Paediatric cancer in Malta

The illness we call cancer has extraordinarily diverse features including its causation, underlying pathology, clinical symptoms, therapeutic response, and outcome or chance of cure. It is a collection of many disorders of cell and tissue function that have one special biological property in common - the territorial expansion of a mutant clone. Cancer develops as a chromosomal gene disorder in single cells. That cell or those cells with the right mutation or combination of mutations will form the winning clone that will acquire the ability to proliferate uncontrollably and spread locally and distantly in spite of the body’s natural defence mechanisms against such rogue cells.peer-reviewe

Griscelli syndrome : a rare neonatal syndrome

Griscelli syndrome was first described by Griscelli and Siccardi in 1978 in a hospital in Paris. It is a rare autosomal recessive disorder resulting in pigmentary dilution of the skin and hair, presence of large clumps of pigment in hair shafts and an accumulation of melanosomes in melanocytes.2 It results in silver-grey hair along with variable cellular immunodeficiency or severe neurological impairment or both. The condition is rare in all countries and up to January 2003 only 60 cases had been described in the world medical literature. In most cases diagnosis occurs between the ages of 4 months to 7 years. The boy discussed here had silvery hair, eyebrows and eyelashes and was admitted at the age of five months to hospital with fever, hepatosplenomegaly and pancytopaenia.peer-reviewe

The cost of blood in paediatric oncology patients

Introduction: Consumption of blood products is significant aiming to treat low cell counts and improve quality of life however 9% to 44% of the total consumption in centres abroad are unjustified. We reviewed thresholds at which blood products were administered and costs incurred by administering blood products at the local paediatric oncology ward at Mater Dei hospital and assessed whether they were inkeeping with local guidelines. Methods: Patient files were analyzed retrospectively for demographics, disease, type and amount of blood products used from January to May 2013. The costs involved were obtained from the Blood Bank at Mater Dei Hospital. The standards used were the protocol by HBB regarding administration on KURA and ‘Supportive care protocols’ in paediatric oncology and haematology. Results: Nine children were given blood products. Red cell products (RCP) use ranged from 0-10 units. and platelets derived products ranged 0- 12 units per patient. haemoglobin levels and platelet counts before transfusions ranged from 3.1 to 8.6g/dL and 9 to 60x109/L respectively. The total cost for the department was €17,950 while the total amount spent for tests done prior to ordering products was €3,276 out of 22 RCP requests for transfusion only once were RCPs transfused above the standard 7g/dL. Platelets were requested 26 times. Documentation regarding the reason for administration was lacking in patient files. Conclusion: The use of blood products is dependent on patient needs and is not influenced by prices. Thresholds at which platelets and RCP are administered vary according to the clinical scenario. Rising costs and shrinking donor pools require blood products to be used judiciously.peer-reviewe

The state of research into children with cancer across Europe : new policies for a new decade

Overcoming childhood cancers is critically dependent on the state of research. Understanding how, with whom and what the research community is doing with childhood cancers is essential for ensuring the evidence-based policies at national and European level to support children, their families and researchers. As part of the European Union funded EUROCANCERCOMS project to study and integrate cancer communications across Europe, we have carried out new research into the state of research in childhood cancers. We are very grateful for all the support we have received from colleagues in the European paediatric oncology community, and in particular from Edel Fitzgerald and Samira Essiaf from the SIOP Europe office. This report and the evidence-based policies that arise from it come at a important junction for Europe and its Member States. They provide a timely reminder that research into childhood cancers is critical and needs sustainable long-term support.peer-reviewe

Development and use of an innovative Gap Finding Tool to create a Pharmaceutical Care Model within a paediatric oncology setting.

INTRODUCTION: A paediatric cancer ward is a setting where pharmacists participate in direct patient care, acting as coordinators between the patient, caregivers and healthcare professionals. The aim of the study was to develop a Gap Finding Tool to support the setting up of a pharmaceutical care model at a Paediatric-Adolescent Cancer Ward. METHODS: The Standards of Practice for Clinical Pharmacy Services by the Society of Hospital Pharmacists of Australia Committee of Specialty Practice in Clinical Pharmacy (2013), the American College of Clinical Pharmacy (2014) and the European Association of Hospital Pharmacists (2014) were used to compile the Gap Finding Tool. The developed Tool was tested for content validity by a panel of experts and subsequently implemented over 2 months. RESULTS: The Gap Finding Tool comprised of nine sections with an average of eight statements each about pharmacy services that should be provided at ward level. For each statement, the rater indicates whether these contributions are provided. When the Tool was implemented at the Paediatric-Adolescent Cancer Ward, four major gaps were identified, namely, absence of a clinical pharmacist, lack of medicines information, vetting of chemotherapy prescriptions by pharmacist with limited access to patient data and lack of pharmacist-input on medicines availability. Processes requiring optimisation included discharge medication advice and documentation processes. CONCLUSION: The developed Gap Finding Tool is an innovative tool which is versatile and can be used in ward or ambulatory clinical settings to identify gaps in pharmaceutical processes and services and compare national or regional practices to international standards

Diagnostics and treatment of diffuse intrinsic pontine glioma : where do we stand?

INTRODUCTION: Diffuse intrinsic pontine glioma (DIPG) is a rare clinically, neuro-radiologically, and molecularly defined malignancy of the brainstem with a median overall survival of approximately 11 months. Our aim is to evaluate the current tendency for its treatment in Europe in order to develop (inter)national consensus guidelines. METHODS: Healthcare professionals specialized in DIPG were asked to fill in an online survey with questions regarding usual treatment strategies at diagnosis and at disease progression in their countries and/or their centers, respectively. RESULTS: Seventy-four healthcare professionals responded to the survey, of which 87.8% were pediatric oncologists. Only 13.5% of the respondents biopsy all of their patients, 41.9% biopsy their patients infrequently. More than half of the respondents (54.1%) treated their patients with radiotherapy only at diagnosis, whereas 44.6% preferred radiotherapy combined with chemotherapy. When the disease progresses, treatment strategies became even more diverse, and the tendency for no treatment increased from 1.4% at diagnosis to 77.0% after second progression. 36.5% of the healthcare professionals treat children younger than 3 years differently than older children at diagnosis. This percentage decreased, when the disease progresses. Most of the participants (51.4%) included less than 25% of their patients in clinical trials. CONCLUSION: This survey demonstrates a large heterogeneity of treatment regimens, especially at disease progression. We emphasize the need for international consensus guidelines for the treatment of DIPG, possible by more collaborative clinical trials

Diagnostics and treatment of diffuse intrinsic pontine glioma: where do we stand?

INTRODUCTION: Diffuse intrinsic pontine glioma (DIPG) is a rare clinically, neuro-radiologically, and molecularly defined malignancy of the brainstem with a median overall survival of approximately 11 months. Our aim is to evaluate the current tendency for its treatment in Europe in order to develop (inter)national consensus guidelines. METHODS: Healthcare professionals specialized in DIPG were asked to fill in an online survey with questions regarding usual treatment strategies at diagnosis and at disease progression in their countries and/or their centers, respectively. RESULTS: Seventy-four healthcare professionals responded to the survey, of which 87.8% were pediatric oncologists. Only 13.5% of the respondents biopsy all of their patients, 41.9% biopsy their patients infrequently. More than half of the respondents (54.1%) treated their patients with radiotherapy only at diagnosis, whereas 44.6% preferred radiotherapy combined with chemotherapy. When the disease progresses, treatment strategies became even more diverse, and the tendency for no treatment increased from 1.4% at diagnosis to 77.0% after second progression. 36.5% of the healthcare professionals treat children younger than 3 years differently than older children at diagnosis. This percentage decreased, when the disease progresses. Most of the participants (51.4%) included less than 25% of their patients in clinical trials. CONCLUSION: This survey demonstrates a large heterogeneity of treatment regimens, especially at disease progression. We emphasize the need for international consensus guidelines for the treatment of DIPG, possible by more collaborative clinical trials.status: publishe

Diagnostics and treatment of diffuse intrinsic pontine glioma: where do we stand?

INTRODUCTION: Diffuse intrinsic pontine glioma (DIPG) is a rare clinically, neuro-radiologically, and molecularly defined malignancy of the brainstem with a median overall survival of approximately 11 months. Our aim is to evaluate the current tendency for its treatment in Europe in order to develop (inter)national consensus guidelines. METHODS: Healthcare professionals specialized in DIPG were asked to fill in an online survey with questions regarding usual treatment strategies at diagnosis and at disease progression in their countries and/or their centers, respectively. RESULTS: Seventy-four healthcare professionals responded to the survey, of which 87.8% were pediatric oncologists. Only 13.5% of the respondents biopsy all of their patients, 41.9% biopsy their patients infrequently. More than half of the respondents (54.1%) treated their patients with radiotherapy only at diagnosis, whereas 44.6% preferred radiotherapy combined with chemotherapy. When the disease progresses, treatment strategies became even more diverse, and the tendency for no treatment increased from 1.4% at diagnosis to 77.0% after second progression. 36.5% of the healthcare professionals treat children younger than 3 years differently than older children at diagnosis. This percentage decreased, when the disease progresses. Most of the participants (51.4%) included less than 25% of their patients in clinical trials. CONCLUSION: This survey demonstrates a large heterogeneity of treatment regimens, especially at disease progression. We emphasize the need for international consensus guidelines for the treatment of DIPG, possible by more collaborative clinical trials

Impact of the COVID-19 pandemic on patients with paediatric cancer in low-income, middle-income and high-income countries: a multicentre, international, observational cohort study

OBJECTIVES: Paediatric cancer is a leading cause of death for children. Children in low-income and middle-income countries (LMICs) were four times more likely to die than children in high-income countries (HICs). This study aimed to test the hypothesis that the COVID-19 pandemic had affected the delivery of healthcare services worldwide, and exacerbated the disparity in paediatric cancer outcomes between LMICs and HICs. DESIGN: A multicentre, international, collaborative cohort study. SETTING: 91 hospitals and cancer centres in 39 countries providing cancer treatment to paediatric patients between March and December 2020. PARTICIPANTS: Patients were included if they were under the age of 18 years, and newly diagnosed with or undergoing active cancer treatment for Acute lymphoblastic leukaemia, non-Hodgkin's lymphoma, Hodgkin lymphoma, Wilms' tumour, sarcoma, retinoblastoma, gliomas, medulloblastomas or neuroblastomas, in keeping with the WHO Global Initiative for Childhood Cancer. MAIN OUTCOME MEASURE: All-cause mortality at 30 days and 90 days. RESULTS: 1660 patients were recruited. 219 children had changes to their treatment due to the pandemic. Patients in LMICs were primarily affected (n=182/219, 83.1%). Relative to patients with paediatric cancer in HICs, patients with paediatric cancer in LMICs had 12.1 (95% CI 2.93 to 50.3) and 7.9 (95% CI 3.2 to 19.7) times the odds of death at 30 days and 90 days, respectively, after presentation during the COVID-19 pandemic (p<0.001). After adjusting for confounders, patients with paediatric cancer in LMICs had 15.6 (95% CI 3.7 to 65.8) times the odds of death at 30 days (p<0.001). CONCLUSIONS: The COVID-19 pandemic has affected paediatric oncology service provision. It has disproportionately affected patients in LMICs, highlighting and compounding existing disparities in healthcare systems globally that need addressing urgently. However, many patients with paediatric cancer continued to receive their normal standard of care. This speaks to the adaptability and resilience of healthcare systems and healthcare workers globally