Clinical trial NCT04037189: Treatment of Leukemia and Lymphoma in Children With Ataxia Telangiectasia
Official title: Treatment of Leukemia and Lymphoma in Children With Ataxia Telangiectasia- A Retrospective Study
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Ataxia Telangiectasia
Leukemia
Lymphoma
Lead sponsor: Rabin Medical Center
Sponsor class: OTHER
Collaborators: name: Israeli Society for Pediatric Hematology-Oncology
name: International BFM Study Group
First posted: 2019-07-30
Enrollment: 250
Sex: ALL
Minimum age (years): 0
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Children and young adults diagnosed with ataxia telangiectasia and leukemia or lymphoma

Brief summary: Ataxia telangiectasia (A-T) is a multisystem disease with diverse manifestations, including progressive neurodegeneration, immunodeficiency, respiratory disease, and genomic instability. One of the most important features of A-T is the increased predisposition to cancer, especially to lymphoid malignancies. Patients with A-T are generally excluded from collaborative clinical trials, their treatment outcomes and toxicity profiles have rarely been reported, and little is currently known concerning the treatment intensity required to provide a reasonable balance between efficacy and toxicity. The aims of this study are to build a large international de-identified database of children with A-T treated for leukemia and lymphoma, to investigate epidemiology and outcome of treatment, toxicity profiles and risk factors which impact outcome, in order to eventually enable the generation of data-based treatment recommendations for this population.

Detailed description: Ataxia telangiectasia (A-T) is a multisystem disease with diverse manifestations, including progressive neurodegeneration, immunodeficiency, respiratory disease, and genomic instability. A-T is caused by biallelic mutations in the ATM gene, a major activator of the cellular response to DNA double strand breaks. One of the most important features of A-T is the increased predisposition to cancer. Lymphoid malignancies represent the majority of cancers. The treatment of cancer in children with A-T is extremely challenging, due to severe co-morbidities and a significantly increased risk of cancer therapy-related toxicities. Patients with A-T are generally excluded from collaborative clinical trials, their treatment outcomes and toxicity profiles have rarely been reported, and little is currently known concerning the treatment intensity required to provide a reasonable balance between efficacy and toxicity. The optimal treatment approach is controversial; some advocate treatment by standard chemotherapeutic protocols, while others advise initial protocol modifications with chemotherapy dose reductions. Due to the rarity of this disorder, there is an unmet need for an international collaboration for data collection concerning treatment, toxicity and outcome in children with cancer and A-T. Data will be collected from patient files, including patient characteristics and history, AT manifestations, malignancy characteristics, treatment, chemotherapy doses, treatment response, toxicity and outcome.

The aims of the study are to build a large international de-identified database of children with A-T treated for leukemia and lymphoma, to investigate epidemiology and outcome of treatment, toxicity profiles and risk factors which impact outcome, in order to eventually enable the generation of data-based treatment recommendations for this population.

This study will not involve the use of specimens or participant contact. All the data required have already been collected during the treatment of the participants, and is available in patient records.

Eligibility criteria: Inclusion Criteria:

* Individuals diagnosed with ataxia telangiectasia and leukemia or lymphoma
* Age 0-21

Exclusion Criteria:

-Age greater than 21 years

Primary outcomes:
measure: Event-free survival
timeFrame: 5 years
measure: Overall survival
timeFrame: 5 years
measure: Cumulative incidence of relapse
timeFrame: 5 years
measure: Cumulative incidence of treatment-related mortality
timeFrame: 2 years
measure: Cumulative incidence of second malignancies
timeFrame: 5 years
Secondary outcomes:
measure: Cause and timing of death
timeFrame: 5 years
measure: Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
timeFrame: 2 years
Other outcomes:
Locations:
city: Petah Tikva
country: Israel
facility: Schneider Children's Medical Center
Study officials:
affiliation: Schneider Children's Medical Center, Israel
name: Sarah Elitzur, MD
role: PRINCIPAL_INVESTIGATOR
References: