Alveolar soft tissue sarcoma | UniteRare Disease Library

Overview

Alveolar soft part sarcoma (ASPS) is a rare malignant soft tissue tumor that accounts for less than 1% of all soft tissue sarcomas. Despite its name, the term 'alveolar' refers to the microscopic pattern of the tumor cells, which resemble the small sacs (alveoli) of the lung, and does not indicate that the tumor originates in the lungs. ASPS is characterized by a specific chromosomal translocation, der(17)t(X;17)(p11;q25), which results in the ASPSCR1-TFE3 fusion gene. This fusion gene drives the tumor's growth and is considered the hallmark molecular feature of the disease. ASPS most commonly arises in the deep soft tissues of the extremities, particularly the thigh, in adults. In children and adolescents, it more frequently occurs in the head and neck region, including the orbit and tongue. The tumor typically presents as a slow-growing, painless mass that may go undetected for a prolonged period. Despite its indolent local growth, ASPS has a high propensity for metastasis, particularly to the lungs, brain, and bones. Metastatic disease may be present at diagnosis or develop years after initial treatment, necessitating long-term surveillance. Treatment of localized ASPS primarily involves wide surgical excision. The tumor is generally resistant to conventional chemotherapy, which distinguishes it from many other soft tissue sarcomas. Radiation therapy may be considered in certain cases, particularly when complete surgical resection is not achievable. In recent years, immune checkpoint inhibitors (such as atezolizumab and pembrolizumab) and anti-angiogenic agents (such as sunitinib and pazopanib) have shown promising activity in advanced or metastatic ASPS and represent an evolving area of treatment. Despite the slow growth pattern, the long-term prognosis can be guarded due to the risk of late metastases, with five-year survival rates being relatively favorable but declining significantly at longer follow-up intervals.

Also known as:

Alveolar soft part sarcoma ASPS

Inheritance

Sporadic

Usually appears on its own, not inherited from a parent

Age of Onset

Childhood to adulthood

Can begin any time from childhood through adulthood

Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

10 events

Mar 2026

ERIBULIN MESYLATE: FDA approved

FDAcompleted

Mar 2026GCAR1, a Chimeric Antigen Receptor (CAR) T-CELL Therapy for Relapsed/Refractory GPNMB-Expressing Solid Tumours

Canadian Cancer Trials Group — PHASE1

TrialNOT YET RECRUITING

Nov 2025A Study of Therapeutic Drug Monitoring-Based Atezolizumab Dosing

National Cancer Institute (NCI) — PHASE1

TrialRECRUITING

Aug 2025A Study to Determine the Safety and Effectiveness of the Investigational Cellular Therapy GCAR1 in a Patient With Alveolar Soft Part Sarcoma

University of Calgary — EARLY_PHASE1

TrialNOT YET RECRUITING

Jan 2025CAR T Therapy With GCAR1 for Relapsed Alveolar Soft Part Sarcoma

University of Calgary — EARLY_PHASE1

TrialACTIVE NOT RECRUITING

Dec 2024Clinical and Ecological Impact of a Primary Care Antimicrobial Stewardship Program Based on Telematic Educational Interviews (TELÉMACO Trial)

Fundación Pública Andaluza para la gestión de la Investigación en Sevilla — NA

TrialRECRUITING

Aug 2024

Tecelra: FDA approved

treatment of adults with unresectable or metastatic synovial sarcoma who have received prior chemotherapy, are HLA-A*02:01P, -A*02:02P, -A02:03P, or -A*02:06P positive and whose tumor expresses the melanoma-associated antigen A4 (MAGE-A4) antigen as determined by FDA-approved or cleared companion diagnostic devices

FDAcompleted

Feb 2024GPNMB-targeting Chimeric Antigen Receptor T-Cell Therapy (GCAR1) for a Patient With Alveolar Soft Part Sarcoma (CLIC-YYC-GPNMB-02)

AHS Cancer Control Alberta — EARLY_PHASE1

TrialACTIVE NOT RECRUITING

Dec 2023EaRly impAct theraPy With Ceftazidime-avibactam Via rapID Diagnostics

National University of Singapore — PHASE4

TrialRECRUITING

Dec 2022The CHAMP ASP: Promoting Physical Activity & Health in Children

University of Michigan — NA

TrialACTIVE NOT RECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

6 available

TECENTRIQ

atezolizumab· Genentech, Inc.

for the treatment of adult and pediatric patients 2 years of age and older with unresectable or metastatic ASPS

View Details →FDA Label ↗Copay Card ↗Patient Assistance ↗

Tecelra

afamitresgene autoleucel· USWM CT, LLC■ Boxed WarningOrphan Drug

treatment of adults with unresectable or metastatic synovial sarcoma who have received prior chemotherapy, are HLA-A*02:01P, -A*02:02P, -A02:03P, or -A*02:06P positive and whose tumor expresses the me…

treatment of adults with unresectable or metastatic synovial sarcoma who have received prior chemotherapy, are HLA-A*02:01P, -A*02:02P, -A02:03P, or -A*02:06P positive and whose tumor expresses the melanoma-associated antigen A4 (MAGE-A4) antigen as determined by FDA-approved or cleared companion diagnostic devices

View Details →FDA Label ↗

Lartruvo

olaratumab· Eli Lilly and CompanyOrphan Drug

In combination with doxorubicin, for the treatment of adult patients with soft tissue sarcoma (STS) with a histologic subtype for which an anthracycline-containing regimen is appropriate and which is …

In combination with doxorubicin, for the treatment of adult patients with soft tissue sarcoma (STS) with a histologic subtype for which an anthracycline-containing regimen is appropriate and which is not amenable to curative treatment with radiotherapy or surgery. (Indication withdrawn)

View Details →Patient Assistance ↗

Halaven

eribulin mesylate· Eisai Inc.Orphan Drug

Treatment of patients with unresectable or metastatic liposarcoma who have received a prior anthracyclne-containing regimen

View Details →FDA Label ↗

Yondelis

trabectedin· Janssen Research & Development, LLCOrphan Drug

For the treatment of patients with unresectable or metastatic liposarcoma or leiomyosarcoma who received a prior anthracycline-containing regimen

View Details →FDA Label ↗

Votrient

pazopanib· Novartis Pharmaceuticals Corp■ Boxed WarningOrphan Drug

Advanced soft tissue sarcoma (STS) who have received prior chemotherapy

View Details →FDA Label ↗Patient Assistance ↗

Clinical Trials

9 recruitingView all trials with filters →

Phase 31 trial

Phase III Trial of Anlotinib, Catequentinib in Advanced Alveolar Soft Part Sarcoma, Leiomyosarcoma, Synovial Sarcoma (APROMISS)

Phase 3

Actively Recruiting

PI: Paul CEO (Advenchen Laboratories, LLC) · Sites: Phoenix, Arizona; Los Angeles, California +22 more · Age: 18–99 yrs

Phase 23 trials

Testing Atezolizumab With Selinexor in People ≥ 12 Years Old With Alveolar Soft Part Sarcoma, The AXIOM Trial

Phase 2

Actively Recruiting

PI: Alice P Chen (National Cancer Institute LAO) · Sites: Los Angeles, California; Los Angeles, California +11 more · Age: 12–99 yrs

A Study of Pembrolizumab Plus Local Chemotherapy Using Isolated Limb Infusion (ILI) for Patients With Sarcoma in the Arm or Leg

Phase 2

Actively Recruiting

PI: Edmund Bartlett, MD (Memorial Sloan Kettering Cancer Center) · Sites: Basking Ridge, New Jersey; Middletown, New Jersey +5 more · Age: 12–99 yrs

Testing Atezolizumab Alone or Atezolizumab Plus Bevacizumab in People With Advanced Alveolar Soft Part Sarcoma

Phase 2

Active

PI: Alice P Chen (National Cancer Institute LAO) · Sites: Buena Park, California; Los Angeles, California +40 more · Age: 2–99 yrs

Phase 11 trial

A Study of Therapeutic Drug Monitoring-Based Atezolizumab Dosing

Phase 1

Actively Recruiting

PI: James L Gulley, M.D. (National Cancer Institute (NCI)) · Sites: Bethesda, Maryland · Age: 18–120 yrs

N/A1 trial

Multimodal Immune Characterization of RAre Soft Tissue Sarcoma - MIRAS Project From SARRA (SARcome RAre) Project of the French Sarcoma Group

N/A

Actively Recruiting

· Sites: Besançon; Bordeaux +18 more · Age: 18–99 yrs

Specialists

Showing 25 of 71View all specialists →

CX

Chunmiao Xu

Specialist