Ontology highlight
ABSTRACT: Rationale
Rhabdomyosarcoma (RMS) is a common soft tissue sarcoma in children with high malignancy. The prognosis of refractory recurrent RMS is extremely poor, and the 5-year survival rate is less than 20%.Patient concerns
We reported a 2-year-old male patient with RMS who underwent 3 operations and 2 recurrences while being treated with regular multidisciplinary therapy.Diagnoses
A diagnosis of embryonal rhabdomyosarcoma with primary bladder (IIIa, TNM stage 2, and medium risk group) was made.Interventions
After repeated recurrence, the patient was treated with chimeric antigen receptor T (CAR-T) cells, which had a safety mechanism and specifically bound the CD56 antigen in the fourth generation.Outcomes
The process of CAR-T cell transfusion was smooth, and there were no significant cytokine release syndrome manifestations after reinfusion. The patient was in complete remission at last follow-up visit after 3.5 years.Conclusion
CD56-CAR-T cell therapy is a safe and effective approach and may be an option for children with solid tumors who are nonresponsive to conventional radiotherapy and chemotherapy, or are unsuitable for hematopoietic stem cell transplantation.
SUBMITTER: Jiang C
PROVIDER: S-EPMC6824760 | biostudies-literature | 2019 Oct
REPOSITORIES: biostudies-literature
Jiang Chiyi C Zhao Wen W Qin Maoquan M Jin Mei M Chang Lungji L Ma Xiaoli X
Medicine 20191001 43
<h4>Rationale</h4>Rhabdomyosarcoma (RMS) is a common soft tissue sarcoma in children with high malignancy. The prognosis of refractory recurrent RMS is extremely poor, and the 5-year survival rate is less than 20%.<h4>Patient concerns</h4>We reported a 2-year-old male patient with RMS who underwent 3 operations and 2 recurrences while being treated with regular multidisciplinary therapy.<h4>Diagnoses</h4>A diagnosis of embryonal rhabdomyosarcoma with primary bladder (IIIa, TNM stage 2, and mediu ...[more]