Summary of major reports on HCT outcomes in MF
| Reference . | Timeline of HCT . | N . | Median age, y (range) . | Conditioning regimen . | % of patients with RIC . | % with MRD . | NRM . | PFS . | OS . | Comment . |
|---|---|---|---|---|---|---|---|---|---|---|
| Guardiola11 | 1979-1997 | 55 | 42 (4-53) | TBI based (63%) | 0 | 90 | 27% at 1 y | 39% at 5 y | 47% at 5 y | Hb < 10 g/dL and osteomyelosclerosis associated with lower survival |
| Deeg10 | 1980-2002 | 56 | 43 (10-66) | Bu/Cy in 78% | 0 | 64 | 14% at 3 mo | NR | 58 at 3 y | Targeted Bu use improved survival; cGVHD 59% at 2 y |
| Daly9 | 1990-2002 | 25 | 48 (45-50) | TBI based (92%) | 0 | 52 | 48% at 1 y | NR | 41 at 2 y | Prohibitive NRM; no benefit of splenectomy |
| Rondelli66 | NR | 21 | 54 (27-68) | Multiple | 100 | 85 | 10% at 1 y | 81% at 2.7 y | 85% at 2.7 y | Extensive cGVHD in 44%; 2 patients needed DLI for 100% donor chimerism; resolution of fibrosis and splenomegaly in majority |
| Kerbauy58 | NR | 104 | 49 (18-70) | Multiple, Bu/Cy (62%) | 9 | 50 | 35% at 5 y | NR | 61% at 5 y | 3 syngeneic donors, 54 of the patients overlapped with a prior report10 ; targeted Bu improved OS; comorbidity score had impact on survival |
| Patriarca65 | 1986-2006 | 100 | 49 (21-68) | Multiple, Bu/Cy 50% of full intensity; Thiotepa + Cy in 46% of RIC | 52 | 78 | 43% at 3 y | 35% at 3 y | 42% at 3 y | AHCT before 1995; unrelated donor and longer interval from diagnosis predicted worse outcome but not conditioning intensity; relapse at 2 y 41%, progressive decline in NRM over 20 y studied |
| Kroger54 | 2002-2007 | 103 | 55 (32-68) | Flu-Bu (100%) | 100 | 32 | 16% at 1 y | 51% at 5 y | 67% at 5 y | First prospective study in MF, cGVHD in 43%; 12% NRM for fully matched donor AHCT; age > 55 y and HLA mismatch adversely affected OS; JAK2-positive recipients had better EFS and OS; splenectomy increased risk of relapse |
| Gupta64 | 1998-2005 | 46 | 47 y MAC; 54 y RIC | Multiple, Cy TBI (96%) for MAC; Flu Bu (70%) for RIC | 50 | 54 | 48% for MAC and 27% for RIC at 3 y | 43% for MAC and 58% for RIC at 3 y | 48% for MAC and 68% for RIC at 3 y | RIC recipients had more advanced disease and poor KPS; low risk of relapse after either conditioning; lower GVHD with novel conditioning possibly related to use of ATG |
| Ballen56 | 1989-2002 | 289 | 47 (18-73) | Multiple, Bu/Cy (43%) | 21 | 56 | 35% siblings 50% for URD at 5 y | 33% siblings 27% for URD at 5 y | 37% siblings 30% for URD at 5 y | Relapse at 5 y, 32% in sibling and 23% in URD; performance status, peripheral blasts sibling donor status impacted OS; RIC was similar in outcomes, except early NRM |
| Alchalby71 | 1999-2009 | 162 | 56 (32-73) | Flu-Bu in 96% | 100 | 27 | 22% at 1 y | 46% at 5 y | 62% at 5 y | 82 patients reported previously54 ; age and HLA mismatch impacted NRM; 23% relapse at 3 y; clearance of mutated JAK2 at median of 96 days, and this reduced relapse risk |
| Bacigalupo63 | 1994-2007 | 46 | 51 (24-67) | Thiotepa-Cy + melphalan | 100 | 65 | 24% at 5 y | NR | 45% at 5 y | A risk score based on transfusion history, spleen > 22 cm and alternative donor use predicted lower OS; no benefit for splenectomy |
| Stewart76 | 1989-2005 | 51 | 49 (19-64) | Multiple, RIC in 47% | 47 | 65 | 41% at 2 y | 44% and 24% at 3 y for MAC and RIC | 44% and 31% at 3 y for MAC and RIC | |
| Robin92 | 1997-2008 | 147 | 53 (20-68) | Multiple | 69 | 61 | 39% at 4 y | 32% at 4 y | 39% at 4 y | 19% patients had LT; poor outcome with mismatched donor |
| Samuelson13 | 1999-2007 | 30 | 65 (60-78) | Multiple | 63 | 50 | 13% at day 100 | 40% at 3 y | 45% at 3 y | Studied outcomes in patients ≥ 60 y, 7 patients had preceding LT |
| Abelsson67 | 1982-2009 | 92 | 46 for MAC, 55 for RIC | Multiple | 56 | 40 | 32% for MAC and 24% for RIC at 2 y | NR | 49% for MAC and 59% for RIC at 5 y | Overall NRM similar, but 5-year OS superior for RIC younger than age 60 years; less advanced MF associated with better OS |
| Nivison-Smith69 | 1993-2005 | 57 | 47 (16-71) | Multiple | 26 | 68 | 25% at 1 y | 58% at 5 y | Poor outcome in patients > 50 y | |
| Ditschkowski70 | 1994-2010 | 76 | 50.5 (22-67) | Multiple | NR | 35 | 36% at 5 y | 50% at 5 y | 53% at 5 y | Significant high risk of relapse in patients without cGvHD; |
| DIPSS was predictive of survival | ||||||||||
| Scott57 | 1990-2009 | 170 | 51.5 (12-78) | Multiple | NR | 50 | 34% at 5 y | 57% at 5 y | 57% at 5 y | Post-HCT success was dependent on pre-HCT DIPSS scores |
| Reference . | Timeline of HCT . | N . | Median age, y (range) . | Conditioning regimen . | % of patients with RIC . | % with MRD . | NRM . | PFS . | OS . | Comment . |
|---|---|---|---|---|---|---|---|---|---|---|
| Guardiola11 | 1979-1997 | 55 | 42 (4-53) | TBI based (63%) | 0 | 90 | 27% at 1 y | 39% at 5 y | 47% at 5 y | Hb < 10 g/dL and osteomyelosclerosis associated with lower survival |
| Deeg10 | 1980-2002 | 56 | 43 (10-66) | Bu/Cy in 78% | 0 | 64 | 14% at 3 mo | NR | 58 at 3 y | Targeted Bu use improved survival; cGVHD 59% at 2 y |
| Daly9 | 1990-2002 | 25 | 48 (45-50) | TBI based (92%) | 0 | 52 | 48% at 1 y | NR | 41 at 2 y | Prohibitive NRM; no benefit of splenectomy |
| Rondelli66 | NR | 21 | 54 (27-68) | Multiple | 100 | 85 | 10% at 1 y | 81% at 2.7 y | 85% at 2.7 y | Extensive cGVHD in 44%; 2 patients needed DLI for 100% donor chimerism; resolution of fibrosis and splenomegaly in majority |
| Kerbauy58 | NR | 104 | 49 (18-70) | Multiple, Bu/Cy (62%) | 9 | 50 | 35% at 5 y | NR | 61% at 5 y | 3 syngeneic donors, 54 of the patients overlapped with a prior report10 ; targeted Bu improved OS; comorbidity score had impact on survival |
| Patriarca65 | 1986-2006 | 100 | 49 (21-68) | Multiple, Bu/Cy 50% of full intensity; Thiotepa + Cy in 46% of RIC | 52 | 78 | 43% at 3 y | 35% at 3 y | 42% at 3 y | AHCT before 1995; unrelated donor and longer interval from diagnosis predicted worse outcome but not conditioning intensity; relapse at 2 y 41%, progressive decline in NRM over 20 y studied |
| Kroger54 | 2002-2007 | 103 | 55 (32-68) | Flu-Bu (100%) | 100 | 32 | 16% at 1 y | 51% at 5 y | 67% at 5 y | First prospective study in MF, cGVHD in 43%; 12% NRM for fully matched donor AHCT; age > 55 y and HLA mismatch adversely affected OS; JAK2-positive recipients had better EFS and OS; splenectomy increased risk of relapse |
| Gupta64 | 1998-2005 | 46 | 47 y MAC; 54 y RIC | Multiple, Cy TBI (96%) for MAC; Flu Bu (70%) for RIC | 50 | 54 | 48% for MAC and 27% for RIC at 3 y | 43% for MAC and 58% for RIC at 3 y | 48% for MAC and 68% for RIC at 3 y | RIC recipients had more advanced disease and poor KPS; low risk of relapse after either conditioning; lower GVHD with novel conditioning possibly related to use of ATG |
| Ballen56 | 1989-2002 | 289 | 47 (18-73) | Multiple, Bu/Cy (43%) | 21 | 56 | 35% siblings 50% for URD at 5 y | 33% siblings 27% for URD at 5 y | 37% siblings 30% for URD at 5 y | Relapse at 5 y, 32% in sibling and 23% in URD; performance status, peripheral blasts sibling donor status impacted OS; RIC was similar in outcomes, except early NRM |
| Alchalby71 | 1999-2009 | 162 | 56 (32-73) | Flu-Bu in 96% | 100 | 27 | 22% at 1 y | 46% at 5 y | 62% at 5 y | 82 patients reported previously54 ; age and HLA mismatch impacted NRM; 23% relapse at 3 y; clearance of mutated JAK2 at median of 96 days, and this reduced relapse risk |
| Bacigalupo63 | 1994-2007 | 46 | 51 (24-67) | Thiotepa-Cy + melphalan | 100 | 65 | 24% at 5 y | NR | 45% at 5 y | A risk score based on transfusion history, spleen > 22 cm and alternative donor use predicted lower OS; no benefit for splenectomy |
| Stewart76 | 1989-2005 | 51 | 49 (19-64) | Multiple, RIC in 47% | 47 | 65 | 41% at 2 y | 44% and 24% at 3 y for MAC and RIC | 44% and 31% at 3 y for MAC and RIC | |
| Robin92 | 1997-2008 | 147 | 53 (20-68) | Multiple | 69 | 61 | 39% at 4 y | 32% at 4 y | 39% at 4 y | 19% patients had LT; poor outcome with mismatched donor |
| Samuelson13 | 1999-2007 | 30 | 65 (60-78) | Multiple | 63 | 50 | 13% at day 100 | 40% at 3 y | 45% at 3 y | Studied outcomes in patients ≥ 60 y, 7 patients had preceding LT |
| Abelsson67 | 1982-2009 | 92 | 46 for MAC, 55 for RIC | Multiple | 56 | 40 | 32% for MAC and 24% for RIC at 2 y | NR | 49% for MAC and 59% for RIC at 5 y | Overall NRM similar, but 5-year OS superior for RIC younger than age 60 years; less advanced MF associated with better OS |
| Nivison-Smith69 | 1993-2005 | 57 | 47 (16-71) | Multiple | 26 | 68 | 25% at 1 y | 58% at 5 y | Poor outcome in patients > 50 y | |
| Ditschkowski70 | 1994-2010 | 76 | 50.5 (22-67) | Multiple | NR | 35 | 36% at 5 y | 50% at 5 y | 53% at 5 y | Significant high risk of relapse in patients without cGvHD; |
| DIPSS was predictive of survival | ||||||||||
| Scott57 | 1990-2009 | 170 | 51.5 (12-78) | Multiple | NR | 50 | 34% at 5 y | 57% at 5 y | 57% at 5 y | Post-HCT success was dependent on pre-HCT DIPSS scores |
MRD indicates matched related donor; PFS, progression-free survival; OS, overall survival; TBI, total body irradiation; Hb, hemoglobin; Bu, busulfan; Cy, cyclophosphamide; cGvHD, chronic GVHD; NR, not reported; DLI, donor lymphocyte infusion; AHCT, allogeneic HCT; EFS, event-free survival; KPS, Karnofsky performance status; and ATG, antithymocyte globulin.