Table 1 Clinical trial and real-world data on the use of ipilimumab in patient subpopulations

Elderly patients

Italian EAP ( >70 years)

DCR: 38%

Generally well tolerated; consistent with wider EAP

Chiarion Sileni et al., 2014 [48]

1- year OS: 38%

2-year OS: 22%

population

Spanish EAP (≥65 years)

DCR: 35%

No increase in toxicity in elderly patients

Lopez Martin et al., 2012 [49]

1- year OS: 21%

US EAP (≥65 years)

Consistent with wider EAP population

Lawrence et al., 2012 [50, 51]

1- year OS: 37%

NYU retrospective analysis

Chandra et al., 2013 [52]

(≥65 years)

Consistent with published data in younger cohorts

DCR: 36%

Uveal melanoma

Italian EAP

DCR: 34%

Safety profile similar to that in cutaneous melanoma

Maio et al., 2013 [53]

1- year OS: 31%

I-OMEAP (10 mg/kg)

Consistent with ipilimumab clinical trials

Danielli et al., 2012 [54]

DCR: 23%

Royal Marsden

Consistent with ipilimumab clinical trials

Khattak et al., 2013 [55]

DCR: 20%

US EAP 1- year OS: 34%

Consistent with wider EAP population

Lawrence et al., 2012 [50, 51]

Multicentre retrospective analysis

Luke et al., 2013 [56]

DCR: 46%

Consistent with ipilimumab clinical trials

Mucosal melanoma

Italian EAP

DCR: 36%

Safety profile similar to that in cutaneous melanoma

Del Vecchio et al., 2013 [57]

1- year OS: 35%

US EAP 1- year OS: 32%

Consistent with wider EAP population

Lawrence et al., 2012 [50, 51]

Multicentre experience

Postow et al., 2013 [58]

DCR: 27%

Consistent with ipilimumab clinical trials

Brain metastases

CA184-042 phase 2 trial (asymptomatic)

DCR: 25%

Safety results consistent with those previously reported in clinical trials

Margolin et al., 2012 [59]

1- year OS: 36%

2-year OS: 21%

NIBIT-M1 phase 2 trial (+fotemustine; asymptomatic)

DCR: 50%

1- year OS: 55%

AEs generally manageable and reversible

Di Giacomo et al., 2012 [60]

2-year OS: 39%

Di Giacomo et al., 2013 [61]

Italian EAP DCR: 27%

Safety results consistent with those previously reported in clinical trials

Queirolo et al., 2014 [62]

1- year OS: 20%

US EAP 1- year OS: 25%

Consistent with wider EAP population

Lawrence et al., 2012 [50, 51]

BRAF/NRAS-mutated melanoma

Phase 2 study CA184-004

(BRAF mutated vs BRAF wild-type)

Shahabi et al., 2012 [63]

DCR: 30% vs 35%

NIBIT-M1 phase 2 trial

(+fotemustine; asymptomatic)

Di Giacomo et al., 2013 [61]

(BRAF mutated vs BRAF wild-type)

DCR: 60% vs 46%

Italian EAP

(BRAF mutated vs BRAF wild-type)

Consistent regardless of BRAF and NRAS mutation status

Queirolo et al., 2014 [62]

DCR: 38% vs 39%

1-year OS: 48% vs 39%

(NRAS mutated vs NRAS wild-type)

DCR: 57% vs 49%

1-year OS: 43% vs 40%

4 institution retrospective analysis

(ipiliumab or tremelimumab)

Similar median OS between patients with BRAF/NRAS-mutated and BRAF/NRAS wild-type melanoma; trend towards improved OS in wild-type population without prior BRAFi/MEKi treatment

Mangana et al., 2013 [46]