Table 4.
Studies depicting outcomes of HSCT in JMML
| (n) Median age | Years of study | Source of Stem cells | Conditioning | GVHD prophylaxis | GVHD | OS% (follow up) [Reference] |
|---|---|---|---|---|---|---|
| (11) 18.6 m | 1998-2013 | BM = 4 | -Bu Cy Mel (8/11) | CSA + steroids (4) | Ac GVHD = 6/11 | 72.7 % (4.2 yr) [71] |
| UCB = 7 | -ATG in 5 | CSA+MMF(1) | Chr GVHD = 3/11 | |||
| (MRD = 3 | CSA+MTX(1) | |||||
| MUD = 1 | TAC+MTX(4) | |||||
| UCB = 7) | ||||||
| (11) 9 m | 1999-2004 | BM = 6 | -Bu Cy Etop (6/11) | CSA+MTX | Ac GVHD = 8/11 | 54.5% (15.5 m) [72] |
| UCB = 4 | -TBI in 3 | Chr GVHD = 4/11 | ||||
| PB = 1 | ||||||
| (15) 13.3 m | 2013-2015 | PB = 1 | -Bu Cy Mel | TAC+MMF | Gr I Ac GVHD = 20% | 47% (29 m) [73] |
| BM = 13 | -Bu Flu | Gr II-IV Ac GVHD = 27% | ||||
| UCB = 1 | -ATG in MUD and UCB HSCT | Chr GVHD = 7% | ||||
| (MRD-5 MUD = 7 | ||||||
| MMUD = 2 | ||||||
| UCB = 1) | ||||||
| (100) 30 m | 1993-2002 | PB = 14 | -Bu Cy Mel | CSA alone in MRD (32/48) | Gr II-IV Ac GVHD = 40/100 | 64% (5 yr) [47] |
| BM = 79 | CSA+MTX+ | Chr GVHD = 13/86 | ||||
| UCB = 7 | ATG+Mo ab for MUD (41/52) | |||||
| (MRD = 48 | ||||||
| MUD = 45 | ||||||
| UCB = 7) | ||||||
| (7) 30 m | 2007-2014 | BM = 3 | -Bu Mel | TAC+MTX | - | 100% (25.3 m) [74] |
| UCB = 3 | -Alemtuzumab in MUD | MTX not used in UCB | ||||
| Not mentioned = 1 | -ATG for UCB | |||||
| (MRD = 2 | ||||||
| MMRD = 1 | ||||||
| MUD = 1 | ||||||
| UCB = 3) | ||||||
| (27) 24 m | 1990-1997 | PB-3 | -Cy Bu Etop (Mel, Thiotepa, and Ara-C were other agents used in different patients) | MTX, CSA, and TAC (different combinations in different patients) | Gr II-IV Ac GVHD = 15/26 | 57.9+-11.0 (4 y) [75] |
| BM = 23 | -TBI in 18 | Chr GVHD = 10/21 | ||||
| UCB = 1 | ||||||
| (MRD = 12 | ||||||
| MMRD = 4 | ||||||
| UCB = 1 | ||||||
| MUD = 10) | ||||||
| (91) 16 m | 1986-2011 | Not mentioned | Bu Cy Mel | Not mentioned | Ac GVHD 48% | 59% (72 m) [69] |
| +- Serotherapy | ||||||
| (129) 24 m | 2000-2011 | PB = 10 | Bu Flu Mel in 59 | TAC+MTX in 73 | Gr II-IV Ac GVHD = 44% | 64% (5 y) [70] |
| BM = 89 | Bu Cy Ara-c in22 | CSA+MTX in 22 | Chr GVHD = 37/107 | |||
| UCB = 30 | TBI based in 20 | |||||
| (MRD = 44 | ||||||
| UD = 85) |
(BM: bone marrow; PB: peripheral blood; UCB: umbilical cord blood; MRD: matched related donor; MUD: matched unrelated donor; MMUD: mismatched unrelated donor; UD: unrelated donor; Bu: busulfan; Cy: cyclophosphamide; Mel: melphalan; Etop: etoposite; ATG: antithymocyte globulin; Flu: fludarabine; TBI: total body irradiation; Ara-C: Cytarabine; CSA: cyclosporine-A; MMF: mycophenolate mofetil; MTX: methotrexate; TAC: tacrolimus; Mo Ab: monoclonal antibodies; Ac GVHD: Acute graft versus host disease; Ch GVHD: chronic GVHD; Gr: grade; HSCT: Hematopoietic stem cell transplant).