little is known about the assessment, management and support of these patients. Thirty-nine semi-structured interviews were conducted with (1) survivors of and (2) people living with rarer cancer (i.e. <5% of cancer burden). Participants were asked about physical and psycho-social needs and service provision. Data were analysed thematically using Atlas ti. Contrary to expectation, disease-free survivors of rarer cancer were indistinguishable from those living with disease in their ability to cope, and range of symptoms and needs. Participants with a clinical nurse specialist (CNS) reported that they were well supported on their return home and their needs were met. Participants without a CNS were referred to the PHCT who were unsure how to assess or support them. These participants felt abandoned. There is a need for the rehabilitation of patients with rarer cancer to strengthen individual coping mechanisms, and family and social support. Although there are resource and training implications, this is a potential role for the PHCT, district nursing in particular, and may lead to more focused and targeted provision of services.

PET scintigraphy of etoposide-induced pulmonary toxicity.

A patient with chronic myelomonocytic leukemia developed drug-induced pulmonary toxicity after using low dose oral etoposide. Because etoposide-induced pulmonary toxicity is an uncommon but serious adverse event, clinicians must be vigilant about the possibility of it, so that the optimal treatment can start as soon as possible. This report demonstrates that PET scintigraphy might be a helpful tool in the early diagnosis of drug-induced pulmonary toxicity.