Characteristics of the study cohort
| Characteristic . | Values . |
|---|---|
| Patients, no. | 53 |
| Median patient age, y (range) | 8.3 (1.4-16.9) |
| 1-5 y, no. (%) | 18 (34) |
| 6-16 y, no. (%) | 35 (66) |
| Male, no. (%) | 29 (55) |
| Diagnosis, no. (%) | |
| ALL | 20 (38) |
| AML | 8 (15) |
| MDS/JMML | 9 (17) |
| CML | 4 (8) |
| SAA | 4 (8) |
| Other* | 8 (15) |
| Conditioning regimen, no. (%) | |
| TBI-based | 24 (45) |
| Busulfan-based | 25 (47) |
| Cyclophosphamide only† | 4 (8) |
| Donor type, no. (%) | |
| Related | 24 (45) |
| Unrelated | 29 (55) |
| Stem cell source, no. (%) | |
| BM | 36 (68) |
| PBSC | 16 (30) |
| BM and PBSC | 1 (2) |
| T-cell depletion, no. (%) | |
| No | 17 (32) |
| In vivo (ATG) | 34 (64) |
| In vitro (CD34+ selection) | 2 (4) |
| GvHD prophylaxis, no. (%) | |
| CSA | 9 (17) |
| CSA + MTX | 42 (79) |
| CSA + MTX + MMF | 2 (4) |
| IVIG after transplantation, no. (%) | 46 (87) |
| Ongoing IST at vaccination‡, no. (%) | 10 (19) |
| Evaluable,§ no. (%) | |
| Efficacy | 53 (100) |
| Safety | 43 (81) |
| Characteristic . | Values . |
|---|---|
| Patients, no. | 53 |
| Median patient age, y (range) | 8.3 (1.4-16.9) |
| 1-5 y, no. (%) | 18 (34) |
| 6-16 y, no. (%) | 35 (66) |
| Male, no. (%) | 29 (55) |
| Diagnosis, no. (%) | |
| ALL | 20 (38) |
| AML | 8 (15) |
| MDS/JMML | 9 (17) |
| CML | 4 (8) |
| SAA | 4 (8) |
| Other* | 8 (15) |
| Conditioning regimen, no. (%) | |
| TBI-based | 24 (45) |
| Busulfan-based | 25 (47) |
| Cyclophosphamide only† | 4 (8) |
| Donor type, no. (%) | |
| Related | 24 (45) |
| Unrelated | 29 (55) |
| Stem cell source, no. (%) | |
| BM | 36 (68) |
| PBSC | 16 (30) |
| BM and PBSC | 1 (2) |
| T-cell depletion, no. (%) | |
| No | 17 (32) |
| In vivo (ATG) | 34 (64) |
| In vitro (CD34+ selection) | 2 (4) |
| GvHD prophylaxis, no. (%) | |
| CSA | 9 (17) |
| CSA + MTX | 42 (79) |
| CSA + MTX + MMF | 2 (4) |
| IVIG after transplantation, no. (%) | 46 (87) |
| Ongoing IST at vaccination‡, no. (%) | 10 (19) |
| Evaluable,§ no. (%) | |
| Efficacy | 53 (100) |
| Safety | 43 (81) |
ALL indicates acute lymphoblastic leukemia; AML, acute myeloblastic leukemia; MDS, myelodysplastic syndrome; JMML, juvenile myelomonocytic leukemia; CML, chronic myelogenous leukemia; SAA, severe aplastic anemia; TBI, total body irradiation; BM, bone marrow; PBSC, peripheral blood stem cells; ATG, anti–thymocyte globulin; GvHD, graft-versus-host disease; CSA, cyclosporin A; MTX, methotrexate; MMF, mycophenolate mofetil; IVIG, intravenous immunoglobulin; IST, immunosuppressive therapy.
Other diagnoses are beta-thalassemia (n = 2), Fanconi anemia (n = 1), unspecified myeloproliferative disease (n = 1), congenital amegakaryocytic thrombocytopenia (n = 1), familial hemophagocytic lymphohistiocytosis (n = 1), mucopolysaccharidosis type I (n = 1), and Diamond-Blackfan anemia (n = 1).
Patients with SAA undergoing BM transplantation from identical sibling donors received cyclophosphamide + ATG for conditioning.
Indications for IST at vaccination were chronic GvHD (n = 9) and mixed hematopoietic chimerism (n = 1). Patients received IST at all 3 vaccinations (n = 8) or at first and second vaccination only (n = 2).
All patients receiving at least one dose of study vaccine were evaluated for toxicity. For analysis of efficacy, patients with complete data on pneumococcal serotype-specific antibody concentrations were included. Reasons for incomplete serologic data were (1) dropping out during the primary vaccination series because of relapse (n = 2) or persistent thrombocytopenia (n = 1), (2) insufficient or delayed serum sampling (n = 6), and (3) administration of intravenous immunoglobulin for varicella zoster virus contact during primary vaccination series (n = 1).