Table 2.

Feasibility testing of the ESMO-MCBS v1.1 for acute lymphoblastic leukaemia (n=5)

Medication	Trial name	Setting	Primary outcome	PFS/EFS control	PFS/EFS gain	PFS/EFS HR	OS control	OS gain	OS HR	RR (DOR)	QOL	Toxicity	ESMO-MCBS score	ESMO-MCBS form	Reference (s)
Blinatumomab versus SOC	TOWER	Relapsed/refractory	OS	12% EFS 6 months	19%	0.55 (0.43–0.71)	4 months	3.7 months	0.71 (0.55– 0.93)	44% vs 25% CRR, gain 19%	Improved (+1 point)		5	2a	17 18
																Inotuzumab ozogamicin versus SOC	INO-VATE	Relapsed/refractory	OS/CRR	1.8 months	3.2 months	0.45 (97.5% CI: 0.34 to 0.61)	6.7 months (10% gain in 2-year survival)	1 month (13% gain in 2-year survival)	0.77 (97.5% CI: 0.58 to 1.03) p= 0.04	81% vs 29% CRR, gain 52%	Improved	Veno-occlusive disease 11% in experimental arm	4*	2a	19 20
Hyper-CVAD + ponatinib		Philadelphia chromosome-positive, upfront. Phase II single arm	EFS	81% 2 years EFS			80% 2 years						Not scoreable		21
																CAR T-cell tisagenlecleucel		Relapsed/refractory, age <21 years, single arm	ORR at 3 months				76% 1 year			81% ORR		>30% grade 3/4 cytokine release syndrome	3	3	22
Ponatinib	PACE	Philadelphia positive resistant to or side effects with dasatinib or nilotinib, or T315I mutation after TKI	Major haematological response within the first 6 months		7% at 12 months			40% at 12 months		Major haematological response: 41% (3 months)			2	3	23

*Based on >10% increase in 2 years of OS improvement.

CAR T- cell, chimeric antigen receptor T-cell therapy; CRR, complete remission rate; DOR, duration of response; EFS, event-free survival;ESMO-MCBS v1.1, European Society for Medical Oncology—Magnitude of Clinical Benefit Scale, version 1.1; Hyper-CVAD, hyperfractionated cyclophosphamide, vincristine, doxorubicin and dexamethasone; ORR, overall response rate; OS, overall survival;PFS, progression-free survival; QOL, quality of life; RR, response rate; SOC, standard of care; TKI, tyrosine kinase inhibitor.