Neoplasms

Blood and Lymph Conditions

All Conditions

Rare Diseases

Leukemia

Myeloproliferative Disorders

Primary Myelofibrosis

Bone Marrow Diseases

Hematologic Diseases

Acute Graft Versus Host Disease

Chronic Myeloproliferative Disorders

Tiziano Barbui, MD

The Fondazione per la Ricerca Ospedale di Bergamo (FROM) decided to propose and promote the completion of ERNEST registry: the aim of the project is to obtain up-to-date estimates of clinical outcome of these patients in primary and secondary Myelofibrosis that have been included in the 'original' ERNEST study. The last was conducted between 2012 and beginning of 2015 including 1292 patients with Myelofibrosis, but it was interrupted in 2015 because of bankruptcy of the previous sponsor.

Follow-up of patients already been registered in the ERNEST registry resulting alive and/or in active surveillance at November 2014 (Blood 2014 124:1849) will be updated retrospectively until December 2020. Data collection will be centralized at Fondazione per la Ricerca Ospedale di Bergamo (FROM), and performed by a web-based and Electronic Data Capture (EDC) system through ad hoc electronic case report forms (e-CRFs). The e-CRFs system will be pre-filled with pre-existing data collected from cases already entered in the registry. Information on therapies received and outcome events occurred (in term of death, evolution to acute leukemia, secondary cancer and cardiovascular complications) until December 2020 will be recorded by authorized participating investigators.

European Registry for Myeloproliferative Neoplasms (MPNs) - Update of ERNEST Study

Occurrence of AL from diagnosis to last observation

Occurrence of ST from diagnosis to last observation

Occurrence of events from diagnosis to last observation

FROM- Fondazione per la Ricerca Ospedale di Bergamo- ETS

Urinary Tract, Sexual Organs, and Pregnancy Conditions

Diseases and Abnormalities at or Before Birth

Infertility

Neoplasms, Second Primary

Genetic Diseases, Inborn

Genital Diseases

Urogenital Diseases

No intervention ; observational

Renske Fles, Phd

Renske Fles

Olga Husson, Phd

Winette van de Graaf, Prof

Rationale: Childhood cancer survivorship attracts attention globally, because successes in treatment have led to increasing number of survivors who reach adulthood, in which survivorship issues affecting health-related quality of life (HRQoL) become prominent. Most paediatric patients are treated intensively with irradiation and/or chemotherapy, which put them at risk for early and/or late adverse medical and psychosocial events. In contrast, much less is known about adolescent and young adult (AYA) cancer patients, diagnosed between 18-39 years, who, with an 80% chance to survive, also have a long life ahead. AYA cancer patients, much more than children, suffer from delay in diagnosis, lack of centralization of care, ageadjusted expertise, and AYA follow-up care. AYAs typically present with a rare tumour: either with a paediatric malignancy (e.g. acute lymphoblastic leukaemia, paediatric brain tumours), a more typical tumour of AYA age (e.g. Hodgkin's disease, germ cell cancer, melanoma, thyroid cancer) or with an adult tumour at unusual young age (e.g. gastrointestinal, lung, breast carcinomas). Next to these differences in epidemiology, the tumour biology, developmental challenges (e.g. forming relationships, becoming financially independent, having children) and treatment regimens differ between AYAs and children, and therefore findings derived from childhood cancer survivors cannot be extrapolated to AYAs. Furthermore, novel treatments with targeted agents or immunotherapy are more likely to be administrated to AYAs compared to children. Finally, a rare group of incurable AYA cancer patients will survive for many years, for whom health outcome and supportive care intervention data are lacking.

Globally, so far, the identification of AYA cancer patient subgroups that might be more susceptible to poor health outcomes has not been systematically addressed. The role of sociodemographic and treatment-associated risks, external exposures (e.g. lifestyle) and host factors (e.g. genetic, biological, physiological); or combinations of influences for impaired (agespecific) health outcomes, remains largely unknown. Understanding who is at risk and why will support the development of evidence-based AYA prevention, treatment and supportive care programs and guidelines, in co-creation with AYA cancer patients.

Objective: To examine the prevalence, risk factors and mechanisms of impaired health outcomes (short- and long-term medical and psychosocial effects and late effects) over time among a population-based sample of AYA cancer patients.

Study design: Prospective, observational cohort study Study population: All AYAs diagnosed (18-39 years at primary diagnosis) with cancer (any type) within the first 3 months after diagnosis (eligibility window of 1 month to ensure all eligible AYA cancer patients can be included) in one of the participating centres (or treated in one of these centres) in The Netherlands.

Main study parameters/endpoints: The main outcomes are medical (e.g. second tumour; survival; fertility) and psychosocial (e.g. distress) health outcomes. Other study parameters (covariates/moderators/mediators) are characteristics of the individual (e.g. age, sex, cultural background, partner status, educational level, occupation, tumour type, disease stage, body composition, comorbid conditions, coping style), characteristics of the environment (e.g. cancer treatment, lifestyle), and genetic and biological factors (e.g. family history of cancer, stress and inflammation markers (e.g. cortisol, IL-6), microbiome).

Nature and extent of the burden and risks associated with participation, benefit and group relatedness: On an individual level, patients who participate are asked to complete questionnaires on an annual basis for at least 10 years. All sample collections will take place at three time points: 0-3 months after diagnosis (baseline), 2 and 5 years; except blood for DNA analyses which will only take place at baseline. The collection of blood, hair and faeces at three occasions is minimally invasive and the risks of blood draws, hair and fecal sampling are negligible. All safety measures and procedures will be performed according to local guidelines. Patients will not experience direct benefit from participation in the COMPRAYA study.

By participating, patients will contribute to a better insight in the prevalence of impaired medical and psychosocial (age-specific) health outcomes in AYA and evidence on factors associated with these health outcomes. This will lead to better and more personalized cancer care and supportive care tools for future AYA cancer patients.

The Prospective Observational COMPRAYA Cohort Study

COMPRehensive Assessment of Prevalence, Risk Factors and Mechanisms of Impaired Medical and Psychosocial Health Outcomes Among Adolescents and Young Adults With Cancer: the Prospective Observational COMPRAYA Cohort Study

Linkage to the Netherlands Cancer Registry (NCR) to asses second malignancies.

Linkage will done with the Personal Records Database (BRP) to have information on survival status.

Psychological distress will be assessed at each time point with the Hospital Anxiety and Depression Scale (HADS), with seven items each for assessing symptoms of anxiety and depression. Answers range from 0 to 3 and scores for each subscale are calculated by addition of the items, with a higher score meaning more anxiety or depression.

A questionnaire will be used to asses fertility problems and their wish for children. Linkage will be done to the Netherlands Perinatal Registry (NPR) to receive data about pregnancy and children.

The EORTC QLQ-C30 is a 30-item HRQoL questionnaire consisting of five functional scales (physical, role, cognitive, emotional and social), a global quality of life scales, symptom scales (fatigue, pain, nausea and vomiting) and a number of single items assessing common symptoms (dyspnea, loss of appetite, sleep disturbance, constipation and diarrhea) and perceived financial impact of the disease. After linear transformation, all scales and single item measures range in score from 0-100. A higher score on the functional scales and global QoL means better functioning and HRQoL, whereas a higher score on the symptom scales means more complaints.

The Netherlands Cancer Institute

Immune System Diseases

Precursor Cell Lymphoblastic Leukemia-Lymphoma

Leukemia, Lymphoid

Lymphoma

Neoplasms by Histologic Type

Lymphoproliferative Disorders

Lymphatic Diseases

Immunoproliferative Disorders

Acute Lymphoblastic Leukemia

Lymphoblastic Lymphoma

Childhood Acute Lymphoblastic Leukemia

Lymphosarcoma

Cases of childhood acute lymphoblastic leukemia (ALL) diagnosed between 1980 and 2007 and included in the clinical trials of the participating ALL study groups

Kjeld Schmiegelow, M.D.

Maria G Valsecchi, M.Sci,

Development of a second neoplasm (SMN) during or after therapy for childhood acute lymphoblastic leukemia (ALL) is a rare event generally associated with a poor prognosis. In this international study we analyze subtypes of SMN in relation to their initial leukemia characteristics and treatment, and their subsequent overall survival.

To explore epidemiology, potential risk factors and survival rates of second cancers occurring as the first event in childhood acute lymphoblastic leukemia the involved study groups will collect anonymous data on all such cases diagnosed within the last decades to form a common database with predefined variables comprising the clinical, biological, and cytogenetic characteristics (myeloid neoplasias only) as well as outcome. Furthermore, we will register the clinical, biological, and cytogenetic characteristics of the acute lymphoblastic leukemia as well as type of treatment given.

Second Malignant Neoplasms After Childhood ALL Therapy

Second Malignant Neoplasms After Childhood ALL Therapy; An International Ponte di Legno Study

Pattern of the main groups of SMN and their clinical charactiristics

Overall survival by the main SMN subtypes (myeloid malignancies, brain tumors, lymphomas, sarcomas, carcinomas, others)

Identification of risk factors linked to the interval to SMN, the subtype of SMN, and the survival after. These risk factors are clinical characteristics of ALL or the anti-ALL therapy administered

Nordic Society for Pediatric Hematology and Oncology

European Organisation for Research and Treatment of Cancer - EORTC

Dana-Farber Cancer Institute

ALL-BFM Study Group

Dutch Childhood Oncology Group

St. Jude Children's Research Hospital

United Kingdom Children's Cancer Study Group

Rigshospitalet, Denmark

Professor

Respiratory Tract (Lung and Bronchial) Diseases

Erdheim-Chester Disease

Histiocytosis, Langerhans-Cell

Histiocytosis

Histiocytosis, Sinus

Sarcoma

Histiocytic Sarcoma

Lung Diseases

Lung Diseases, Interstitial

Respiratory Tract Diseases

Histiocytosis, Non-Langerhans-Cell

Histiocytic Disorders, Malignant

Lipogranulomatosis

Langerhans Cell Histiocytosis

Rosai-Dorfman Disease

Polymorphic Reticulosis

Soft Tissue Sarcoma

Non-Langerhans-Cell Histiocytosis

Gaurav Goyal, MD

The purpose of the study is to describe the burden of chronic health conditions, psychological dysfunction, chronic pain, healthcare utilization, worse health-related quality of life, overall mortality, and cause-specific mortality among individuals with histiocytic disorders

Background

Over the last decade, major advances have occurred in histiocytic disorders with the discovery of MAPK-ERK pathway mutations leading to targeted therapeutics using BRAF- and MEK-inhibitors. However, there is a lack of large studies informing the burden of morbidity and mortality among people with histiocytosis. Institutional studies in pediatric LCH suggest that survivors suffer from long-term impairment of health related quality of life, cognitive dysfunction, pituitary dysfunction, and hearing difficulties in 20-50% cases. Studies from the investigative team and others have also shown a high incidence of acute myeloid leukemia and other second primary malignancies in pediatric and adult LCH. Chronic medical conditions may arise as a function of the disease biology or due to cancer therapy, as seen in other hematologic malignancies.

Design

Retrospective cohort study aimed at determining the risk of chronic health conditions and cause-specific mortality in pediatric and adult patients with histiocytic disorders. The study will include patients from the Histiocytosis Association registry and other institutions including UAB (n\~6000). The participants will complete a validated questionnaire capturing details of chronic health conditions, health related quality of life, cognitive/psychological function, and healthcare utilization.

Future directions

The results from our study will be instrumental in formulating follow-up guidelines for histiocytic disorders and developing targeted survivorship programs to improve overall outcomes.

Histiocytic Disorder Follow-up Study

The CTCAE criteria for CHCs distinguishes each acute and chronic condition (including SPMs) in persons with cancer from grades 1 through 4 with unique clinical descriptions of severity (grade 1, mild; grade 2, moderate; grade 3, severe; grade 4, life-threatening/disabling). The main outcome will be the number of distinct CTCAE grade 3-4 conditions among LCH cases compared with unaffected (non-cancer) controls.

Causes of death will be grouped into primary cancer (LCH)-related mortality (PCRM) and non-primary cancer-related mortality (NPCRM - death from all causes other than the index LCH diagnosis) by two independent investigators.

We will build a prediction model that has a good discrimination for predicting CHC, SPMs, and psychological outcomes separately using data from the LCH (case) cohort.

University of Alabama at Birmingham

Principal Investigator

Symptoms and General Pathology

Recurrence

Disease Attributes

Antineoplastic Agents

All Drugs and Chemicals

Dostarlimab

4 intravenous injections maximum of dostarlimab, 500mg, every 3 weeks

Dostarlimab should initiated within 6 months after the end of treatment for FPC.

Jean-Yves BLAY, MD, PhD

Zoé NEVIERE

Xavier DURANDO

Jean-Yves BLAY, MD,PhD

Victor SARRADIN

Thomas PUDLARZ

PredoSTAR is a multicenter, randomized, open-label phase II study proposed to patients at high risk of SPC and in whom the treatment of the FPC does not include immunotherapy. Dostarlimab treatment will be started within 6 months after the completion of treatment for localized FPC (i.e. after the end of last CT, RT cure or surgery with a wash-out period of 4 weeks before to start Dostarlimab). Eligible patients will be randomized (1:1) to receive:

* Arm Dostarlimab : 4 intravenous (IV) injections of dostarlimab, Q3W or
* Arm No treatment

Analysis will be performed according the ITT principe and will include all randomized patients. Incidence of events by arm will be compared assuming a Poisson distribution. Cumulative incidence curves against time will we compared between treatment arms. Follow-up of each patient will be calculated from randomisation to death or last follow-up for alive patients.

PREventing Second Cancers With DOSTARlimab

A Multicenter, Open-label, Randomized Phase II Study Aiming to Assess the Clinical Impact of Dostarlimab on Occurrence of Second Primary Cancer in Patients With Cured Primary Cancer

Second primary invasive cancer must be centrally histologically confirmed

Only for Arm A : any AEs graded using Common Terminology Criteria for Adverse Events (CTCAE) V5.0.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
European Registry for Myeloproliferative Neoplasms (MPNs) - Update of ERNEST Study	NCT04153305	Myelofibrosis		18 Years -	FROM- Fondazione per la Ricerca Ospedale di Bergamo- ETS
The Prospective Observational COMPRAYA Cohort Study	NCT04682470	Second Cancer Survivorship Fertility Issue... Distress, Emoti... Lifestyle Genetic Disease Cancer	No intervention...	18 Years - 39 Years	The Netherlands Cancer Institute
Second Malignant Neoplasms After Childhood ALL Therapy	NCT01476462	Acute Lymphobla...		- 20 Years	Rigshospitalet, Denmark
Histiocytic Disorder Follow-up Study	NCT05915208	Histiocytosis Langerhans Cell... Erdheim-Chester... Rosai Dorfman D... Xanthogranuloma Malignant Histi...		18 Years - 89 Years	University of Alabama at Birmingham
The Prospective Observational COMPRAYA Cohort Study	NCT04682470	Second Cancer Survivorship Fertility Issue... Distress, Emoti... Lifestyle Genetic Disease Cancer	No intervention...	18 Years - 39 Years	The Netherlands Cancer Institute
Second Malignant Neoplasms After Childhood ALL Therapy	NCT01476462	Acute Lymphobla...		- 20 Years	Rigshospitalet, Denmark
PREventing Second Cancers With DOSTARlimab	NCT05855811	Recurrent Cance... Primary Cancer Second Cancer	Dostarlimab	18 Years -	Centre Leon Berard
PREventing Second Cancers With DOSTARlimab	NCT05855811	Recurrent Cance... Primary Cancer Second Cancer	Dostarlimab	18 Years -	Centre Leon Berard

Spots Global Cancer Trial Database for second cancer