Alveolar Rhabdomyosarcoma

A form of RHABDOMYOSARCOMA occurring mainly in adolescents and young adults, affecting muscles of the extremities, trunk, orbital region, etc. It is extremely malignant, metastasizing widely at an early stage. Few cures have been achieved and the prognosis is poor. "Alveolar" refers to its microscopic appearance simulating the cells of the respiratory alveolus. (Holland et al., Cancer Medicine, 3d ed, p2188)

Also Known As:

Networked: 378 relevant articles (13 outcomes, 23 trials/studies)

Relationship Network

Disease Context: Research Results

Related Diseases

1.	Neoplasms (Cancer)
2.	Ewing Sarcoma (Sarcoma, Ewing)
3.	Embryonal Rhabdomyosarcoma
4.	Neuroblastoma
5.	Sarcoma (Soft Tissue Sarcoma)

Experts

1.	Schäfer, Beat W: 11 articles (01/2021 - 08/2004)
2.	Wachtel, Marco: 11 articles (01/2021 - 08/2004)
3.	Barr, Frederic G: 10 articles (01/2021 - 08/2004)
4.	Keller, Charles: 10 articles (06/2019 - 10/2005)
5.	Chen, Taosheng: 6 articles (01/2021 - 11/2009)
6.	Houghton, Peter J: 6 articles (01/2019 - 03/2008)
7.	Koscielniak, Ewa: 5 articles (01/2022 - 01/2012)
8.	Helman, Lee J: 5 articles (01/2020 - 03/2002)
9.	Kurmasheva, Raushan T: 5 articles (01/2019 - 04/2005)
10.	Hollenbach, Andrew D: 5 articles (10/2017 - 10/2007)

Drugs and Biologics

Drugs and Important Biological Agents (IBA) related to Alveolar Rhabdomyosarcoma:

1.	DNA (Deoxyribonucleic Acid)IBA 10/01/2001 - "Alveolar rhabdomyosarcoma (ARMS), a paediatric soft tissue malignant tumour, is characterized by the recurrent translocation t(2;13)(q35;q14) that fuses the 5' DNA binding domain-encoding sequences of the Pax3 gene with the 3' sequences of the FKHR gene. " 12/01/1995 - "The FKHR gene, which contains a forkhead DNA-binding motif, is fused to either PAX3 or PAX7 by the t(2;13) or t(1;13) translocation in alveolar rhabdomyosarcoma,respectively. " 01/01/2019 - "Early detection of the PAX3-FOXO1 fusion gene in circulating tumor-derived DNA in a case of alveolar rhabdomyosarcoma." 01/01/2001 - "We have demonstrated that synthetic repressors combining PAX3 DNA binding domains with different repression domains, KRAB or SNAG, are able to specifically inhibit malignant growth and suppress tumorigenesis in alveolar rhabdomyosarcoma tumor cells transformed by the translocation-derived chimeric transcriptional activator, PAX3-FKHR. " 07/01/2000 - "The t(2;13) chromosomal translocation in alveolar rhabdomyosarcoma tumors (ARMS) creates an oncogenic transcriptional activator by fusion of PAX3 DNA binding motifs to a COOH-terminal activation domain derived from the FKHR gene. "
2.	Proteins (Proteins, Gene)FDA Link 01/01/2021 - "Meanwhile, in alveolar rhabdomyosarcoma (ARMS), PAX3-FOXO1 fusion protein is associated with increased aggressiveness and poor prognosis. " 01/01/2019 - "OLIG2 is a marker of the fusion protein-driven neurodevelopmental transcriptional signature in alveolar rhabdomyosarcoma." 12/01/2018 - "Alveolar rhabdomyosarcoma (ARMS) patients have a poor prognosis, and this is primarily due to overexpression of the oncogenic fusion protein PAX3-FOXO1. " 08/07/2014 - "The Polycomb group (PcG) protein EZH2 supports the survival of PAX3-FOXO1 alveolar rhabdomyosarcoma by repressing FBXO32 (Atrogin1/MAFbx)." 01/31/2013 - "Alveolar rhabdomyosarcoma-associated proteins PAX3/FOXO1A and PAX7/FOXO1A suppress the transcriptional activity of MyoD-target genes in muscle stem cells."
3.	MyogeninIBA 05/01/2021 - "Correction: A human Myogenin promoter modified to be highly active in alveolar rhabdomyosarcoma drives an effective suicide gene therapy." 01/01/2021 - "A human Myogenin promoter modified to be highly active in alveolar rhabdomyosarcoma drives an effective suicide gene therapy." 06/01/2013 - "Compared with ALK-negative alveolar rhabdomyosarcomas, ALK-positive ones are presented with metastatic spread more frequently and showed a greater extent of myogenin reactivity. " 02/27/2014 - "A paradoxical feature of alveolar rhabdomyosarcoma (ARMS), a prevalent soft tissue sarcoma in children arising from cells with a myogenic phenotype, is the inability of these cells to undergo terminal differentiation despite the expression of MYOGENIN. " 01/01/2013 - "However, the recognition of new variants of embryonal rhabdomyosarcoma (ERMS), shifts in the diagnostic criteria of alveolar rhabdomyosarcoma (ARMS), the increasing use of myogenin immunohistochemistry and recognition of the distinct biologic properties associated with fusion status all raised questions about the continued use of this classification system in the diagnosis and treatment of patients with RMS. "
4.	CurcuminIBA 01/01/2013 - "These data support testing of curcumin as a radiosensitizer for the clinical treatment of alveolar rhabdomyosarcoma. " 01/01/2013 - "In this study, we have demonstrated that inhibiting radiation-induced NF-κΒ activity by either pharmacologic (curcumin) or genetic (SR-IκBα) means significantly enhanced the efficacy of radiation therapy in the treatment of alveolar rhabdomyosarcoma cells and xenografts. "
5.	DesminIBA 01/01/2021 - "Here, we present a case of a 33-year-old male presented with a poorly differentiated desmin positive alveolar rhabdomyosarcoma in the left arm. " 11/01/2011 - "A diagnosis of alveolar rhabdomyosarcoma (RMS) was reached via histopathology and confirmed with positive immunohistochemical staining for desmin. " 08/01/1982 - "The results suggest that demonstration of desmin may be a helpful adjunct tool in the diagnosis of poorly differentiated alveolar rhabdomyosarcomas." 05/01/2008 - "The new immunohistochemical markers desmin and myogenin in combination with molecular biological detection of specific translocations in alveolar rhabdomyosarcoma improved diagnostic capacities. " 01/01/2020 - "The Immunohistochemical analysis revealed intense positivity for myogenin and desmin, favoring the diagnosis of alveolar rhabdomyosarcoma. "
6.	MethyltransferasesIBA 01/01/2018 - "Biopsy results suggested alveolar rhabdomyosarcoma bearing a methylated O6-methylguanine-DNA methyltransferase (MGMT) gene promoter. " 09/24/1999 - "Compared to normal skeletal muscle, the average increase of DNA methyltransferase expression was 6.7-fold (6.7 +/-()0.96) in the embryonal tumors and 3.7-fold (3.7 +/- 0.46) in the alveolar rhabdomyosarcomas. " 01/01/2020 - "We found that treatment of alveolar rhabdomyosarcoma (aRMS) cells with Guadecitabine (SGI-110), a next-generation DNA methyltransferase inhibitor (DNMTi), resulted in a significant reduction of FGFR4 protein levels, 5 days post treatment. "
7.	Histone Deacetylase InhibitorsIBA 01/01/2019 - "We previously identified the class I-specific histone deacetylase inhibitor, entinostat (ENT), as a pharmacological agent that transcriptionally suppresses the PAX3:FOXO1 tumor-initiating fusion gene found in alveolar rhabdomyosarcoma (aRMS), and we further investigated the mechanism by which ENT suppresses PAX3:FOXO1 oncogene and demonstrated the preclinical efficacy of ENT in RMS orthotopic allograft and patient-derived xenograft (PDX) models. " 01/01/2019 - "Entinostat, a selective class I histone deacetylase inhibitor, has been reported to enhance the activity of cytotoxic agents and suppress expression of PAX3-FOXO1 in alveolar rhabdomyosarcoma (ARMS). "
8.	Etoposide (VP 16)FDA LinkGeneric 02/01/1997 - "Long-lasting complete remission after prolonged administration of etoposide in a child with a second recurrence of alveolar rhabdomyosarcoma." 02/01/1997 - "The authors report a case of alveolar rhabdomyosarcoma (RMS) of the thigh complicated by two successive distant relapses shortly after radio- and chemotherapy, treated with etoposide, and resulting in complete long-lasting remission. " 01/01/2012 - "We report for the first time the impact of neoadjuvant oral low-dose chemotherapy consisting of oral trofosfamide, idarubicin, and etoposide (O-TIE) in the case of alveolar rhabdomyosarcoma (RMS) in the lower jaw of an 18-year-old woman at 27 weeks of gestation, without fetal complications and a highly efficient anti-tumor response. "
9.	ZSTK474IBA 01/01/2023 - "We also demonstrated the efficacy of ZSTK474 in a preclinical model, particularly in cell lines from synovial sarcoma (SS), Ewing's sarcoma (ES) and alveolar rhabdomyosarcoma (ARMS), all of which harbor chromosomal translocations. " 10/12/2018 - "In addition, ZSTK474 induced apoptosis selectively in Ewing's sarcoma (RD-ES and A673), alveolar rhabdomyosarcoma (SJCRH30) and synovial sarcoma (SYO-1, Aska-SS and Yamato-SS) cell lines, all of which harbor chromosomal translocation and resulting oncogenic fusion genes, EWSR1-FLI1, PAX3-FOXO1 and SS18-SSX, respectively. "
10.	entinostat (MS 275)IBA 01/01/2019 - "We previously identified the class I-specific histone deacetylase inhibitor, entinostat (ENT), as a pharmacological agent that transcriptionally suppresses the PAX3:FOXO1 tumor-initiating fusion gene found in alveolar rhabdomyosarcoma (aRMS), and we further investigated the mechanism by which ENT suppresses PAX3:FOXO1 oncogene and demonstrated the preclinical efficacy of ENT in RMS orthotopic allograft and patient-derived xenograft (PDX) models. " 01/01/2019 - "Entinostat, a selective class I histone deacetylase inhibitor, has been reported to enhance the activity of cytotoxic agents and suppress expression of PAX3-FOXO1 in alveolar rhabdomyosarcoma (ARMS). "

Therapies and Procedures

1.	Therapeutics 01/01/2018 - "Finally, the improved nanocarrier pharmacokinetics led to prolonged median survival of mice bearing a clinically relevant PDX model of alveolar rhabdomyosarcoma from 19 days for the untreated controls to 27 days for the targeted therapy." 02/01/1989 - "Despite the limited statistical evidence, there is some indication that intensive therapy improved the prognosis of children with localized alveolar rhabdomyosarcoma (RMS)." 09/01/2014 - "Here we outline a study in alveolar rhabdomyosarcoma, in which we use gene expression profiling and a series of drug prediction algorithms combined with a matched patient-derived xenograft (PDX) model to test bioinformatically predicted therapies. " 01/01/2023 - "Anomalies of the FOXO1 gene in alveolar rhabdomyosarcoma are associated with a worse clinical prognosis, which determines the high value of studying the status of this gene when choosing a therapy strategy. " 11/22/2016 - "Success of current therapies is still limited and outcome is particularly poor for metastatic alveolar rhabdomyosarcoma (aRMS). "
2.	Drug Therapy (Chemotherapy) 06/01/2021 - "DCX and CRABP2 are candidate genes for differential diagnosis between pre-chemotherapy embryonic and alveolar rhabdomyosarcoma in pediatric patients." 05/01/2021 - "We present the case of a 13 year old boy, with sudden onset painful unilateral visual loss, prior to commencing chemotherapy for alveolar rhabdomyosarcoma. " 01/01/2021 - "A bioengineering method for modeling alveolar Rhabdomyosarcoma and assessing chemotherapy responses." 12/01/2015 - "Alveolar rhabdomyosarcoma with massive disseminated intravascular coagulopathy treated with systemic chemotherapy." 11/16/2015 - "PBT was performed safely and effectively for a 1-year-old girl with alveolar rhabdomyosarcoma with liver and cardiac invasion that was resistant to surgery and chemotherapy. "
3.	Radiotherapy 01/01/2020 - "We evaluated clinical and treatment planning records of patients aged ≤21 years with parameningeal alveolar rhabdomyosarcoma treated with definitive or adjuvant radiation therapy at our institution. " 01/01/2020 - "To determine patterns of failure, clinical outcomes, and prognostic factors among pediatric patients treated with radiation therapy for parameningeal alveolar rhabdomyosarcoma. " 04/01/2019 - "NRF2 orchestrates the redox regulation induced by radiation therapy, sustaining embryonal and alveolar rhabdomyosarcoma cells radioresistance." 11/23/2015 - "Radiotherapy to metastatic disease sites prevented in-field progression in all five patients with metastatic alveolar rhabdomyosarcoma. " 09/01/2015 - "Radiation therapy may increase metastatic potential in alveolar rhabdomyosarcoma."
4.	Lasers (Laser) 12/01/2003 - "In this study, 12 cases of well-defined sarcomas including Ewings sarcoma/primitive neuroectodermal tumors (ES/PNET), synovial sarcoma (SS), alveolar rhabdomyosarcoma (ARMS), and desmoplastic small round cell tumors (DSRCT) were used to collect specific numbers of cells by laser capture microdissection (LCM), subsequently used for RT-PCR to detect specific chimeric gene transcripts. "
5.	Chemoradiotherapy 07/17/2015 - "We report a rare case of childhood alveolar rhabdomyosarcoma of the mediastinum with vascular invasion treated with chemoradiotherapy and debulking surgery. " 07/01/2009 - "Alveolar rhabdomyosarcoma arising from the infraspinatus muscle in a child treated with subtotal scapulectomy and chemoradiotherapy: a case study." 02/01/1991 - "Two adolescent females with metastatic alveolar rhabdomyosarcoma underwent intensive chemoradiotherapy and bone marrow transplantation, one autologous and one allogeneic. "