Eva Frey

Validation of the German version of the Pediatric Quality of Life InventoryTM (PedsQLTM) in childhood cancer patients off treatment and children with epilepsy

The Pediatric Quality of Life InventoryTM (PedsQLTM) is a relatively new instrument developed in the US to assess health-related quality of life (HRQL) in healthy and ill children and adolescents. It is quick and easy to use, suitable for assessment by children and parents and based on a modular approach with a generic measure addressing all general domains of HRQL and disease-specific modules. We report here the results of translating the original US generic core instrument and the cancer module into German and testing the German PedsQLTM in samples of paediatric patients who have been treated for cancer or epilepsy. Like in studies with the original instrument, score distributions tended to be skewed toward higher HRQL, but a full response range was demonstrated for each item. The German PedsQLTM is reliable in terms of internal consistency with the majority of scales exceeding a Cronbachs α of 0.70. Clinical validity was confirmed by differences between the cancer and epilepsy groups in the expected direction and by the ability of the PedsQLTM to discriminate between subjects with different degrees of medical and psychosocial sequelae. Comparing our findings to US results, the German PedsQLTM seems to be equivalent to the original version. Future methodologic research should evaluate construct validity, sensitivity and responsiveness, and test the usefulness of the instrument in other clinical populations and healthy children.

Survivorship after childhood cancer: PanCare: A European Network to promote optimal long-term care.

Survival after childhood cancer has improved substantially over recent decades. Although cancer in childhood is rare increasingly effective treatments have led to a growing number of long-term survivors. It is estimated that there are between 300,000 and 500,000 childhood cancer survivors in Europe. Such good survival prospects raise important questions relating to late effects of treatment for cancer. Research has shown that the majority will suffer adverse health outcomes and premature mortality compared with the general population. While chronic health conditions are common among childhood cancer survivors, each specific type of late effect is very rare. Long-term effects must be considered particularly when addressing complex multimodality treatments, and taking into account the interaction between aspects of treatment and genotype. The PanCare Network was set up across Europe in order to effectively answer many of these questions and thereby improve the care and quality of life of survivors. The need for a structured long-term follow-up system after childhood cancer has been recognised for some time and strategies for implementation have been developed, first nationally and then trans-nationally, across Europe. Since its first meeting in Lund in 2008, the goal of the PanCare Network has been to coordinate and implement these strategies to ensure that every European survivor of childhood and adolescent cancer receives optimal long-term care. This paper will outline the structure and work of the PanCare Network, including the results of several European surveys, the start of two EU-funded projects and interactions with relevant stakeholders and related projects.

The views of European clinicians on guidelines for long-term follow-up of childhood cancer survivors.

Evidence‐based guidelines are needed to guide effective long‐term follow‐up (LTFU) of childhood cancer survivors (CCS) at risk of late adverse effects (LAEs). We aimed to ascertain the use of LTFU guidelines throughout Europe, and seek views on the need for pan‐European LTFU guidelines.

Gastrointestinal stromal tumours in children and young adults: a clinicopathologic series with long-term follow-up from the database of the Cooperative Weichteilsarkom Studiengruppe (CWS).

BACKGROUND Studies on gastrointestinal stromal tumours (GIST) in the paediatric population are limited to case reports or small case series. PATIENTS AND METHODS We conducted a retrospective study to describe the long-term outcome of children and adolescents with GIST registered in the database of the Cooperative Weichteilsarkom Studiengruppe (CWS). RESULTS Sixteen patients (female, n = 11) were identified. Median age at diagnosis was 13.5 years. In four female patients presence of thoracic masses in addition to GIST led to the diagnosis of complete or incomplete Carney triad. Three female patients had metastatic disease at diagnosis, the remaining thirteen GIST were localised. The stomach was the most common primary site of the tumour, followed by the small bowel and colon/abdomen. All patients underwent tumour resection. Receptor tyrosine kinase inhibitors (RTKI) were administered in five patients. With a median follow-up of 96 months all patients are alive, nine of them in first CR. Four female patients developed local or distant recurrence; three of them achieved second CR and one a PR. Two individuals have extensive progressive (n = 1) or stable (n = 1) disease. Estimated progression-free survival at 5 years is 0.63 (95%CI: 0.50-0.86). CONCLUSIONS Although long-term overall survival is favourable, approximately 30 percent of patients develop disease progression. International cooperation in registration, tissue collection and molecular studies are required to obtain reliable data on the clinical course of these rare tumours in the paediatric population. Biological studies are a prerequisite for initiation of studies with RTKI.

Tumor- and treatment-related side effects after multimodal therapy of childhood intracranial germ cell tumors

Multimodality treatment approaches have dramatically improved the outcome of patients with intracranial germ cell tumors and are resulting in an increasing number of long‐term survivors. The aim of the present study was to evaluate prospectively the development of side effects in children, adolescents and young adults after treatment for intracranial germ cell tumors. Nine patients with a median age of 14 y at diagnosis and a median follow‐up of 7.25 y underwent a detailed long‐term evaluation including physical and neuro‐ophthalmologic examinations, routine laboratory and endocrine stimulation tests, neuropsychometric testing, audiometry and spirometry at repeated intervals. Endocrine deficiencies requiring hormone replacement therapy occurred in all patients. Neuro‐ophthalmologic side effects were observed in 8 of the 9 patients, urinary electrolyte wasting in 4 of the 9, alopecia in 3 of the 9 and high‐frequency hearing loss in 2 of the 9. Neuropsychologic examinations revealed pathologic results in all five tested patients.

Transition guidelines: An important step in the future care for childhood cancer survivors. A comprehensive definition as groundwork

Evidence-based clinical practice guidelines are essential to ensure that childhood cancer survivors at risk of chronic health conditions receive effective long-term follow-up care. However, adult survivors of childhood cancer are not always engaged in recommended health promotion and follow-up practices, as many centres do not have a formal transition programme that prepares survivors and their families for successful transfer from child-centred to adult-oriented healthcare. The need for a specific pan-European guideline for the transition of care for childhood cancer survivors has been recognised. The first step is to define the concept of transition of care for survivors of childhood cancer based on existing evidence.

Transitional care of a childhood cancer survivor to adult services: facilitating the process of individual access to different models

Purpose of reviewIn the past 20 years, several papers have been published concerning healthcare transition from pediatric after-care to long-term follow-up in adult health service for childhood cancer survivors. Initially, the papers were optimistic and included guidelines and models of care. However, after this optimistic beginning, more recent research has found that transitional care is not a successful process. Recent findingsAlthough the number of papers regarding transition in the past 2 years is few, they include revealing results about barriers and facilitators of the transition process. SummaryThere is some evidence that joint pediatric/adult transition clinics are more successful in providing transitional care. The use of tools to assess individual psychological factors is important for the transition process and could lead to more individualized models of transition.

Balancing the benefits and harms of thyroid cancer surveillance in survivors of Childhood, adolescent and young adult cancer : Recommendations from the international Late Effects of Childhood Cancer Guideline Harmonization Group in collaboration with the PanCareSurFup Consortium

Radiation exposure to the thyroid gland during treatment of childhood, adolescent and young adult cancer (CAYAC) may cause differentiated thyroid cancer (DTC). Surveillance recommendations for DTC vary considerably, causing uncertainty about optimum screening practices. The International Late Effects of Childhood Cancer Guideline Harmonization Group, in collaboration with the PanCareSurFup Consortium, developed consensus recommendations for thyroid cancer surveillance in CAYAC survivors. These recommendations were developed by an international multidisciplinary panel that included 33 experts in relevant medical specialties who used a consistent and transparent process. Recommendations were graded according to the strength of underlying evidence and potential benefit gained by early detection and appropriate management. Of the two available surveillance strategies, thyroid ultrasound and neck palpation, neither was shown to be superior. Consequently, a decision aid was formulated to guide the health care provider in counseling the survivor. The recommendations highlight the need for shared decision making regarding whether to undergo surveillance for DTC and in the choice of surveillance modality.

Natural course of HCV infection in childhood cancer survivors

Goals of workTo describe the course of hepatitis C in a cohort of 105 survivors after childhood cancer.Patients and methodsData on chemo/radiotherapy, clinical status, serial alanine aminotransferase (ALT) evaluation, and virological parameters after the end of treatment were collected for each patient. Liver biopsies, when performed, were centrally evaluated by a pathologist.Main resultsAll patients were alive at the end of follow-up and did not show hepatic insufficiency. ALT evaluation along the entire follow-up showed a moderate (87%) or a remarkable (13%) cytolytic pattern. Young age at diagnosis, hematopoietic stem cell transplantation, and duration of infection significantly correlate with a worse hepatic activity. Type of tumor and chemo and/or radiotherapy regimens did not influence the pattern of hepatic cytolysis. Liver biopsy, centrally reviewed in 30% of the cohort, showed one case of cirrhosis and mild fibrosis in 71% of the group. Higher degrees of fibrosis did not seem to be related to any exposition to chemo/radiotherapy but correlated significantly with the more remarkable cytolytic course.ConclusionsThe outcome of hepatitis C in our patients is comparable to the one described in European cohorts of adult cancer survivors and perinatally infected subjects. Nevertheless, progression to high degrees of hepatic damage has to be monitored by a careful follow-up.

Nachsorge im Erwachsenenalter

ZusammenfassungKrebserkrankungen und ihre Therapie können viele Jahre nach Therapieende zu chronischen oder spät auftretenden Problemen führen. Die bisher bekannten wichtigsten Spätfolgen im Erwachsenenalter sind durch Radio- und Chemotherapie mit Alkylanzien bedingte Zweittumoren, endokrinologische Spätfolgen sowie kardiovaskuläre Probleme, die durch eine häufig auftretende Adipositas und konsekutives metabolisches Syndrom verstärkt werden. Das Wissen um die Spätfolgen im Erwachsenenalter geht mit neuen Aufgaben einher, die nur in Zusammenarbeit von pädiatrischen Onkologen und Erwachsenenspezialisten verschiedener Disziplinen angegangen werden können. Dafür benötigen wir wissenschaftlich erhobene Daten als Grundlagen, andererseits müssen wir neue Betreuungsmodelle entwickeln, die prinzipiell die gleichen Aufgaben haben, aber in ihrer Detailstruktur sehr ortsabhängig sind. Als wesentliche Grundlage für eine gute Versorgung im Erwachsenenalter hat sich eine gute Information der Patienten selbst und der sie betreuenden Hausärzte erwiesen.AbstractCancer and its treatment can lead to chronic or late occurring problems many years after therapy. The most important known late effects include second primary malignancies, caused by radiotherapy or alkylating agents, cardiomyopathy, endocrine late effects, and cardiovascular problems which are often exacerbated by obesity and subsequent metabolic syndrome. Knowledge about late effects in adulthood brings new problems, which can only be addressed in cooperation between pediatric oncologists and adult specialists in various disciplines. While scientifically gathered information is required to form a basis, we also need to develop new treatment models, which in principal have the same goals, but which are wholly location-specific. Ensuring that both patients and their treating physicians are well informed has been proven to form a strong basis for good treatment in adulthood.