Neoplasms

All Conditions

Behaviors and Mental Disorders

Skin and Connective Tissue Diseases

Rare Diseases

Fibromatosis, Aggressive

Aggression

Fibroma

Neoplasms, Fibrous Tissue

Neoplasms, Connective Tissue

Neoplasms, Connective and Soft Tissue

Neoplasms by Histologic Type

Desmoid Tumor

Participants will have a clinical diagnosis of desmoid fibromatosis, either new or newly recurrent

MRI with gadolinium contrast will be the imaging modality of choice. In patients with contraindications to MRI who instead undergo baseline evaluation by CT scan, tumor evaluation will be by serial CT scans with IV contrast throughout the study period.

Cross-sectional imaging

Aimee M Crago, MD, PhD

Meera Hameed, MBBS

The purpose of this study is to closely observe people with desmoid-type fibromatosis over 1 months.

Observing People With Desmoid-Type Fibromatosis

A Prospective Analysis of Active Observation in Patients With Desmoid-Type Fibromatosis

Memorial Sloan Kettering Cancer Center supports the international committee of medical journal editors (ICMJE) and the ethical obligation of responsible sharing of data from clinical trials. The protocol summary, a statistical summary, and informed consent form will be made available on clinicaltrials.gov when required as a condition of Federal awards, other agreements supporting the research and/or as otherwise required. Requests for deidentified individual participant data can be made beginning 12 months after publication and for up to 36 months post publication. Deidentified individual participant data reported in the manuscript will be shared under the terms of a Data Use Agreement and may only be used for approved proposals. Requests may be made to: crdatashare@mskcc.org.

The primary objective of this study is to estimate progression-free survival at 12 months, with the events defined by progression via radiographic criteria (RECIST 1.1).

Memorial Sloan Kettering Cancer Center

Pierre AULOGE, MD

Afshin GANGI, MD, PhD

Roberto Luigi CAZZATO, MD

Julien GARNON, MD

François SEVERAC, Statistician

Desmoid tumors, also called aggressive fibromatosis, are rare, locally invasive tumors with no potential for metastasis. The incidence is approximately 2 to 4 per million per year in the general population. The "watch and wait" policy is the standard of care for newly diagnosed patients in Europe. Patients who progress have first-line medical treatment, surgery is no longer the treatment because there is a high rate of recurrence (\> 60%) and radiotherapy is not very suitable because the patients are often young and the tumors are large. Cryoablation seems to be an effective therapeutic option that should be evaluated.

Cryoablation for Advanced and Refractory Desmoid Tumors

University Hospital, Strasbourg, France

Antineoplastic Agents

Antisickling Agents

All Drugs and Chemicals

Hydroxyurea

Enzyme Inhibitors

Patients will take oral hydroxyurea with a starting dose of 20 mg/kg daily. Patients will remain on hydroxyurea as long as there is a response or stability in tumor size and associated toxicities are manageable. Maximum treatment duration will be one year.

Richard B Womer, M.D.

The purpose of this study is to examine the response rate of desmoid tumors to hydroxyurea. The investigators hypothesize that hydroxyurea will be a safe, non-toxic alternative to aggressive surgery or chemotherapy for this difficult to treat tumor.

The treatment of desmoid tumors is complicated by benign histology and potentially aggressive and recurrent behavior. Invasive or intensive treatments with surgery or radiation therapy can lead to good tumor control, but at the expense of significant side effects along with a high risk of local recurrence after surgical excision. Low dose regimens of standard chemotherapy drugs have been favored as a conservative first-line treatment; however, refractory and recurrent tumors are not uncommon. The purpose of this study is to investigate the efficacy and safety of using hydroxyurea, an anti-neoplastic agent with relatively few side-effects, as a novel treatment for primary, unresectable desmoid tumors, desmoid tumors that have had an incomplete primary resection, or desmoid tumors that have recurred after other therapy.

Patients presenting to our oncology center with a new diagnosis of desmoid tumor, or a recurrent desmoid tumor, will be eligible for this study. After informed consent, physical examination, and baseline imaging studies (CT/MRI), patients will receive oral hydroxyurea at a starting dose of 20 mg/kg daily. Laboratory tests will be performed at set intervals to maintain the appropriate dose and monitor for cytopenias. Clinical evaluations, physical examinations, and/or imaging studies will be conducted every three months to assess for changes in tumor size. Patients will remain on hydroxyurea as long as there is a response or stability in tumor size and associated toxicities are manageable. Data will be collected regarding the response to the drug, the duration of the response, and the occurrence of adverse events. The goal of the analysis will be to determine the efficacy of hydroxyurea as a treatment modality for desmoid tumors.

Effect of Hydroxyurea as Treatment for Primary Desmoid Tumors

Effect of Hydroxyurea as Treatment for Primary Desmoid Tumors in Adults and Children (CHP-914)

Children's Hospital of Philadelphia

Anti-Inflammatory Agents

Triamcinolone

Triamcinolone Acetonide

Triamcinolone hexacetonide

Triamcinolone diacetate

Glucocorticoids

Hormones

Hormone Antagonists

Immunosuppressive Agents

Immunologic Factors

A concentration of 40 mg/ml of Triamcinolone Acetonide will be used for injections with a total dose of 120 mg of Triamcinolone given per injection. The injections will be performed under ultrasound guidance by a fellowship-trained musculoskeletal radiologist. The injection locations will be left to the discretion of the radiologist with the request to attempt to distribute the drug throughout the tumor. A total of three injections will be performed at six week intervals.

Benjamin K Wilke, MD

There is research supporting treatment of superficial fibromatoses (palmar fibromatosis and keloids) with triamcinolone acetonide injections. These lesions are histologically similar to deep fibromatoses (desmoid tumors). Currently there is little literature evaluating the response of desmoid tumors to injections of triamcinolone acetonide. The investigators aim to perform a pilot study evaluating the response of desmoid tumors to intralesional triamcinolone. If positive results are observed (based on RECIST criteria), then a phase II study will be initiated.

Desmoid tumors are proliferations of fibroblasts and myofibroblasts, intermixed with abundant dense collagen. Histologically they are similar to superficial fibromatoses such as palmar (Dupuytren's disease) and plantar fibromatoses, as well as keloids. Unlike the superficial fibromatoses, desmoid tumors are located in the deep tissue and stratified based on an abdominal or extra-abdominal location.

There are currently many treatment options for desmoid tumors. Historically, wide surgical resection was the treatment of choice. This often resulted in a disfiguring appearance and recurrence was common, with rates between 30 - 40% following resection. Additionally, radiation and systemic therapies are performed, with an approximate 26% rate of objective response, based on RECIST. These therapies are however not without side effects. Observation initially is also a reasonable approach as a recent study reported up to 60% of desmoids demonstrating stable disease and 18% spontaneously regressing over a five year period. Longer follow-up studies are needed to determine the true natural history of desmoid tumors, but there currently is a need for an alternative treatment strategy with increased efficacy and fewer side-effects once observation has failed.

Corticosteroids, such as triamcinolone acetonide, have long been used in the treatment of hypertrophic scars and keloids in order to decrease the size of the lesion. Proposed mechanisms of action of corticosteroids on keloids and hypertrophic scars include a decrease in the production of collagen, dissolution of insoluble collagen (collagenolysis), a decrease in the local inflammatory process, and an increased rate of apoptosis of fibroblast and inflammatory cells. Recently, reports have evaluated the use of triamcinolone acetonide in Dupuytren's disease, a superficial fibromatosis. A randomized controlled trial evaluated range of motion in patients following needle aponeurotomy alone and in combination with serial triamcinolone injections. They found improved range of motion in the triamcinolone cohort up to 24 months following treatment. Similarly, Ketchum et al reported that 97% of Dupuytren's nodules showed clinical regression following an average of 3.2 intralesional injections with triamcinolone acetonide, although half of these patients did experience reactivation of the disease at 1-3 years following treatment.

Currently there is limited reported experience with treatment of desmoid tumors by steroid therapy. Rhee et al reported a case of a chest wall desmoid tumor that recurred after two surgical resections and postoperative radiation therapy. They treated the lesion with weekly intralesional injections of 120 mg of triamcinolone acetonide for 4 weeks. At six months they noted a reduction in the size of the lesion. Similarly, Umemoto et al. reported a case of a 37 year old male with familial polyposis coli and intra-abdominal desmoid tumors. He was treated with oral prednisolone therapy with gradual regression of the lesions. At one year and six months following his last operation, the patient had no signs of recurrence of the desmoid tumors.

Based on this background information we aim to perform a pilot study of 10 patients with a histologically confirmed diagnosis of extra-abdominal aggressive fibromatosis (desmoid tumor) in order to determine the response rate of intralesional injections of triamcinolone acetonide. Response will be evaluated using the World Health Organization (WHO) response criteria, total volume of the tumor, T2 signal hyperintensity, and RECIST.

A Pilot Study of Intralesional Injection of Triamcinolone Acetonide for Desmoid Tumors

Defined as any untoward or undesirable experience associated with the use of a medical product.

Including hospitalization, prolonged disability/incapacity, life-threatening adverse experience, birth defect, and death.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Observing People With Desmoid-Type Fibromatosis	NCT04281381	Desmoid Fibroma...	Cross-sectional...	18 Years -	Memorial Sloan Kettering Cancer Center
Observing People With Desmoid-Type Fibromatosis	NCT04281381	Desmoid Fibroma...	Cross-sectional...	18 Years -	Memorial Sloan Kettering Cancer Center
Cryoablation for Advanced and Refractory Desmoid Tumors	NCT06113094	Desmoid Tumors		18 Years -	University Hospital, Strasbourg, France
Effect of Hydroxyurea as Treatment for Primary Desmoid Tumors	NCT00978146	Desmoid Tumors Fibromatosis	Hydroxyurea	- 21 Years	Children's Hospital of Philadelphia
Observing People With Desmoid-Type Fibromatosis	NCT04281381	Desmoid Fibroma...	Cross-sectional...	18 Years -	Memorial Sloan Kettering Cancer Center
A Pilot Study of Intralesional Injection of Triamcinolone Acetonide for Desmoid Tumors	NCT03627741	Fibromatosis	Triamcinolone A...	18 Years - 89 Years	Mayo Clinic
Observing People With Desmoid-Type Fibromatosis	NCT04281381	Desmoid Fibroma...	Cross-sectional...	18 Years -	Memorial Sloan Kettering Cancer Center

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