Case reports and current status of all patients
| Patient No. . | Reason for Transplant . | Stem Cell Source . | Donor . | Conditioning . | Comments . |
|---|---|---|---|---|---|
| 1 | Neutropenia | Bone marrow | HLA-identical sibling | CY | Graft rejected; autologous engraftment with recurring neutropenia; subsequent availability of r-HuG-CSF has allowed complete response. |
| 2 | Nonresponder | Bone marrow | HLA-identical sibling | BU/CY | GVHD grade I; no problems at time of report. |
| 3 | Nonresponder | Bone marrow | HLA-identical sibling | BU/CY + ATG | Fungal infection pre-BMT; treated successfully. |
| 4 | Nonresponder | PBPCs | HLA-identical sibling | BU/CY | Alive and well. |
| 5 | Nonresponder | Bone marrow | Single-antigen-mismatched unrelated | BU/CY | Acute GVHD grade IV of intestines, resolved; chronic GVHD skin; failure to thrive syndrome. |
| 6 | Pancytopenia | Bone marrow | Single-antigen-mismatched father | BU/CY | Died on day +111 of grade IV GVHD and septicemia. |
| 7 | Nonresponder | PBPCs | Haploidentical father | BU/CY + ATG + Thiotepa | Died on day +70 of multiorgan failure. |
| 8 | Nonresponder | Bone marrow, cord blood | HLA-identical sibling | BU/CY + ATG | Cystectomy due to hemorrhagic cystitis; presently alive and well. |
| 9 | Partial response; recurrent infections | Bone marrow | HLA-identical sibling | BU/CY + ATG | Persistent quadriparesis; neurological symptoms were present before BMT. |
| 10 | G-CSF receptor mutation | Bone marrow | HLA-identical sibling | BU/CY + melphalan | Acute GVHD grade III of skin and intestines; chronic GVHD of skin; fungal pneumonia developed after treatment with steroids; stable hematopoiesis. |
| 11 | Partial response; thoracic granulomatous infection | Cord blood | HLA-identical sibling | BU/CY + Thiotepa | Complete engraftment; no problems reported. |
| Patient No. . | Reason for Transplant . | Stem Cell Source . | Donor . | Conditioning . | Comments . |
|---|---|---|---|---|---|
| 1 | Neutropenia | Bone marrow | HLA-identical sibling | CY | Graft rejected; autologous engraftment with recurring neutropenia; subsequent availability of r-HuG-CSF has allowed complete response. |
| 2 | Nonresponder | Bone marrow | HLA-identical sibling | BU/CY | GVHD grade I; no problems at time of report. |
| 3 | Nonresponder | Bone marrow | HLA-identical sibling | BU/CY + ATG | Fungal infection pre-BMT; treated successfully. |
| 4 | Nonresponder | PBPCs | HLA-identical sibling | BU/CY | Alive and well. |
| 5 | Nonresponder | Bone marrow | Single-antigen-mismatched unrelated | BU/CY | Acute GVHD grade IV of intestines, resolved; chronic GVHD skin; failure to thrive syndrome. |
| 6 | Pancytopenia | Bone marrow | Single-antigen-mismatched father | BU/CY | Died on day +111 of grade IV GVHD and septicemia. |
| 7 | Nonresponder | PBPCs | Haploidentical father | BU/CY + ATG + Thiotepa | Died on day +70 of multiorgan failure. |
| 8 | Nonresponder | Bone marrow, cord blood | HLA-identical sibling | BU/CY + ATG | Cystectomy due to hemorrhagic cystitis; presently alive and well. |
| 9 | Partial response; recurrent infections | Bone marrow | HLA-identical sibling | BU/CY + ATG | Persistent quadriparesis; neurological symptoms were present before BMT. |
| 10 | G-CSF receptor mutation | Bone marrow | HLA-identical sibling | BU/CY + melphalan | Acute GVHD grade III of skin and intestines; chronic GVHD of skin; fungal pneumonia developed after treatment with steroids; stable hematopoiesis. |
| 11 | Partial response; thoracic granulomatous infection | Cord blood | HLA-identical sibling | BU/CY + Thiotepa | Complete engraftment; no problems reported. |
CY indicates cyclophosphamide; BU, busulfan; ATG, antithymocyte globulin; r-HuG-CSF, recombinant human granulocyte colony-stimulating factor; GVHD graft-versus-host disease; BMT, bone marrow transplantation; PBPCs, peripheral blood progenitor cells.