Rhabdomyosarcoma is a malignant soft tissue tumour that develops from cells of the transversely striated muscle. The incidence is most common in children and adolescents, accounting for 3% of all paediatric tumours and 50% of paediatric soft tissue sarcomas, according to the National Library of Medicine. The disease has a high degree of malignancy. The prognosis of treatment depends on the stage of cancer. The earlier the pathology is diagnosed, the higher the chances of successful treatment. In order not to miss precious time – parents need to know more information about this disease.
What is rhabdomyosarcoma?
Rhabdomyosarcoma is a malignant tumour of musculoskeletal origin. It is among the most common soft tissue sarcomas occurring in childhood.
The share of the disease accounts for about 3% of all oncopathologies in children. As a rule, the disease affects the head and neck region, urogenital tract, retroperitoneal space and extremities. Boys get the disease more often than girls.
Rhabdomyosarcoma is an aggressive tumour. It grows rapidly and quickly metastasizes. The causes of the development of the disease are unknown. However, its appearance is influenced by some syndromes and congenital anomalies. Among them are:
Beckwith-Wiedemann syndrome;
Neurofibromatosis type I (NF1);
Li-Fraumeni syndrome;
Pleuropulmonary blastoma;
Noonan syndrome;
Costello syndrome.
WHERE TO TREAT RHABDOMYOSARCOMA?
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Read: Types of rhabdomyosarcoma
Embryonal rhabdomyosarcoma
This tumour accounts for 70% of all cases of rhabdomyosarcoma. The pathology occurs in children younger than 5 years of age. It affects the head and neck, bladder, vagina or prostate and testicles.
Alveolar rhabdomyosarcoma
The disease develops in older children and adolescents. This type of cancer is more aggressive than embryonal cancer. The tumour metastasises rapidly. It occurs in the large muscles of the arms, legs, and trunk.
Groups of rhabdomyosarcomas
The clinical group of the disease is based on whether the surgeon can remove the tumour. According to this criterion, rhabdomyosarcomas are divided into the following groups:
Group 1 | This includes localised tumours that have not spread to the lymph nodes. Such tumours can be completely removed surgically. |
Group 2 | Group 2 tumours can be surgically removed, but cancer cells remain on the margins or lymph node involvement is present. |
Group 3 | These are tumours that have not spread to other organs, but for whatever reason cannot be removed surgically (often because of their localisation). |
Group 4 | This group includes tumours that have spread to distant areas of the body. They cannot be removed through surgery. |
Symptoms of rhabdomyosarcoma
Symptoms of the disease depend on the type and localisation of the neoplasm. Often tumours do not manifest themselves in any way until they reach a large size. The first signs of oncopathology – soreness and swelling in the area of the development of the malignant process.
As the disease spreads, patients experience increased fatigue, headaches, nausea, dizziness and loss of appetite.
Symptoms of advanced rhabdomyosarcoma include:
bone pain;
persistent cough;
weakness;
weight loss.
NHS Inform highlights the following symptoms, depending on the location of the pathology:
If the tumour is in the head or neck area, there is discharge from the nose or throat. If the tumour is located near the eye, it may look swollen.
A tumour in the abdominal area causes pain or discomfort in the epigastric region and difficulty defecating.
A tumour in the bladder area causes blood in the urine and difficulty urinating.
Alveolar rhabdomyosarcoma in children affects the muscles of the limbs and trunk. Pain associated with such tumours may be mistaken for a severe bruise or injury. It is therefore important to see a doctor immediately if such symptoms occur.
Stages of rhabdomyosarcoma
Stage 1 | Early-stage tumours are found in ‘favourable locations’ such as the orbital areas around the eyes, in the reproductive organs (testicles or ovaries), ureters, urethra or around the gallbladder. The neoplasms have not spread to lymph nodes. |
Stage 2 | Stage 2 tumours may spread to the lymph nodes. They do not exceed 5 cm in diameter but are found in “unfavourable sites” – any areas not in stage 1. |
Stage 3 | Tumours develop in areas that are not characteristic of stage 1 disease. Their size exceeds 5 cm. Metastases to lymph nodes are present. |
Stage 4 | Tumours of any localisation that have spread to lymph nodes and distant organs, in particular the lungs. |
RHABDOMYOSARCOMA TREATMENT ABROAD
Contact an experienced oncologist for a personalised course of rhabdomyosarcoma treatment. Foreign doctors successfully help children with tumours of any complexity, even stage 4 alveolar rhabdomyosarcoma. Leave an application on the MediGlobus website. We will help you find a clinic, doctor and organise your trip.
Diagnosis of rhabdomyosarcoma
Diagnosis of rhabdomyosarcoma begins with a thorough history and physical examination. Depending on the location of the tumour, imaging studies such as X-rays, CT, MRI and PET-CT are performed. Genetic tests are also performed.
To confirm the suspected diagnosis, a biopsy is required. Options for this test include a fine-needle biopsy (using a small needle to aspirate a tissue sample) or an open biopsy. The resulting material is viewed under a microscope.
A sentinel lymph node biopsy is performed to detect metastatic disease. This test involves injecting a blue dye and a radioactive indicator into the tumour and then taking a sample of neighbouring lymph nodes, which are stained blue. Bone scans, chest CT scans, bone marrow biopsies, etc. are also done to look for metastases.
According to the American Cancer Society, about 20% of patients have metastases in the bone marrow and lungs at the time of diagnosis.
Treatment methods for rhabdomyosarcoma
The choice of cancer treatment methods depends on the stage of the disease, the age of the patient, the localisation and size of the tumour. In some cases, bone marrow transplantation is indicated. The National Cancer Institute distinguishes 3 main methods of therapy: surgery, chemotherapy, and radiation therapy. In addition, good results in treatment are achieved by new methods – immune and targeted therapy. These methods are undergoing clinical trials, the initial results of which have shown high efficacy and safety of the drugs.
Surgery | Surgery is one of the priority treatment methods. Its main goal is the total removal of cancer cells. Surgery offers the best possibilities for long-term control of the disease. The type of intervention depends on the location of the tumour. Resection of the tumour is only performed if it can be removed without significantly damaging or altering the person’s appearance. If this is not possible, chemotherapy and/or radiation therapy are first resorted to. The combination of these methods can reduce the size of the tumour and the degree of trauma to healthy tissue. |
Chemotherapy | Rhabdomyosarcomas respond well to chemotherapy. After the use of chemo, about 80% of tumours significantly decrease in size. The method is used before and after surgery. It reduces the size of the neoplasm and destroys residual cancer cells after surgical removal. |
Radiation therapy | Radiotherapy is used together with chemo to shrink the tumour before surgery. It is also used after surgical removal of cancer, particularly when the surgeon cannot remove the tumour completely. The method is indicated as palliative therapy. |
Immunotherapy | Immunotherapy aims to mobilise the patient’s immune system to fight cancer. This method includes various approaches including vaccine therapy and immune checkpoint inhibitor therapy. Vaccine therapy uses substances that stimulate the body’s immune system to seek out and destroy tumours. Immune checkpoint inhibitors, such as Ipilimumab, block the CTLA-4 or PD-1 and PD-L1 proteins, allowing immune cells to recognise and destroy cancer cells more effectively. These methods are being actively investigated for the effective treatment of metastatic or recurrent rhabdomyosarcoma in children, promising new perspectives in the fight against this type of cancer. |
Targeted therapies | Targeted therapy as part of rhabdomyosarcoma treatment is a technique that uses drugs to identify and attack specific cancer cells. Inhibitors of mTOR and tyrosine kinase inhibitors identify abnormal cells by specific targets and block their ability to develop and divide. At the same time, healthy cells remain safe. Thus, this type of treatment is less harmful to the body than chemotherapy or radiation therapy. The drugs (Sirolimus and MK-1775) are being studied as potential therapeutic agents for the treatment of advanced and recurrent rhabdomyosarcoma. |
A new treatment for rhabdomyosarcoma
In 2023, the journal Molecular Therapy published the results of a study conducted by scientists from the Leibniz Institute for Ageing in Jena and Brandenburg University of Technology in Cottbus-Senftenberg, Germany. The scientists looked at the protein TRPS1, which was found to play a key role in tumour growth. They found that TRPS1 levels are elevated in cancer cells and blocking this protein allows tumour cells to turn into muscle cells, a type of cell that can no longer divide. Thus, the neoplasm stops growing.
“By studying human embryonal rhabdomyosarcoma tumour cells, we found that TRPS1 protein levels are permanently elevated,” explained Professor von Maltzahn. – In skeletal muscle tissue, elevated TRPS1 levels lead to impaired stem cell differentiation, and in embryonal rhabdomyosarcoma cells it leads to increased cell growth, which promotes tumour growth.”
“Based on these promising results, we believe we have found an effective novel therapeutic approach to treat embryonal rhabdomyosarcomas, which in the future could be used in addition to conventional chemotherapy and radiotherapy to stop tumour growth and spread,” explains Dr Hüttner, highlighting the relevance of the findings.
Prognosis of rhabdomyosarcoma treatment abroad
According to the American Society of Clinical Oncology, treatment prospects vary depending on the tumour location, stage of disease and age of the child. Children between 1 and 9 years of age have a better prognosis for oncotherapy than patients younger or older than this age range. Five-year survival rates for patients with stage 1-2 rhabdomyosarcoma range from 70% to 90%. For children with stage 3 oncopathology, the rate is about 50%. At stage 4 of the disease, the 5-year survival threshold is crossed by no more than 20% of patients.
According to the National Library of Medicine, the survival rate for alveolar rhabdomyosarcoma is 49%, and for embryonal tumours, it is 67%.
Where is rhabdomyosarcoma treated abroad?
Foreign citizens choose clinics in Turkey, Israel, Germany, Spain, South Korea, China and Austria for treatment of rhabdomyosarcoma. The leading medical centres are:
Summary
Rhabdomyosarcoma is the most common paediatric soft tissue carcinoma. It is characterised by an aggressive course. The neoplasm rapidly grows and metastases. Approximately 70% of cases occur in children under 5 years of age, with 2% present at birth. The prognosis of treatment depends on the stage of cancer. The earlier the pathology is diagnosed, the higher the chances of success in the fight against it.
Symptoms of the disease depend on the type and localisation of the tumour. The first signs of rhabdomyosarcoma are soreness and swelling in the area of the tumour.
Diagnosis of rhabdomyosarcoma begins with a thorough anamnesis and physical examination. Depending on the location of the cancer cells, imaging studies such as X-ray, CT, MRI, and PET-CT may be performed.
Foreign nationals choose clinics in Turkey, Israel, Germany, Spain, South Korea and Austria to fight cancer.
The prospects of rhabdomyosarcoma treatment are influenced by the localisation of the tumour, the stage of the disease and the general condition of the child. For rhabdomyosarcoma, children aged 1 to 9 years have a better prognosis than patients younger or older than this age range. The overall 5-year survival rate is 70%.
To organise rhabdomyosarcoma treatment abroad, please contact the coordinating physicians of the MediGlobus international medical platform. Our experts will answer all your questions. They will help you choose a clinic and a doctor, as well as guide you on the cost of services in a foreign medical centre. Submit your application!
Sources:
- 1. National Library of Medicine
- 2. NHS Inform
- 3. National Cancer Institute
- 4. Childhood Rhabdomyosarcoma Treatment
- 5. American Cancer Society
- 6. Molecular Therapy
- 7. News-Medical Life Sciences
- 8. American Society of Clinical Oncology
- 9. National Library of Medicine – Cancer
