Characteristics of early stem cell transplantation (SCT) patients
| Age, y/sex . | MPN phase . | Blast phase . | Induction . | Response . | SCT . | Outcome . |
|---|---|---|---|---|---|---|
| 56/F | Primary MF for 54 mo; 3 prior therapies; diploid karyotype; JAK2 not known | 18/32% PB/BM blasts; myeloid phenotype; Der(6)t(1;6)(q23;p25) JAK-2− | Idarubicin + HDAC, then SDAC consolidation × 1 | CR | HLA-identical sibling, fludarabine, melphalan, and gemtuzumab ozogamicin | Alive in ongoing CR at 34 mo |
| 52/M | Primary MF for 37 mo; 1 prior therapy; diploid karyotype; JAK2 not known | 48/22% PB/BM blasts; myeloid phenotype; diploid karyotype; JAK-2+ | Idarubicin + HDAC | CRi | HLA-identical sibling, fludarabine and busulfan | Relapsed 5 mo after SCT, alive in second CRi 19 mo after re-induction chemotherapy and withdrawal of immunosuppression |
| 63/M | Primary MF for 34 mo; 3 prior therapies; diploid karyotype; JAK2+ | 11/20% PB/BM blasts; myeloid phenotype; complex karyotype; JAK-2+ | Idarubicin + HDAC | CRi | HLA-identical sibling, fludarabine, melphalan, and gemtuzumab ozogamicin | Alive in ongoing CRi at 14 mo; JAK-2 negative |
| 60/M | Primary MF for 30 mo; MUD SCT 11 mo earlier; diploid karyotype; JAK-2− | 38/35% PB/BM blasts; myeloid phenotype; T(3;8)(q27;p23) JAK-2− | Second SCT with same donor | CRi | Matched unrelated donor, fludarabine, melphalan, gemtuzumab ozogamicin, and ATG | Alive 4 mo after second SCT in ongoing CRi, with azacytidine maintenance; |
| 49/M | Primary MF for 39 mo; 1 prior therapy; diploid karyotype; JAK-2 not known | 70% BM blasts; undifferentiated phenotype; cytogenetics failed; JAK-2 not known | Vincristine + prednisone | PR (10% BM blasts) | HLA-identical sibling, busulphan and thiotepa | Alive in ongoing CR at 131 mo |
| 58/M | Post-ET MF for 31 mo; 2 prior therapies; karyotype not known; JAK-2 not known | 31/34% PB/BM blasts; myeloid phenotype; diploid karyotype; JAK-2− | Idarubicin + HDAC | CR* | HLA-identical sibling, fludarabine and bulsulphan | Died in remission 6 mo after SCT, from liver GVHD |
| 60/F | Post-ET MF for 19 mo; MUD SCT 5 months earlier; complex karyotype; JAK-2+ | 0/38% PB/BM blasts; myeloid phenotype; complex karyotype; JAK-2+ | Idarubicin + HDAC | CRi | Second SCT with same MUD donor, fludarabine, melphalan, and gemtuzumab ozogamicin | Died in remission 3 mo after second SCT, from multiple transplant-related complications including GVHD, microangiopathy, and sepsis |
| 53/M | Primary MF for 25 mo; 2 prior therapies; diploid karyotype; JAK-2 not known | 21/13% PB/BM blasts; myeloid phenotype; diploid karyotype; JAK-2 not known | Stem cell transplantation | CR | MUD, fludarabine, busulphan, and ATG | Alive in ongoing CR at 77 mo |
| Age, y/sex . | MPN phase . | Blast phase . | Induction . | Response . | SCT . | Outcome . |
|---|---|---|---|---|---|---|
| 56/F | Primary MF for 54 mo; 3 prior therapies; diploid karyotype; JAK2 not known | 18/32% PB/BM blasts; myeloid phenotype; Der(6)t(1;6)(q23;p25) JAK-2− | Idarubicin + HDAC, then SDAC consolidation × 1 | CR | HLA-identical sibling, fludarabine, melphalan, and gemtuzumab ozogamicin | Alive in ongoing CR at 34 mo |
| 52/M | Primary MF for 37 mo; 1 prior therapy; diploid karyotype; JAK2 not known | 48/22% PB/BM blasts; myeloid phenotype; diploid karyotype; JAK-2+ | Idarubicin + HDAC | CRi | HLA-identical sibling, fludarabine and busulfan | Relapsed 5 mo after SCT, alive in second CRi 19 mo after re-induction chemotherapy and withdrawal of immunosuppression |
| 63/M | Primary MF for 34 mo; 3 prior therapies; diploid karyotype; JAK2+ | 11/20% PB/BM blasts; myeloid phenotype; complex karyotype; JAK-2+ | Idarubicin + HDAC | CRi | HLA-identical sibling, fludarabine, melphalan, and gemtuzumab ozogamicin | Alive in ongoing CRi at 14 mo; JAK-2 negative |
| 60/M | Primary MF for 30 mo; MUD SCT 11 mo earlier; diploid karyotype; JAK-2− | 38/35% PB/BM blasts; myeloid phenotype; T(3;8)(q27;p23) JAK-2− | Second SCT with same donor | CRi | Matched unrelated donor, fludarabine, melphalan, gemtuzumab ozogamicin, and ATG | Alive 4 mo after second SCT in ongoing CRi, with azacytidine maintenance; |
| 49/M | Primary MF for 39 mo; 1 prior therapy; diploid karyotype; JAK-2 not known | 70% BM blasts; undifferentiated phenotype; cytogenetics failed; JAK-2 not known | Vincristine + prednisone | PR (10% BM blasts) | HLA-identical sibling, busulphan and thiotepa | Alive in ongoing CR at 131 mo |
| 58/M | Post-ET MF for 31 mo; 2 prior therapies; karyotype not known; JAK-2 not known | 31/34% PB/BM blasts; myeloid phenotype; diploid karyotype; JAK-2− | Idarubicin + HDAC | CR* | HLA-identical sibling, fludarabine and bulsulphan | Died in remission 6 mo after SCT, from liver GVHD |
| 60/F | Post-ET MF for 19 mo; MUD SCT 5 months earlier; complex karyotype; JAK-2+ | 0/38% PB/BM blasts; myeloid phenotype; complex karyotype; JAK-2+ | Idarubicin + HDAC | CRi | Second SCT with same MUD donor, fludarabine, melphalan, and gemtuzumab ozogamicin | Died in remission 3 mo after second SCT, from multiple transplant-related complications including GVHD, microangiopathy, and sepsis |
| 53/M | Primary MF for 25 mo; 2 prior therapies; diploid karyotype; JAK-2 not known | 21/13% PB/BM blasts; myeloid phenotype; diploid karyotype; JAK-2 not known | Stem cell transplantation | CR | MUD, fludarabine, busulphan, and ATG | Alive in ongoing CR at 77 mo |
Three patients developed acute leukemia after SCT: 2 developed blast phase after receiving SCT in chronic phase, and 1 relapsed after receiving SCT as consolidation therapy for blast phase. All 3 had best T-cell chimerism of less than 65% from their initial SCT.
MF indicates myelofibrosis; PB, peripheral blood; BM, bone marrow; HDAC, high-dose Ara-C; SDAC, standard-dose Ara-C; CR, complete remission; CRi, CR with incomplete blood recovery; GVHD, graft-versus-host disease; MUD, matched unrelated donor; and ATG, antithymocyte globulin.
Early evidence of relapse (7% marrow blasts) 2 weeks before conditioning.