xRead - Nasal Obstruction (September 2024) Full Articles

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KUANetal.

TABLE XXIII.A.3 Evidence surrounding role of chemotherapy in pediatric rhabdomyosarcoma.

Clinical endpoints

Study

Year LOE Study Design Study groups

Conclusions

1. Improved EFS for PM-RMS but no difference in EFS or OS in entire cohort 2. No difference in grade 3/4 toxicities

1. OS 2. EFS 3. Grades 3/4 toxicities 1. OS 2. EFS 3. Toxicities

Four-drug (VAIA) ( n = 78) versus six-drug chemo

Sparber-Sauer et al. 1231

2022 2

Randomized controlled trial

(CEVAIE) ( n = 75) inPM-RMS

Oberlin et al. 1234 2012 2

Randomized controlled trial

IVA( n = 86) versus six-drug chemo (CEVAIE) combo ( n = 83) in PM-RMS All patients < 3years of age assigned to CEVAIE VAC( n = 302) versus VAC/VTC( n = 292) in intermediate-risk RMS VAC( n = 235) versus VAI ( n = 222) versus VIE( n = 236) in RMS, IRS stage 2/3 VAC( n = 58) versus VACA and cisplatin ( n = 113) versus VACA, cisplatin, and etoposide ( n = 118) in RMS IRSG 3

1. No difference in survival

between three- and six-drug regimens

2. Higher rates of grade 3/4 leukopenia, neutropenia, thrombocytopenia, and

stomatitis in six-drug regimen cohort including all nonmetastatic RMSs No overall survival difference between two chemotherapy regiments No difference according to chemotherapy regiment for cohort that included IRSG 2 and 3PM-RMS No difference in CRS or survival among the three chemotherapy regiments 1. 5-year OS of 67% in cohort of PM-RMS patients receiving chemotherapy 2. 5-year CR: 73% 3. No difference between two chemotherapy regiments 1. 5-year EFS of 65% for all PM-RMSs in cohort 2. Different chemotherapy regiment depending on RMS type and nodal disease

Arndt et al. 1233

OS

2009 2

Randomized controlled trial Randomized controlled trial Randomized controlled trial

Crist et al. 1226

2001 2

FFS

Crist et al. 1232

1. OS 2. PFS 3. CR

1995 2

Maurer et al. 1227

1993 2

Randomized controlled trial

Randomized

1. OS 2. CR

chemotherapy protocol Received VAC or

VACA( n = 144) in PM-RMS IRSG 3

EFS

Koscielniak et al. 1246

2022 3

Prospective cohort

Risk stratified. IVA: Standard

(embryonal, N0 PM-RMS). VAIA ± maintenance: other higher risk PM-RMS ( n = 114)

Arndt et al. 1233

2009 3

Prospective cohort

VAC( n = 101) in PM-RMS with intracranial extension

1. OS 2. FFS

4-year OS of 71% and 4-year FFS of 68% in patients with PM-RMS with intracranial extension

(Continues)

done cautiously. The majority of patients in these stud ies were IRSG 3, meaning they either did not have initial surgery or had unresectable disease. Additionally, other studies have shown that not all subsites of PM-RMS carry similar prognosis and have suggested subdivision into

favorable and unfavorable PM site. Unfavorable PM sites include paranasal sinus, infratemporal fossa, and PPF, while nasal cavity is a favorable PM site. 1224,1225 Intrathecal chemotherapy does not appear to confer survival benefit in high-risk PM-RMS.