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Targeted Therapy
Source: Childhood Pulmonary Inflammatory Myofibroblastic Tumors Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: December 3, 2024 · Captured 2026-09-09.
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Context: Treatment of Childhood Pulmonary Inflammatory Myofibroblastic Tumors
Patients with unresectable or recurrent tumors may respond to crizotinib if the ALK variant is present and crizotinib administration is followed by complete or incomplete resection. Treatment with ceritinib and entrectinib have also produced objective responses.
Evidence (targeted therapy):
Crizotinib. The U.S. Food and Drug Administration (FDA) approved crizotinib for use in patients aged 1 year and older with unresectable, recurrent, or refractory ALK-positive IMTs.
Crizotinib. The U.S. Food and Drug Administration (FDA) approved crizotinib for use in patients aged 1 year and older with unresectable, recurrent, or refractory ALK-positive IMTs.
One study included 14 patients with IMTs who were treated with crizotinib.[4][Level of evidence C3]
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Crizotinib. The U.S. Food and Drug Administration (FDA) approved crizotinib for use in patients aged 1 year and older with unresectable, recurrent, or refractory ALK-positive IMTs. › One study included 14 patients with IMTs who were treated with crizotinib.[4][Level of evidence C3]
Five patients had complete responses, seven patients had partial responses, and the remaining two patients had stable disease.
Crizotinib. The U.S. Food and Drug Administration (FDA) approved crizotinib for use in patients aged 1 year and older with unresectable, recurrent, or refractory ALK-positive IMTs. › One study included 14 patients with IMTs who were treated with crizotinib.[4][Level of evidence C3]
No patients experienced a relapse when this article was published.
Crizotinib. The U.S. Food and Drug Administration (FDA) approved crizotinib for use in patients aged 1 year and older with unresectable, recurrent, or refractory ALK-positive IMTs.
An extensive review confirmed that crizotinib was effective in children who had IMTs (with various tumor sites).[5]
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Ceritinib. In a multicenter phase I study, seven of ten patients with IMTs had objective responses to ceritinib.[6]
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Entrectinib. In a phase I/II study of entrectinib, two patients with IMTs and ALK fusions experienced a complete response and a partial response, respectively.[7]
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Alectinib. A case report described the successful treatment of a patient with an IMT and a FN1::ALK gene fusion using alectinib, a second-generation ALK inhibitor.[8]
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A retrospective, international, multicenter study analyzed patients younger than 21 years with ROS1-altered IMTs who were enrolled in either the European paediatric Soft Tissue Sarcoma Study Group (EpSSG) NRSTS-2005 study or the Soft Tissue Sarcoma Registry. Primary surgery was recommended if a microscopic radical resection without disfigurement was feasible. Of the 19 patients, 12 received neoadjuvant systemic therapy as first-line treatment (high-dose steroids, n = 2; vinorelbine/vinblastine with methotrexate, n = 6; ROS1 inhibitors, n = 8). With a median follow-up of 2.8 years, seven patients had an event. The 3-year event-free survival rate was 41% (95% CI, 11%–71%), and the overall survival rate was 100%. While many patients in this series received crizotinib, the specific ROS1 inhibitor used for each patient was not specified.[9]
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For more information about the treatment of this tumor, see the Inflammatory myofibroblastic tumor and epithelioid inflammatory myofibroblastic sarcoma section in Childhood Soft Tissue Sarcoma Treatment.
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Preserved source evidence · Not medical advice