Complete Radiological Remission Research Articles

Hepatic involvement in granulomatosis with polyangiitis - diagnostic difficulties.

We report the case of a 57-year-old Caucasian man who presented with dry cough, haemoptysis, fever, lung nodules, erythrocyturia, and acute hepatitis. After a lung biopsy, the patient was diagnosed with granulomatosis with polyangiitis. The diagnosis was supported by the presence of anti-proteinase-3 anti-neutrophil cytoplasmic antibodies. The most common causes of liver damage are excluded.The patient was treated with prednisone and cyclophosphamide, which resulted in remission of chest CT findings and improvement in liver function tests. During the flare, new lung infiltrations as well as elevation of liver enzymes were present. Treatment with rituximab resulted in complete clinical and radiological remission and normalisation of liver function tests. What makes this case worth reporting is the rare liver involvement in the GPA. After exclusion of coexistence of autoimmune liver disease and drug-induced liver lesion, organ involvement was supported by the reversion of abnormal LFT after the immunosuppression of GPA.

Image-guided interstitial high-dose-rate brachytherapy for locally recurrent uterine cervical cancer: A single-institution study

PurposeThe aim of this study was to investigate the efficacy and safety of image-guided high-dose-rate (HDR) interstitial brachytherapy (ISBT) for reirradiation of locally recurrent uterine cervical cancer. Methods and MaterialsBetween 2008 and 2015, patients receiving reirradiation using HDR-ISBT for local gross recurrence of uterine cervical cancer after definitive or postoperative radiotherapy were analyzed retrospectively. The prescription doses per fraction ranged 2.5–6.0 Gy, whereas the cumulative equivalent doses in 2 Gy fractions ranged 48.6–82.5 Gy. The effects of prognostic factors on the local control (LC), progression-free survival, and overall survival were analyzed, and late toxicity data were evaluated. ResultsEighteen patients were included in the analysis, with a median followup of 18.1 months. A tumor response was obtained in all patients, with radiological and pathological complete remission seen in 12 (66.7%) patients. The 2-year LC, progression-free survival, and overall survival rates for all patients were 51.3%, 20.0%, and 60.8%, respectively. The hemoglobin level and maximum tumor diameter were shown to be statistically significant prognostic factors for LC (p = 0.028 and 0.009, respectively). Late ≥ Grade 2 adverse events were observed in 5 patients (27.8%). ConclusionsImage-guided HDR-ISBT for the reirradiation of locally recurrent uterine cervical cancer may play an important role for local tumor control in a subgroup of patients. However, the treatment indication must be weighed against the risk of higher-grade late toxicity.

Abstract B01: Association of cholangiocarcinoma and hepatocellular carcinoma: Which one should be treated?

Abstract Introduction: The combination of cholangiocarcinoma and hepatocellular carcinoma is a rare entity. Methods: We will present a clinical case involving a combination of cholangiocarcinoma and hepatocellular carcinoma in a patient with post-viral C cirrhosis. Results: A 60 Y.O male know with a personal history of hepatitis treated in 1969 by an unknown injectable treatment for two years, was presented at the consultation for evolving abdominal bloating. CT-scan revealed ascites with a total portal vein thrombosis and a heterogeneous tumor occupying the right liver without “wash-out” sign. The patient was put under anticoagulation with LMWH. After a week, liver MRI found that the ascites has totally dried, the thrombosis disappeared, and revealed that the tumor had a typical aspect of cholangiocarcinoma. Alfa-fetal protein test was to 1156 IU / ml. Liver biopsy was contradictory; on some pieces hepatocellular carcinoma was found and on others cholangiocarcinoma. Because Sorafenib is a multikinase inhibitor with anti-angiogenic and anti-proliferative action it can be effective on many tumors as they use the same ligands and signaling pathways similar to proliferate, it was chosen instead of chemotherapy at 400 mg/day. Sure enough, after two weeks of treatment, alfa fetoprotein decreased to 834 IU/ml and after two months biology and imaging found a satisfactory partial response. Currently, after six months of treatment, the patient is in complete clinical, biological and radiological remission except that the treatment cannot be interrupted for the time. Surgeons are discussing a partial surgery to the residual liver retraction. Conclusion: This association is rare and needs deep research especially molecular testing for patients who have a spectacular response. Citation Format: Khaoula Mazouzi. Association of cholangiocarcinoma and hepatocellular carcinoma: Which one should be treated? [abstract]. In: Proceedings of the AACR International Conference: New Frontiers in Cancer Research; 2017 Jan 18-22; Cape Town, South Africa. Philadelphia (PA): AACR; Cancer Res 2017;77(22 Suppl):Abstract nr B01.

Initial phase 1 study of WT2725 dosing emulsion in patients with advanced malignancies.

2066 Background: WT2725 is a Wilms’ tumor (WT1)-derived-oligopeptide vaccine intended to induce WT1 specific cytotoxic T-lymphocytes against WT1 positive tumors in HLA-A*0201+ or HLA-A*0206+. This first in human study of WT2725 was conducted to evaluate the safety, tolerability, and efficacy. Methods: Subjects with progressive or recurrent glioblastoma (GBM), acute myeloid leukemia (AML) (patients in morphologic remission with minimal residual disease determined by WT1 RT-PCR were allowed), non-small cell lung cancer (NSCLC), or ovarian cancer despite standard therapy were treated with 0.3, 0.9, 3, and 9 mg of WT2725 subcutaneously (s.c.) every week for 4 weeks, then every other week for 6 weeks, and every 4 weeks thereafter until progression or other discontinuation event (part 1); and with 18 mg and 27 mg of WT2725 s.c. every week for 8 weeks, then every other week for 10 weeks, and every 4 weeks thereafter until progression or other discontinuation event (part 2). Responses were evaluated by Immune-related Response Criteria (irRC) for solid tumors and modified International Working Group (IWG) for AML. Results: 62 subjects were dosed, the most frequent adverse events were grade 1 injection site reactions (no grade 3 injection site reactions or dose limiting toxicities observed). Of the 21 GBM subjects who were dosed, seven survived for ≥ 1 year (33%). Of these seven subjects, three survived for ≥18 months (14%), and two for ≥2 years (both in complete radiologic remission, 9.5%). One of the seven subjects with ≥1 year survival had previous treatment with bevacizumab. Two subjects continue dosing (1 complete response with no measurable disease >3 years and 1 partial response > 13 months on study). Results in subjects with non-GBM tumors (12 subjects with AML, 21 with ovarian CA, 7 with NSCLC, and 1 other cancer) are not presented here. WT1-specific CTLs and tumor burden was monitored. WT1 specific CTLs were associated with decrease of tumor burden in two patients with low tumor burden. Some patients with high disease burden did not respond. Conclusions: Preliminary data suggests WT2725 is well tolerated with potential antitumor activity in GBM patients, supporting future development of WT2725 for treatment of GBM in selected subjects. Clinical trial information: NCT01621542.

Ovarian Germ Cell Tumours: Clinicopathological Features and Treatment Outcomes

Malignant germ cell ovarian tumors (MOGCTs) represent less than 20% of ovarian cancers. We conducted a retrospective study of 16 cases of (MOGCTs) diagnosed from January 2008 to December 2013. Results: During this time 26 cases of non epithelial ovarian malignancy were diagnosed which 16 are germinal tumors 61%. The age of patients ranged from 16 to 60 years. Abdominal mass was the principal symptom, it was present in 2/3 of cases (66%). All patients were surgically assessed with ovariectomy that was associated with hysterectomy in two patients. Chemotherapy was administered in 15 patients and BEP was the commonest regimen used. After a mean follow up of 25 months we obtained a clinical, biological and radiologic complete remission in 1/3 of cases, 3 recurrences , 2 deaths. Overall survival: After a median follow up of 25 months. We noted 2 death. The median OS was not reached. The 2-year OS rate was 93.8%. Event free survival (EFS): Events were defined as death, recurrence or progression of the disease. The median EFS was not reached.The 2-year EFS rate was 73%. Conclusion: (MOGCTs)are rare; they generally affect the young woman and are diagnosed at an early stage. Surgery has an important role.

Fatigue Syndrome in Sarcoidosis

Sarcoidosis is an inflammatory disease of unknown etiology. Most commonly it results in the formation of non-caseating granulomas in intrathoracic lymph nodes and lung parenchyma, but the clinical course and picture may be complicated by extrapulmonary involvement and many non-respiratory signs and symptoms which are directly related to the disease. In addition, sarcoidosis patients may suffer from a plethora of symptoms of uncertain or unknown origin. Fatigue is one of these symptoms, and according to some authors it is reported by the majority of patients with active sarcoidosis, but also by a smaller proportion of patients with inactive sarcoidosis, or even with complete clinical and radiological remission. Therefore the term fatigue syndrome is frequently used to name this clinical problem. The definition of fatigue syndrome in sarcoidosis is imprecise and the syndrome is usually recognized by use of validated questionnaires. In this review the uptodate knowledge in this field was presented and different challenges connected with this syndrome were described.

Role of follow-up endoscopic examination in treatment response assessment for patients with gastric diffuse large B cell lymphoma

Objective: According to lymphoma guidelines, gastric diffuse large B cell lymphoma (DLBCL) patients should undergo regular computed tomography (CT) and/or positron emission tomography (PET) examinations to assess treatment response. Endoscopic examinations are not indicated in the guidelines. The aim of this study was to investigate the utility of endoscopic examinations during and after treatment for DLBCL.Methods: We reviewed the patients diagnosed with gastric DLBCL at Seoul St. Mary’s Hospital. All patients underwent endoscopy and radiologic examinations at every follow-up appointment. Radiologic response was defined according to World Health Organization criteria and endoscopic response was determined based on the Groupe d’Etude des Lymphomes de l’Adult grading system that is widely used in post-treatment evaluation of gastric MALT lymphoma.Results: Forty-five patients were analyzed. Within a median follow-up period of 34 months, 35 patients achieved both radiologic and endoscopic complete remission (CR). The median times to endoscopic and radiologic CR were not significantly different (21 versus 16 weeks, p = 0.118). However, in 25 patients with stage I disease, endoscopic CR [median (range), 20 (11–36)] was achieved later than radiologic CR [median (range), 13 (8–36)] (p = 0.027). Among 40 patients who achieved radiologic CR, 35 patients who also achieved endoscopic CR maintained remission during the follow-up. Two of the five patients who achieved radiologic CR without endoscopic CR experienced recurrence.Conclusions: In gastric DLBCL patients, endoscopic response does not always correlate with radiologic response and might predict disease recurrence. We suggest that follow-up endoscopic examination with biopsy should be performed in addition to radiologic examination.

Successful treatment of arsenic-induced lung malignancy: a case report

Environmental arsenicosis is a serious public health problem in parts of Eastern India. Chronic arsenicosis is known to cause malignancy of various organs. We here report a case of simultaneous lung and skin squamous cell cancer in a 49-year-old male subject. His regular tube well drinking water was found to contain high levels of arsenic (0.125 mg/L), and his hair and nail samples also tested positive for arsenic. He was treated with chemotherapy (gemcitabine/carboplatin), followed by radiotherapy. He responded to the treatment with complete radiological remission. This case highlights a serious public health concern for arsenic affected parts of India.

画像上完全消退後に再出現した肺浸潤性粘液腺癌の1例

症例は68歳，女性．胸部単純X線写真にて右肺下葉に異常陰影を指摘され紹介受診．CTにて右S9に最大径40mm大の腫瘤影を認めたが，経過観察中にこの異常陰影は縮小し，その後に完全な消失が確認された．3年後に検診の胸部単純X線写真にて再度右肺下葉の異常陰影を指摘され，胸部CTにて3年前の異常陰影と同一部位に腫瘤性陰影が認められ，CTおよびPET上の所見は3年前の陰影と同様であった．気管支鏡下の細胞診にて悪性所見を認め，下葉切除術を施行．病理組織学的検査にて浸潤性粘液腺癌と診断された．術後経過は良好であり，術後1年10カ月経過した現在に至るまで再発は認められていない．画像上完全消退後に再出現した極めて稀な肺癌であることも否定できない1例と考えられ，抗核抗体による自己免疫が関与した可能性も含め若干の文献的考察を加え報告する．

Hepatic Parenchymal Heterogeneity on Contrast-enhanced CT Scans Following Oxaliplatin-based Chemotherapy: Natural History and Association with Clinical Evidence of Sinusoidal Obstruction Syndrome.

To evaluate the natural history and contrast material-enhanced computed tomographic (CT) features of postoxaliplatin heterogeneity of liver parenchyma (POHL) and to investigate the association of POHL with clinical factors and biomarkers of sinusoidal obstruction syndrome (SOS). The retrospective study was approved by the institutional review board, and informed consent was waived. Two hundred seventy patients (159 men, 111 women; age range, 31-92 years) who underwent oxaliplatin-based chemotherapy (OBC) and serial contrast-enhanced CT were consecutively registered. POHL severity was independently scored by two abdominal imagers, who were blinded to the clinical data, using a six-point scale (POHL presence, ≥4), followed by a consensus review. Complete radiologic remission was determined by consensus on the disappearance of heterogeneity on CT images. The association of POHL severity score with CT-based quantitative (ie, change in spleen size and blood-free hepatic parenchymal attenuation) and laboratory values (ie, aspartate aminotransferase, alanine transaminase, and platelet count), as well as time to complete radiologic remission, were evaluated with the Spearman rank test. Multivariate analysis was performed to determine the association between clinical factors of SOS (ie, age, sex, number of OBC sessions, chemotherapy regimen, bevacizumab use, and presence of concomitant hepatic metastasis) and POHL development. RESULTS Interobserver agreement was excellent (κ = 0.90). POHL was present in 167 (61.9%) of 270 patients, and the number of OBC sessions was significantly associated with POHL development (odds ratio, 1.138; 95% confidence interval: 1.039, 1.245; P = .005). POHL severity score was correlated with quantitative CT and laboratory values (P < .05 for all statistical analysis). Peripheral distribution (103 of 167, 61.7%) and right lobar predominance (103 of 165, 62.4%) were the most common POHL features. The mean time to complete radiologic remission, which was correlated with POHL severity score, was 82.5 days ± 68.8 after OBC discontinuation. POHL development is associated with increased number of OBC sessions, and POHL severity was correlated with various biomarkers of SOS.

The benefit of consolidation radiotherapy to initial disease bulk in patients with advanced Hodgkin's disease who achieved complete remission after standard chemotherapy.

Background/purposeThe aim of this study was to evaluate the role of consolidation radiotherapy (RT) in advanced-stage Hodgkin’s disease (HD) with initial bulky sites after radiological complete remission (CR) or partial response (PR) with positron emission tomography-negative (metabolic CR) following standard chemotherapy (ABVD [Adriamycin, bleomycin, vinblastine, and dacarbazine]) six to eight cycles.Patients and methodsAdult patients with advanced-stage HD treated at our institute during the period 2006 to 2012 were retrospectively evaluated. One hundred and ninety-two patients with initial bulky disease size (>7 cm) who attained radiological CR/PR and metabolic CR were included in the analysis. One hundred and thirteen patients who received radiotherapy (RT) as consolidation postchemotherapy (RT group) were compared to 79 patients who did not receive RT (non-RT group). Disease-free (DFS) and overall survival (OS) rates were estimated using the Kaplan–Meier method and were compared according to treatment group by the log-rank tests at P ≤0.05 significance level.ResultsThe mean age of the cohort was 33 (range: 14 to 81) years. Eighty-four patients received involved-field radiation and 29 patients received involved-site RT. The RT group had worse prognostic factors compared to the non-RT group. Thirteen (12%) relapses occurred in the RT group, and 19 (24%) relapses occurred in the non-RT group. Nine patients (8%) in the RT group died, compared to eleven patients (14%) in the non-RT group. Second malignancies were seen in only five patients: three patients in the RT group compared to two patients in the non-RT group. At 5 years, overall DFS was 79%±9% and OS was 85%±9%. There was significant statistical difference between the RT group and the non-RT group regarding 5-year DFS: 86%±7% and 74%±9%, respectively (P ≤0.02). However, the 5-year OS was 90%±5% for the RT group and 83%±8% for the non-RT group, with no statistical difference (P ≤0.3). Conclusion: The results of our study suggest that consolidation RT in patients with advanced-stage HD with initial bulky disease who had postchemotherapy radiologic CR or PR with metabolic CR improved the DFS.

THU0394 Long-Term Therapy with Canakinumab in Two Patients with Refractory Chronic Autoinflammatory Arthritis

BackgroundCanakinumab, a human anti-IL-1β monoclonal antibody, has been licensed for the treatment of cryopyrin-associated periodic syndrome (CAPS), while few reports have shown benefit in patients with refractory familial Mediterranean fever...

Marker (+) CNS germ cell tumors in remission: Are surveillance MRI scans necessary?

Patients with marker (+) CNS germ cell tumors are usually followed with both surveillance MRI scans and serum tumor markers. We hypothesized that patients with elevated serum tumor markers at diagnosis who achieve a complete biochemical and radiological remission may not need surveillance MRI scans. We retrospectively identified 31 patients with CNS germ cell tumors who presented with an elevated serum tumor marker at the time of diagnosis. We reviewed the records of those patients who (1) achieved a complete biochemical and radiological remission and (2) later suffered tumor recurrence to determine whether the recurrence was detectable biochemically, radiologically, or via both modalities. Nine patients suffered tumor recurrence following initial remission. All 9 had elevated serum tumor markers at recurrence and 8 had MRI evidence of recurrence. The 1 patient with isolated biochemical evidence of recurrence developed MRI evidence of recurrence 15 months later without intervening treatment. One other patient (not one of the 9) had a secondary malignancy (anaplastic astrocytoma) identified by brain MRI scan. Patients with CNS germ cell tumors who present with elevated serum tumor markers at diagnosis and achieve a complete biochemical and radiological remission may not need surveillance MRI scans to monitor for recurrence, but MRI scans may be considered to monitor for secondary malignancy. If other series replicate these findings, surveillance via monitoring of serum tumor markers only could be done and omission or reduction of the frequency of surveillance MRI scans could save a significant amount of money.

Phase II trial of docetaxel, cisplatin and 5FU chemotherapy in locally advanced and metastatic penis cancer (CRUK/09/001)

Background:Penis cancer is rare and clinical trial evidence on which to base treatment decisions is limited. Case reports suggest that the combination of docetaxel, cisplatin and 5-flurouracil (TPF) is highly active in this disease.Methods:Twenty-nine patients with locally advanced or metastatic squamous carcinoma of the penis were recruited into a single-arm phase II trial from nine UK centres. Up to three cycles of chemotherapy were received (docetaxel 75 mg m−2 day 1, cisplatin 60 mg m−2 day 1, 5-flurouracil 750 mg m−2 per day days 1–5, repeated every 3 weeks). Primary outcome was objective response (assessed by RECIST). Fourteen or more responses in 26 evaluable patients were required to confirm a response rate of 60% or higher (Fleming-A'Hern design), warranting further evaluation. Secondary endpoints included toxicity and survival.Results:10/26 evaluable patients (38.5%, 95% CI: 20.2–59.4) achieved an objective response. Two patients with locally advanced disease achieved radiological complete remission. 65.5% of patients experienced at least one grade 3/4 adverse event.Conclusion:Docetaxel, cisplatin and 5FU did not reach the pre-determined threshold for further research and caused significant toxicity. Our results do not support the routine use of TPF. The observed complete responses support further investigation of combination chemotherapy in the neoadjuvant setting.

Successful resection of hepatocellular cancer not amenable to Milan criteria and durable complete remission induced by systemic polichemotherapy after development of metastases – should we think about revising the current treatment guidelines in selected patients?

ObjectivesTo refresh clinical diagnostic and therapeutic dilemmas in patients presenting with hepatocellular cancer (HCC) and to report a rare success of systemic polichemotherapy in metastatic HCC.MethodsCase report of a patient with successfully resected HCC although initially deemed inoperable according to current guidelines, and who was successfully treated by systemic polichemotherapy after development of metastatic disease, resulting in a sustained complete remission.ResultsWe describe a 71-year-old female with HCC initially treated by atypical liver resection, although not amenable to initial surgery according to current treatment guidelines, which resulted in 6 months disease-free interval. After development of pulmonary metastases, the patient was treated by systemic polichemotherapy, due to local unavailability of novel biologic agents. After 3 months of chemotherapy biochemical remission was confirmed, and after 10 months of active treatment complete radiological remission was verified according to Response Evaluation Criteria in Solid Tumors (RECIST) criteria, now exceeding 9 months in duration.ConclusionThere is an increasing body of evidence that criteria for surgical interventions in HCC should be revised and expanded, and our case is an example of such an approach. Although novel biologic therapies are not widely available in all regions of the world due to their cost, currently there are no hard recommendations for use of chemotherapy in such areas. Since this is a large problem in clinical practice, we conclude that chemotherapy should be offered to selected patients of good performance status if novel agents are unavailable.

PTH-092 Interval Scanning with Magnetic Resonance Enterography Demonstrates Response to Anti-Tnf Therapy and has Utility in Reassessment of Crohn’S Disease

IntroductionNICE guidelines mandate yearly reassessment of disease activity for those treated with anti-TNF therapy (ATT). Magnetic resonance enterography (MRE) is established in the assessment of small bowel Crohn’s disease, however,...

Can combination metronomic therapy overcome chemoresistance in cholangiocarcinoma? A literature review

Cholangiocarcinoma (CCa) is relatively resistant to chemotherapy as well as radiation therapy, and complete resection is the main curative therapy for these patients. The prognosis for patients with unresectable intrahepatic CCa (iCCa) is extremely poor. A 55-year-old woman presented at our hospital with abdominal pain. After evaluation, she was diagnosed to have multifocal iCCa. She did not opt for standard chemotherapy and therefore received oral metronomic therapy with a combination of celecoxib, etoposide, and cyclophosphamide for a total of 30 months. Presently, she is 57 months post diagnosis and 27 months post cessation of all treatment and continues to be in complete radiological remission. In the present report, we review the literature and discuss whether metronomic scheduling of biologic agents and anticancer drugs will be able to overcome chemoresistance and improve the outcome in cholangiocarcinoma. References for the review were identified through searches of Pubmed for the last 10 years as well as searches of the files of the authors themselves. The final list was generated on the basis of originality and relevance to this review.

Persistent Complete Response After Single-agent Sunitinib Treatment in a Case of TFE Translocation Positive Relapsed Metastatic Pediatric Renal Cell Carcinoma

Forty percent of renal cell carcinomas (RCCs) in childhood are characterized by translocation involving transcription factor E3 (TFE3) family members. Here, we describe a case of TFE3-positive RCC in which metastatic relapse to the mediastinal lymph nodes and pulmonary nodules was treated with single-agent sunitinib, a multitargeted tyrosine inhibitor. Complete radiologic remission was achieved after only 3 courses of treatment, and surgical exploration of metastases failed to identify any residual viable disease. The published experience of sunitinib in TFE-RCC is limited, and prospective evaluation of its activity in a larger number of patients is warranted.

Sustained Complete Remission of Metastatic Hepatocellular Carcinoma with Single Agent Sorafenib

Hepatocellular carcinoma (HCC) is the third highest cause of cancer-related death, with 626,000 deaths worldwide annually [1]. Additionally, the incidence rate in the developed world is increasing, with an estimated incidence of 81,000 new cases per year [1]. The standard treatment regimen for HCC is dependent on whether the patient has local or metastatic disease. Local disease is treated with radio-ablation, resection, trans-arterial chemo-embolization, and/or orthotopic liver transplant. These treatments can produce 5-year survival rates up to 60–70 % [2]. However, the average survival for patients with metastatic disease is 7.9 months [3]. Sorafenib is a small molecule serine/threonine/tyrosine kinase inhibitor that, in the 2008 landmark Sorafenib Hepatocellular Carcinoma Assessment Randomized Protocol (SHARP) trial, demonstrated an approximately 3-month increase in survival rate in the setting of metastatic HCC [3]. To date, only three published studies have demonstrated complete remission in the setting of advanced or metastatic HCC [4, 5, 6].We present a case of a patient with metastatic HCC who achieved sustained, complete biochemical remission and near-complete radiological remission for 42 months while on sorafenib.

Local therapy is critical in localised pelvic rhabdomyosarcoma: Experience of the International Society of Pediatric Oncology Malignant Mesenchymal Tumor (SIOP-MMT) committee

BackgroundLocalised pelvic rhabdomyosarcomas (pRMS) are rare tumours with a poorer prognosis than the majority of RMS. This study analysed patient outcome according to the type of local therapy delivered and the effect of disease-related factors on prognosis. Patients and methods97 children with localised pRMS were enrolled in the SIOP-MMT84, 89 and 95 studies. After primary surgery or biopsy, all children received ifosfamide/actinomycin/vincristine-based chemotherapy. Radiotherapy and surgery were planned in patients failing to achieve complete remission. ResultsMedian age at diagnosis was 52months [5months–18years]. IRS staging was I for five patients, II for 15 and III for 77. Patients had embryonal RMS (N=41), alveolar RMS (N=29), botryoid RMS (N=3), or not otherwise specified RMS (N=24). Outcome87 patients achieved local control (90%), 37 relapsed (43%), mainly locally (84%). With a median follow-up of more than 10years [4–22years], 5-year OS was 66% (95% CI: 56–75%) and EFS was 52% (95% CI: 42–61%). Among the 18 IRS-I/II patients treated without radiotherapy, 15 survived. Seven out of the 20 IRS-III patients treated without local therapy died. In multivariate analysis, IRS staging, age greater than 10years and lymph node involvement had a negative impact on OS. Perineal/perianal locations had a trend towards a worse prognosis. ConclusionpRMS still have a relatively poor prognosis. Radiotherapy or brachytherapy is necessary for all IRS-III patients including those with radiological complete remission after neoadjuvant chemotherapy with or without surgery. Radiotherapy may be withheld in IRS-I patients and children under 3years with IRS-II pRMS.