Low-risk Group

The COG-D9602 study stratified 388 patients with low-risk embryonal rhabdomyosarcoma into two groups.[173] Treatment for subgroup A patients (n = 264; Stage 1 Group I/IIA, Stage 2 Group I, and Stage 1 Group III orbit) consisted of VA for 48 weeks with or without RT. Patients with subgroup B disease (n = 78; Stage 1 Group IIB/C, Stage I Group III nonorbit, Stage 2 Group II, and Stage 3 Group I/II disease) received VAC (total cumulative cyclophosphamide dose of 28.6 g/m2). Radiation doses were reduced from 41.4 Gy to 36 Gy for Stage 1 Group IIA patients and from 50 Gy or 59 Gy to 45 Gy for Group III orbit patients.

The COG-D9602 study stratified 388 patients with low-risk embryonal rhabdomyosarcoma into two groups.[173] Treatment for subgroup A patients (n = 264; Stage 1 Group I/IIA, Stage 2 Group I, and Stage 1 Group III orbit) consisted of VA for 48 weeks with or without RT. Patients with subgroup B disease (n = 78; Stage 1 Group IIB/C, Stage I Group III nonorbit, Stage 2 Group II, and Stage 3 Group I/II disease) received VAC (total cumulative cyclophosphamide dose of 28.6 g/m2). Radiation doses were reduced from 41.4 Gy to 36 Gy for Stage 1 Group IIA patients and from 50 Gy or 59 Gy to 45 Gy for Group III orbit patients.

For subgroup A patients, the 5-year overall FFS rate was 89%, and the OS rate was 97%.

The COG-D9602 study stratified 388 patients with low-risk embryonal rhabdomyosarcoma into two groups.[173] Treatment for subgroup A patients (n = 264; Stage 1 Group I/IIA, Stage 2 Group I, and Stage 1 Group III orbit) consisted of VA for 48 weeks with or without RT. Patients with subgroup B disease (n = 78; Stage 1 Group IIB/C, Stage I Group III nonorbit, Stage 2 Group II, and Stage 3 Group I/II disease) received VAC (total cumulative cyclophosphamide dose of 28.6 g/m2). Radiation doses were reduced from 41.4 Gy to 36 Gy for Stage 1 Group IIA patients and from 50 Gy or 59 Gy to 45 Gy for Group III orbit patients.

For subgroup B patients, the 5-year FFS rate was 85%, and the OS rate was 93%.

The COG-ARST0331 trial evaluated a refinement of therapy for two subsets of low-risk patients.[55] For subset 1 patients, this study reduced the length of therapy by using only four cycles of VAC (cumulative cyclophosphamide dose of 4.8 g/m2) followed by four VA cycles over 22 weeks. Group II and III patients received local RT. For subset 2 patients, the goal of this study was to reduce the total cumulative cyclophosphamide dose, compared with the previous IRS-IV study, without compromising FFS, and to decrease the risk of permanent infertility. Patients received four cycles of VAC (equivalent cyclophosphamide dose as subset 1) followed by VA over 46 weeks.[54][Level of evidence B4]

The COG-ARST0331 trial evaluated a refinement of therapy for two subsets of low-risk patients.[55] For subset 1 patients, this study reduced the length of therapy by using only four cycles of VAC (cumulative cyclophosphamide dose of 4.8 g/m2) followed by four VA cycles over 22 weeks. Group II and III patients received local RT. For subset 2 patients, the goal of this study was to reduce the total cumulative cyclophosphamide dose, compared with the previous IRS-IV study, without compromising FFS, and to decrease the risk of permanent infertility. Patients received four cycles of VAC (equivalent cyclophosphamide dose as subset 1) followed by VA over 46 weeks.[54][Level of evidence B4]

Subset 1 enrolled 271 newly diagnosed patients with low-risk embryonal rhabdomyosarcoma, defined as patients who presented with Stage 1 or Stage 2 tumors; Group I or Group II tumors; or Stage 1, Group III orbital tumors. This noninferiority trial used a fixed outcome on the basis of expected FFS for similar patients treated in the D9602 trial.[55]

The COG-ARST0331 trial evaluated a refinement of therapy for two subsets of low-risk patients.[55] For subset 1 patients, this study reduced the length of therapy by using only four cycles of VAC (cumulative cyclophosphamide dose of 4.8 g/m2) followed by four VA cycles over 22 weeks. Group II and III patients received local RT. For subset 2 patients, the goal of this study was to reduce the total cumulative cyclophosphamide dose, compared with the previous IRS-IV study, without compromising FFS, and to decrease the risk of permanent infertility. Patients received four cycles of VAC (equivalent cyclophosphamide dose as subset 1) followed by VA over 46 weeks.[54][Level of evidence B4] › Subset 1 enrolled 271 newly diagnosed patients with low-risk embryonal rhabdomyosarcoma, defined as patients who presented with Stage 1 or Stage 2 tumors; Group I or Group II tumors; or Stage 1, Group III orbital tumors. This noninferiority trial used a fixed outcome on the basis of expected FFS for similar patients treated in the D9602 trial.[55]

There were 35 treatment failures observed (48.8 expected).

The COG-ARST0331 trial evaluated a refinement of therapy for two subsets of low-risk patients.[55] For subset 1 patients, this study reduced the length of therapy by using only four cycles of VAC (cumulative cyclophosphamide dose of 4.8 g/m2) followed by four VA cycles over 22 weeks. Group II and III patients received local RT. For subset 2 patients, the goal of this study was to reduce the total cumulative cyclophosphamide dose, compared with the previous IRS-IV study, without compromising FFS, and to decrease the risk of permanent infertility. Patients received four cycles of VAC (equivalent cyclophosphamide dose as subset 1) followed by VA over 46 weeks.[54][Level of evidence B4] › Subset 1 enrolled 271 newly diagnosed patients with low-risk embryonal rhabdomyosarcoma, defined as patients who presented with Stage 1 or Stage 2 tumors; Group I or Group II tumors; or Stage 1, Group III orbital tumors. This noninferiority trial used a fixed outcome on the basis of expected FFS for similar patients treated in the D9602 trial.[55]

The 3-year FFS rate was 89%, and the OS rate was 98%. Thus, shorter duration of therapy did not appear to compromise outcome in these patients.

The COG-ARST0331 trial evaluated a refinement of therapy for two subsets of low-risk patients.[55] For subset 1 patients, this study reduced the length of therapy by using only four cycles of VAC (cumulative cyclophosphamide dose of 4.8 g/m2) followed by four VA cycles over 22 weeks. Group II and III patients received local RT. For subset 2 patients, the goal of this study was to reduce the total cumulative cyclophosphamide dose, compared with the previous IRS-IV study, without compromising FFS, and to decrease the risk of permanent infertility. Patients received four cycles of VAC (equivalent cyclophosphamide dose as subset 1) followed by VA over 46 weeks.[54][Level of evidence B4]

Subset 2 included patients with Stage 1, Group III nonorbital tumors or Stage 3, Group I/II embryonal tumors. Treatment consisted of four cycles of VAC chemotherapy followed by 12 cycles of VA therapy.[53,54]

The COG-ARST0331 trial evaluated a refinement of therapy for two subsets of low-risk patients.[55] For subset 1 patients, this study reduced the length of therapy by using only four cycles of VAC (cumulative cyclophosphamide dose of 4.8 g/m2) followed by four VA cycles over 22 weeks. Group II and III patients received local RT. For subset 2 patients, the goal of this study was to reduce the total cumulative cyclophosphamide dose, compared with the previous IRS-IV study, without compromising FFS, and to decrease the risk of permanent infertility. Patients received four cycles of VAC (equivalent cyclophosphamide dose as subset 1) followed by VA over 46 weeks.[54][Level of evidence B4] › Subset 2 included patients with Stage 1, Group III nonorbital tumors or Stage 3, Group I/II embryonal tumors. Treatment consisted of four cycles of VAC chemotherapy followed by 12 cycles of VA therapy.[53,54]

Among 66 eligible patients, there were 20 failures, with an estimated 3-year FFS rate of 70% and an OS rate of 92%.

The COG-ARST0331 trial evaluated a refinement of therapy for two subsets of low-risk patients.[55] For subset 1 patients, this study reduced the length of therapy by using only four cycles of VAC (cumulative cyclophosphamide dose of 4.8 g/m2) followed by four VA cycles over 22 weeks. Group II and III patients received local RT. For subset 2 patients, the goal of this study was to reduce the total cumulative cyclophosphamide dose, compared with the previous IRS-IV study, without compromising FFS, and to decrease the risk of permanent infertility. Patients received four cycles of VAC (equivalent cyclophosphamide dose as subset 1) followed by VA over 46 weeks.[54][Level of evidence B4] › Subset 2 included patients with Stage 1, Group III nonorbital tumors or Stage 3, Group I/II embryonal tumors. Treatment consisted of four cycles of VAC chemotherapy followed by 12 cycles of VA therapy.[53,54]

FFS rates at 3 years were even worse (57%) for girls with genital tract tumors.

The COG-ARST0331 trial evaluated a refinement of therapy for two subsets of low-risk patients.[55] For subset 1 patients, this study reduced the length of therapy by using only four cycles of VAC (cumulative cyclophosphamide dose of 4.8 g/m2) followed by four VA cycles over 22 weeks. Group II and III patients received local RT. For subset 2 patients, the goal of this study was to reduce the total cumulative cyclophosphamide dose, compared with the previous IRS-IV study, without compromising FFS, and to decrease the risk of permanent infertility. Patients received four cycles of VAC (equivalent cyclophosphamide dose as subset 1) followed by VA over 46 weeks.[54][Level of evidence B4] › Subset 2 included patients with Stage 1, Group III nonorbital tumors or Stage 3, Group I/II embryonal tumors. Treatment consisted of four cycles of VAC chemotherapy followed by 12 cycles of VA therapy.[53,54]

Using reduced total cyclophosphamide, researchers observed suboptimal FFS rates among patients with subset 2 low-risk rhabdomyosarcoma. Eliminating RT for girls with Group III vaginal tumors in combination with reduced total cyclophosphamide appeared to contribute to the suboptimal outcome. However, the OS rate appeared to be similar to the OS rate in previous studies with higher-dose cyclophosphamide. These patients (Stage I, Group III nonorbit and Stage 3, Group I/II) are now being treated in the intermediate-risk ARST1431 (NCT02567435) trial.

The COG-ARST0331 trial evaluated a refinement of therapy for two subsets of low-risk patients.[55] For subset 1 patients, this study reduced the length of therapy by using only four cycles of VAC (cumulative cyclophosphamide dose of 4.8 g/m2) followed by four VA cycles over 22 weeks. Group II and III patients received local RT. For subset 2 patients, the goal of this study was to reduce the total cumulative cyclophosphamide dose, compared with the previous IRS-IV study, without compromising FFS, and to decrease the risk of permanent infertility. Patients received four cycles of VAC (equivalent cyclophosphamide dose as subset 1) followed by VA over 46 weeks.[54][Level of evidence B4]

For patients with an orbital primary tumor who achieved only a partial response or stability after 12 weeks of induction chemotherapy, the 5-year FFS rate was only 84%, compared with 100% for patients who achieved a CR.[71][Level of evidence C2]

The EpSSG RMS-2005 study prospectively evaluated the reduction in chemotherapy for patients with low-risk embryonal histology rhabdomyosarcoma. The study enrolled patients from October 2005 to December 2016.

The EpSSG RMS-2005 study prospectively evaluated the reduction in chemotherapy for patients with low-risk embryonal histology rhabdomyosarcoma. The study enrolled patients from October 2005 to December 2016.

The study enrolled 178 patients with Group 1 N0 disease (subgroups A and B).[174][Level of evidence B4]

The EpSSG RMS-2005 study prospectively evaluated the reduction in chemotherapy for patients with low-risk embryonal histology rhabdomyosarcoma. The study enrolled patients from October 2005 to December 2016. › The study enrolled 178 patients with Group 1 N0 disease (subgroups A and B).[174][Level of evidence B4]

The 5-year EFS rate was 90.8% (95% CI, 85.0%–94.4%), and the OS rate was 95.7% (95% CI, 90.5%–98.1%).

The EpSSG RMS-2005 study prospectively evaluated the reduction in chemotherapy for patients with low-risk embryonal histology rhabdomyosarcoma. The study enrolled patients from October 2005 to December 2016. › The study enrolled 178 patients with Group 1 N0 disease (subgroups A and B).[174][Level of evidence B4]

Subgroup A: Patients younger than 10 years with tumors smaller than 5 cm received eight courses of VA therapy for 22 weeks. The EFS rate for this subgroup of 70 patients was 95.5% (95% CI, 86.8%–98.5%), and the OS rate was 100%.

The EpSSG RMS-2005 study prospectively evaluated the reduction in chemotherapy for patients with low-risk embryonal histology rhabdomyosarcoma. The study enrolled patients from October 2005 to December 2016. › The study enrolled 178 patients with Group 1 N0 disease (subgroups A and B).[174][Level of evidence B4]

Subgroup B: Patients who were older than 10 years or had tumors larger than 5 cm received four courses of IVA (VA plus ifosfamide) and five courses of VA for 25 weeks. The EFS rate for this subgroup was 87.8% (95% CI, 79.3%–93.0%), and the OS rate was 93% (95% CI, 84.8%–96.8%).

The EpSSG RMS-2005 study prospectively evaluated the reduction in chemotherapy for patients with low-risk embryonal histology rhabdomyosarcoma. The study enrolled patients from October 2005 to December 2016. › The study enrolled 178 patients with Group 1 N0 disease (subgroups A and B).[174][Level of evidence B4]

Treatment with VA for eight courses was effective and well tolerated by subgroup A patients with low-risk embryonal rhabdomyosarcoma. A reduction from nine courses of IVA in previous studies to four courses of IVA plus five courses of VA also produced good results.

The EpSSG RMS-2005 study prospectively evaluated the reduction in chemotherapy for patients with low-risk embryonal histology rhabdomyosarcoma. The study enrolled patients from October 2005 to December 2016.

The study enrolled 359 patients in subgroup C. Patients in subgroup C had localized, node-negative, IRS Group II/III, nonalveolar rhabdomyosarcoma at favorable sites: orbit (164; 45.7%), head and neck nonparameningeal (77; 21.4%), and genitourinary nonbladder/prostate (118; 32.9%).[175][Level of evidence B4]

The EpSSG RMS-2005 study prospectively evaluated the reduction in chemotherapy for patients with low-risk embryonal histology rhabdomyosarcoma. The study enrolled patients from October 2005 to December 2016. › The study enrolled 359 patients in subgroup C. Patients in subgroup C had localized, node-negative, IRS Group II/III, nonalveolar rhabdomyosarcoma at favorable sites: orbit (164; 45.7%), head and neck nonparameningeal (77; 21.4%), and genitourinary nonbladder/prostate (118; 32.9%).[175][Level of evidence B4]

The 5-year EFS rate was 77.4% (95% CI, 72.5%–81.6%), and the OS rate was 93.5% (95% CI, 90.1%–95.8%).

The EpSSG RMS-2005 study prospectively evaluated the reduction in chemotherapy for patients with low-risk embryonal histology rhabdomyosarcoma. The study enrolled patients from October 2005 to December 2016. › The study enrolled 359 patients in subgroup C. Patients in subgroup C had localized, node-negative, IRS Group II/III, nonalveolar rhabdomyosarcoma at favorable sites: orbit (164; 45.7%), head and neck nonparameningeal (77; 21.4%), and genitourinary nonbladder/prostate (118; 32.9%).[175][Level of evidence B4]

Patients were to receive nine cycles of chemotherapy. Those who were receiving local primary tumor radiation therapy were to receive five cycles of IVA followed by four cycles of VA. For patients who were not receiving radiation therapy, nine cycles of IVA were planned. The actual treatments delivered were:

The EpSSG RMS-2005 study prospectively evaluated the reduction in chemotherapy for patients with low-risk embryonal histology rhabdomyosarcoma. The study enrolled patients from October 2005 to December 2016. › The study enrolled 359 patients in subgroup C. Patients in subgroup C had localized, node-negative, IRS Group II/III, nonalveolar rhabdomyosarcoma at favorable sites: orbit (164; 45.7%), head and neck nonparameningeal (77; 21.4%), and genitourinary nonbladder/prostate (118; 32.9%).[175][Level of evidence B4] › Patients were to receive nine cycles of chemotherapy. Those who were receiving local primary tumor radiation therapy were to receive five cycles of IVA followed by four cycles of VA. For patients who were not receiving radiation therapy, nine cycles of IVA were planned. The actual treatments delivered were:

Lower-dose alkylator chemotherapy (n = 7).

The EpSSG RMS-2005 study prospectively evaluated the reduction in chemotherapy for patients with low-risk embryonal histology rhabdomyosarcoma. The study enrolled patients from October 2005 to December 2016. › The study enrolled 359 patients in subgroup C. Patients in subgroup C had localized, node-negative, IRS Group II/III, nonalveolar rhabdomyosarcoma at favorable sites: orbit (164; 45.7%), head and neck nonparameningeal (77; 21.4%), and genitourinary nonbladder/prostate (118; 32.9%).[175][Level of evidence B4] › Patients were to receive nine cycles of chemotherapy. Those who were receiving local primary tumor radiation therapy were to receive five cycles of IVA followed by four cycles of VA. For patients who were not receiving radiation therapy, nine cycles of IVA were planned. The actual treatments delivered were:

Lower-dose alkylator chemotherapy and radiation therapy (n = 132).

The EpSSG RMS-2005 study prospectively evaluated the reduction in chemotherapy for patients with low-risk embryonal histology rhabdomyosarcoma. The study enrolled patients from October 2005 to December 2016. › The study enrolled 359 patients in subgroup C. Patients in subgroup C had localized, node-negative, IRS Group II/III, nonalveolar rhabdomyosarcoma at favorable sites: orbit (164; 45.7%), head and neck nonparameningeal (77; 21.4%), and genitourinary nonbladder/prostate (118; 32.9%).[175][Level of evidence B4] › Patients were to receive nine cycles of chemotherapy. Those who were receiving local primary tumor radiation therapy were to receive five cycles of IVA followed by four cycles of VA. For patients who were not receiving radiation therapy, nine cycles of IVA were planned. The actual treatments delivered were:

Higher-dose alkylator chemotherapy (n = 113).

The EpSSG RMS-2005 study prospectively evaluated the reduction in chemotherapy for patients with low-risk embryonal histology rhabdomyosarcoma. The study enrolled patients from October 2005 to December 2016. › The study enrolled 359 patients in subgroup C. Patients in subgroup C had localized, node-negative, IRS Group II/III, nonalveolar rhabdomyosarcoma at favorable sites: orbit (164; 45.7%), head and neck nonparameningeal (77; 21.4%), and genitourinary nonbladder/prostate (118; 32.9%).[175][Level of evidence B4] › Patients were to receive nine cycles of chemotherapy. Those who were receiving local primary tumor radiation therapy were to receive five cycles of IVA followed by four cycles of VA. For patients who were not receiving radiation therapy, nine cycles of IVA were planned. The actual treatments delivered were:

Higher-dose alkylator chemotherapy and radiation therapy (n = 100).

The EpSSG RMS-2005 study prospectively evaluated the reduction in chemotherapy for patients with low-risk embryonal histology rhabdomyosarcoma. The study enrolled patients from October 2005 to December 2016. › The study enrolled 359 patients in subgroup C. Patients in subgroup C had localized, node-negative, IRS Group II/III, nonalveolar rhabdomyosarcoma at favorable sites: orbit (164; 45.7%), head and neck nonparameningeal (77; 21.4%), and genitourinary nonbladder/prostate (118; 32.9%).[175][Level of evidence B4]

Delayed primary tumor excision was considered for patients with IRS stage III tumors.

The EpSSG RMS-2005 study prospectively evaluated the reduction in chemotherapy for patients with low-risk embryonal histology rhabdomyosarcoma. The study enrolled patients from October 2005 to December 2016. › The study enrolled 359 patients in subgroup C. Patients in subgroup C had localized, node-negative, IRS Group II/III, nonalveolar rhabdomyosarcoma at favorable sites: orbit (164; 45.7%), head and neck nonparameningeal (77; 21.4%), and genitourinary nonbladder/prostate (118; 32.9%).[175][Level of evidence B4]

Lower-dose alkylator chemotherapy and radiation therapy achieved a 5-year OS rate of 93.7%, but the comparison with higher-dose alkylator chemotherapy with or without radiation therapy was not significant (P = .8003). Adjuvant radiation therapy improved the 5-year EFS rate (84.7% vs. 65.2% without radiation therapy; P < .0001), but not OS (P = .9298).

The EpSSG RMS-2005 study prospectively evaluated the reduction in chemotherapy for patients with low-risk embryonal histology rhabdomyosarcoma. The study enrolled patients from October 2005 to December 2016. › The study enrolled 359 patients in subgroup C. Patients in subgroup C had localized, node-negative, IRS Group II/III, nonalveolar rhabdomyosarcoma at favorable sites: orbit (164; 45.7%), head and neck nonparameningeal (77; 21.4%), and genitourinary nonbladder/prostate (118; 32.9%).[175][Level of evidence B4]

Omitting radiation therapy for orbital tumors reduced the 5-year OS rate (87.1% vs. 97.3% for those who received radiation therapy; P = .0257).

The EpSSG RMS-2005 study prospectively evaluated the reduction in chemotherapy for patients with low-risk embryonal histology rhabdomyosarcoma. The study enrolled patients from October 2005 to December 2016. › The study enrolled 359 patients in subgroup C. Patients in subgroup C had localized, node-negative, IRS Group II/III, nonalveolar rhabdomyosarcoma at favorable sites: orbit (164; 45.7%), head and neck nonparameningeal (77; 21.4%), and genitourinary nonbladder/prostate (118; 32.9%).[175][Level of evidence B4]

After an R0 resection (negative margins) (n = 60), radiation therapy did not significantly improve EFS or OS.

The COG and EpSSG studies defined low-risk patient populations, largely based on histology. Genomic classification refined the risk classification for rhabdomyosarcoma and will be used in future COG studies.[176] Tumor samples from patients enrolled in COG trials (1988–2017), U.K. MMT, and RMS-2005 studies (1995–2016) were subjected to custom-capture sequencing. DNA from 641 patients was suitable for analysis. Variants, indels, gene deletions, and amplifications were identified, and survival analysis was performed.

The COG and EpSSG studies defined low-risk patient populations, largely based on histology. Genomic classification refined the risk classification for rhabdomyosarcoma and will be used in future COG studies.[176] Tumor samples from patients enrolled in COG trials (1988–2017), U.K. MMT, and RMS-2005 studies (1995–2016) were subjected to custom-capture sequencing. DNA from 641 patients was suitable for analysis. Variants, indels, gene deletions, and amplifications were identified, and survival analysis was performed.

A median of one variant per tumor was found.

The COG and EpSSG studies defined low-risk patient populations, largely based on histology. Genomic classification refined the risk classification for rhabdomyosarcoma and will be used in future COG studies.[176] Tumor samples from patients enrolled in COG trials (1988–2017), U.K. MMT, and RMS-2005 studies (1995–2016) were subjected to custom-capture sequencing. DNA from 641 patients was suitable for analysis. Variants, indels, gene deletions, and amplifications were identified, and survival analysis was performed.

In FOXO1 fusion–negative cases, any variant of RAS pathway members was found in more than 50% of cases. In 21% of cases, no putative driver variant was identified.

The COG and EpSSG studies defined low-risk patient populations, largely based on histology. Genomic classification refined the risk classification for rhabdomyosarcoma and will be used in future COG studies.[176] Tumor samples from patients enrolled in COG trials (1988–2017), U.K. MMT, and RMS-2005 studies (1995–2016) were subjected to custom-capture sequencing. DNA from 641 patients was suitable for analysis. Variants, indels, gene deletions, and amplifications were identified, and survival analysis was performed.

Variants in BCOR (15%), NF1 (15%), and TP53 (13%) were found at a higher incidence than previously reported.

The COG and EpSSG studies defined low-risk patient populations, largely based on histology. Genomic classification refined the risk classification for rhabdomyosarcoma and will be used in future COG studies.[176] Tumor samples from patients enrolled in COG trials (1988–2017), U.K. MMT, and RMS-2005 studies (1995–2016) were subjected to custom-capture sequencing. DNA from 641 patients was suitable for analysis. Variants, indels, gene deletions, and amplifications were identified, and survival analysis was performed.

TP53 variants were associated with worse outcomes in both fusion-negative and fusion-positive cases.

The COG and EpSSG studies defined low-risk patient populations, largely based on histology. Genomic classification refined the risk classification for rhabdomyosarcoma and will be used in future COG studies.[176] Tumor samples from patients enrolled in COG trials (1988–2017), U.K. MMT, and RMS-2005 studies (1995–2016) were subjected to custom-capture sequencing. DNA from 641 patients was suitable for analysis. Variants, indels, gene deletions, and amplifications were identified, and survival analysis was performed.

Variants of MYOD1 were associated with a dismal survival.

The COG and European investigators pooled the results of patients with rhabdomyosarcoma who had definitive surgery of the primary tumor before the initiation of systemic chemotherapy.[177] A total of 113 patients aged 0 to 18 years were identified and enrolled from January 1995 to December 2016 in COG (n = 64) and European protocols. Patients with genitourinary nonbladder and prostate rhabdomyosarcomas were excluded. The recommended chemotherapy in the European protocols was VA for 24 weeks or ifosfamide plus VA. The COG protocols recommended VA for 48 weeks or VA plus cyclophosphamide.

The COG and European investigators pooled the results of patients with rhabdomyosarcoma who had definitive surgery of the primary tumor before the initiation of systemic chemotherapy.[177] A total of 113 patients aged 0 to 18 years were identified and enrolled from January 1995 to December 2016 in COG (n = 64) and European protocols. Patients with genitourinary nonbladder and prostate rhabdomyosarcomas were excluded. The recommended chemotherapy in the European protocols was VA for 24 weeks or ifosfamide plus VA. The COG protocols recommended VA for 48 weeks or VA plus cyclophosphamide.

With a median follow-up of 97.5 months, the 5-year PFS rate was 80.0% (71.2%–86.4%), and the OS rate was 92.5% (85.6%–96.2%). There were no significant differences in outcomes between the chemotherapy regimens.

The COG and European investigators pooled the results of patients with rhabdomyosarcoma who had definitive surgery of the primary tumor before the initiation of systemic chemotherapy.[177] A total of 113 patients aged 0 to 18 years were identified and enrolled from January 1995 to December 2016 in COG (n = 64) and European protocols. Patients with genitourinary nonbladder and prostate rhabdomyosarcomas were excluded. The recommended chemotherapy in the European protocols was VA for 24 weeks or ifosfamide plus VA. The COG protocols recommended VA for 48 weeks or VA plus cyclophosphamide.

Tumor size (<5 cm vs. >5 cm) significantly influenced OS (96.2% [88.6%–98.8%] vs. 80.6% [59.5%–91.4%]; P = .01).

The COG and European investigators pooled the results of patients with rhabdomyosarcoma who had definitive surgery of the primary tumor before the initiation of systemic chemotherapy.[177] A total of 113 patients aged 0 to 18 years were identified and enrolled from January 1995 to December 2016 in COG (n = 64) and European protocols. Patients with genitourinary nonbladder and prostate rhabdomyosarcomas were excluded. The recommended chemotherapy in the European protocols was VA for 24 weeks or ifosfamide plus VA. The COG protocols recommended VA for 48 weeks or VA plus cyclophosphamide.

The authors suggested that to reduce the burden of treatment, VA for 24 weeks may be considered in children with tumors smaller than 5 cm.