ABSTRACT
Objective:
To investigate the
efficacy and
safety of targeted
therapy with
Crizotinib for
children with ALK
gene mutation positive inflammatory myofibroblastic
tumor (IMT).
Methods:
A retrospective
analysis was performed on 4
children with ALK
gene mutation positive IMT admitted to Shanghai
Children′s
Hospital from January 2019 to June 2021.Among them, 3 cases were given the targeted
drug Crizotinib[280 mg/(m 2·
time), q12h] orally, and 1 case was observed after complete
tumor resection to analyze the
efficacy and
adverse drug reactions.
Results:
All 4 cases were
male,
aged from 2 years and 3 months to 11 years and 3 months.The
tumors originated from the
abdominal cavity in 2 cases, the right
orbit in 1 case, and the right
lung in 1 case.Pathological
immunohistochemistry and fluorescence in situ hybridization were both positive for ALK
gene mutation, and complete remission was achieved after comprehensive
treatment.Among them, 3
patients were treated with oral
Crizotinib, and 2
patients were tried to stop taking the
drug for 1 year, relapsed 1 month later, and still achieved complete remission after the second
treatment.The 4 cases were followed up for 8-30 months, and all survived.All the cases showed no
abnormalities in
blood image,
liver and
kidney function, myocardial
enzyme profile, cardiac function,
hearing and
vision, and 2 cases showed prolonged Q-T interval in the
course of
Crizotinib treatment, which could be recovered by temporary withdrawal of
drug, and no abnormality in
electrocardiogram was found in continued
drug use.
Conclusions:
Crizotinib was used to treat ALK
mutation positive IMT, shrink
tumor and consolidate postoperative
treatment, which is a good choice for IMT in
children with difficult surgical resection and refractory
recurrence.