Clinical trial NCT03971305: A Prospective Multicenters Clinical Cohort Study of Stratified Treatment of Chinese Children With Systemic ALK(+) ALCL
Official title: A Prospective Multicenters Clinical Cohort Study of the Efficacy and Safety of Stratified Risk Factors for Treatment of Chinese Children With Systemic ALK-positive Anaplastic Large Cell Lymphoma
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: MRD
MDD
Lymphoma, Nonhodgkin
Anaplastic Lymphoma
Pediatric Cancer
Lead sponsor: Sun Yat-sen University
Sponsor class: OTHER
First posted: 2019-06-03
Enrollment: 300
Sex: ALL
Minimum age (years): 1
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Systemic ALK-positive anaplastic large cell lymphoma patients # age at diagnosis \< 18 years.

Brief summary: With the development of molecular biology and precise medical treatment, new challenges have been raised in the diagnosis and treatment of non-Hodgkin lymphoma (NHL) in children. In recent years, the criteria for clinical staging and efficacy evaluation of NHL in children have been updated. Studies in Germany and the United States have shown that pathological types of systemic anaplastic large cell lymphoma (ALCL) in children and adolescents, minimal disseminated disease (MDD) in peripheral blood or bone marrow and minimal residual disease (MRD) are significantly associated with prognosis, suggesting that these factors need to be combined in risk stratification of ALCL patients. Recent studies have also suggested that PET/CT is helpful in evaluating residual lesions in patients with lymphoma after chemotherapy. In order to keep pace with the times in the diagnosis, clinical staging, risk stratification, efficacy evaluation and treatment of NHL in children. We adjusted the original NHL-BFM-90/95 regimen, mainly in the aspects of clinical staging, efficacy evaluation, risk stratification and treatment regimen，etc.

Detailed description: Research purpose:

1. To study the efficacy and safety of SCCCG-ALCL-2017 regimen in children with systemic ALK-positive anaplastic large cell lymphoma.
2. To explore the correlation between MDD or MRD in peripheral blood or bone marrow and the treat response and survival.
3. To explore the feasibility of risk stratification combined with adverse pathological types, dangerous organ invasion and MDD.
4. To investigate the effect of vinblastine maintenance chemotherapy on survival of patients with MRD-positive in peripheral blood after treatment.

Eligibility criteria: Inclusion Criteria:

1. Age \< 18 years old
2. Pathologically confirmed systemic ALK-positive anaplastic large cell lymphoma
3. Newly diagnosed patients
4. Informed consent of guardian of children patients

Exclusion Criteria:

1. Secondary immunodeficiency disease
2. Second neoplasm
3. Primary cutaneous anaplastic large cell lymphoma
4. Recurrent and progressive patients.

Primary outcomes:
measure: Event-free survival (EFS)
timeFrame: through study completion, maximal eight years
Secondary outcomes:
measure: Overall survival (OS)
timeFrame: through study completion, maximal eight years
measure: Relapse-free survival (RFS)
timeFrame: through study completion, maximal eight years
measure: Response rate (RR)
timeFrame: on an average 3 weeks after finish of treatment
measure: Adverse event rate
timeFrame: from the first day of protocol defined treatment until two years after start of protocol defined treatment
Other outcomes:
Locations:
city: Guangzhou
country: China
facility: Sun Yat-sen University Cancer Center
state: Guangdong
Study officials:
affiliation: Sun Yat-sen University
name: Sun Xiaofei
role: STUDY_DIRECTOR
References:
citation: Burkhardt B, Zimmermann M, Oschlies I, Niggli F, Mann G, Parwaresch R, Riehm H, Schrappe M, Reiter A; BFM Group. The impact of age and gender on biology, clinical features and treatment outcome of non-Hodgkin lymphoma in childhood and adolescence. Br J Haematol. 2005 Oct;131(1):39-49. doi: 10.1111/j.1365-2141.2005.05735.x.
citation: Lamant L, McCarthy K, d'Amore E, Klapper W, Nakagawa A, Fraga M, Maldyk J, Simonitsch-Klupp I, Oschlies I, Delsol G, Mauguen A, Brugieres L, Le Deley MC. Prognostic impact of morphologic and phenotypic features of childhood ALK-positive anaplastic large-cell lymphoma: results of the ALCL99 study. J Clin Oncol. 2011 Dec 10;29(35):4669-76. doi: 10.1200/JCO.2011.36.5411. Epub 2011 Nov 14.
citation: Damm-Welk C, Mussolin L, Zimmermann M, Pillon M, Klapper W, Oschlies I, d'Amore ES, Reiter A, Woessmann W, Rosolen A. Early assessment of minimal residual disease identifies patients at very high relapse risk in NPM-ALK-positive anaplastic large-cell lymphoma. Blood. 2014 Jan 16;123(3):334-7. doi: 10.1182/blood-2013-09-526202. Epub 2013 Dec 2.
citation: Sun X, Zhen Z, Lin S, Zhu J, Wang J, Lu S, Chen Y, Zhang F, Sun F, Li P. Treatment outcome of Chinese children with anaplastic large cell lymphoma by using a modified B-NHL-BFM-90 protocol. Pediatr Hematol Oncol. 2014 Sep;31(6):518-27. doi: 10.3109/08880018.2014.939793. Epub 2014 Aug 12.
citation: Le Deley MC, Rosolen A, Williams DM, Horibe K, Wrobel G, Attarbaschi A, Zsiros J, Uyttebroeck A, Marky IM, Lamant L, Woessmann W, Pillon M, Hobson R, Mauguen A, Reiter A, Brugieres L. Vinblastine in children and adolescents with high-risk anaplastic large-cell lymphoma: results of the randomized ALCL99-vinblastine trial. J Clin Oncol. 2010 Sep 1;28(25):3987-93. doi: 10.1200/JCO.2010.28.5999. Epub 2010 Aug 2.