TABLE 2

Clinical trials of moDC-based vaccination in AML

Case reports are excluded from this overview.

Ref.	Logistics										N	Clinical Results^{^a}
Ref.	Source/Isolation Method	Culture Time	Culture Medium	Differentiation	Maturation Regimen	Target Antigen	Antigen Source	Loading Method	Other Aspects	Admin. Route	N	Clinical Results^{^a}
Lee et al., 2004	PBSC harvest/CD14⁺ isolation	8–9 days	AIM V	GM-CSF+IL-4	TNF-α	NA	AML cell lysate^{^b}	Pulsing	/	SC/IV	2	Disease progression (2)
Kitawaki et al., 2011a	Leukapheresis/Elutriation	3 or 6 days	CellGro	GM-CSF+IL-4	OK-432	WT1, hTERT, etc.	Apoptotic AML cells^{^c}	Pulsing	adjuvant OK-432	ID	4	Disease stabilization (2)
Kitawaki et al., 2011a	Leukapheresis/Elutriation	3 or 6 days	CellGro	GM-CSF+IL-4	OK-432	WT1, hTERT, etc.	Apoptotic AML cells^{^c}	Pulsing	adjuvant OK-432	ID	4	↓ AML blast count (1)
Ogasawara and Ota, 2014; Ota and Ogasawara, 2014	Leukapheresis/Adherence	ND	ND	GM-CSF+IL-4	OK-432+PGE₂	WT1	Peptide	Pulsing	adjuvant OK-432	ID	7^{^d}	↓ WT1 mRNA level (2)
DiPersio et al., 2009; Khoury et al., 2010	Leukapheresis/ Adherence	ND	ND	Unspecified cytokine mix	Unspecified cytokine mix	hTERT	mRNA	Electroporation	+LAMP	ID	21	DFS of 79% [12m]
Van Tendeloo et al., 2010; Berneman et al., 2014	Leukapheresis/CD14⁺ isolation	8 days	CellGro +1% hAB	GM-CSF+IL-4	TNF-α+IL-1 +IL-6+PGE₂	WT1	mRNA	Electroporation	+/−LAMP	ID	30	Induction of CR (2/3)
												Induction of CMR (8/23)
												Continued CR [63m] (8)
												Disease stabilization (3)
Kitawaki et al., 2011b	Leukapheresis/Elutriation	ND	ND	GM-CSF+IL-4	TNF-α+PGE₂	WT1	Peptide	Pulsing	+ZOL	ID/IV	3	Disease stabilization (1)
Kitawaki et al., 2011b	Leukapheresis/Elutriation	ND	ND	GM-CSF+IL-4	TNF-α+PGE₂	WT1	Peptide	Pulsing	+ZOL	ID/IV	3	↓ AML blast count (1)
Rosenblatt et al., 2014	Leukapheresis/Adherence	7–10 days	ND	GM-CSF+IL-4	TNF-α	MUC1, WT1, PRAME, etc.	AML cells	Fusion hybrids (PEG)	adjuvant GM-CSF	SC	13	Continued CR [23m] (9)
Schnorfeil et al., 2014; Subklewe et al., 2014	Leukapheresis/Adherence	3 days	ND	GM-CSF+IL-4	R-848+poly(I:C)+other^{^e}	WT1, PRAME	mRNA	Electroporation	/	ID	2^{^f}	ND (ongoing study)

CD14⁺ isolation, magnetic isolation of CD14 monocytes; C(M)R, complete (molecular) remission; DFS, disease-free survival; hAB, human AB serum; hTERT, human telomerase reverse transcriptase; LAMP, inclusion of lysosome-associated membrane protein in mRNA construct; MUC1, mucin 1; ND, no data; PBSC, peripheral blood stem cell; PEG, polyethylene glycol; PRAME, preferentially expressed antigen in melanoma; ZOL, in combination with zoledronate.
↵^a The number in parentheses indicates the number of patients in whom the designated clinical effect was observed. The number in brackets indicates the follow up-time (in months).
↵^b Prepared by repeated freeze-thaw cycles.
↵^c Prepared by γ-irradiation.
↵^d Including 2 patients with acute lymphoblastic leukemia.
↵^e Including TNF-α, IL-1β, IFN-γ, PGE₂.
↵^f Schedules to include 20 patients.