Overview of outcomes of patients with BDPCN from different therapy approaches with a focus on post-HSCT outcomes
| Author and study period . | Type of study . | Therapy details∗ . | Response rates [(N)/%]† . | OS dynamics . | SCT-specific outcomes . |
|---|---|---|---|---|---|
| Roos-Weil et al33 (EBMT analysis; 2000-2009) | Retrospective (only patients receiving transplant included) | Nontargeted AML/ALL-type = 27 NHL-type = 7 | N/A | Allo-SCT in CR1 = 19 Allo-SCT ≥ CR1 = 15 3 y OS = 43% | |
| Laribi et al121 2001-2017 | Retrospective | Nontargeted AML-type = 53 ALL-type = 96 NHL-type = 150 | AML-type Rx f/b SCT = 14/16 (88%) ALL-type Rx f/b SCT = 31/33 (94%) NHL-type Rx f/b SCT = 12/12 (100%) | Median OS = 18 mo | 5 years OS AML-type therapy f/b allo-SCT = 59.5% ALL-type therapy f/b allo-SCT = 47.3% NHL-type therapy f/b allo-SCT = 71.1% No allo-SCT = 9.6% for leukemia-type therapy, 8.3% for lymphoma-type therapy |
| Yun et al72 2001-2019 | Retrospective | Mixed AML-like = 1 ALL-type = 11 NHL-like = 10 SL-401 = 12 | N/A 11 (100) 9 (90) 9 (75) | mOS with SCT = NR mOS without SCT ≈ 3 y | |
| Pagano et al10 2005-2011 | Retrospective | Nontargeted AML-type = 26 ALL-type = 15 | 7/16 (44) 10/15 (67) | Median OS = 8.7 mo After AML-type Rx = 7.1 mo After ALL-type Rx = 12.3 mo | SCT = 6 mOS with SCT = 22.7 mo mOS without SCT= 7.1 mo |
| Aoki et al31 (JSHCT analysis; 2002-2015) | Retrospective (only patients receiving transplant included) | Nontargeted AML-type =4 ALL-type = 10 NHL-type = 11 | N/A | 4 y OS = 65% After AML-type Rx = 67% After ALL-type Rx = 70% After NHL-type Rx = 62% | 4 y OS After auto-SCT = 82% After allo-SCT = 69% |
| Taylor et al24 2000-2017 | Retrospective | Nontargeted AML-type = 9 ALL-type = 35 Others = 10 | N/A | 2 y OS = 49% | SCT = 25; (allo = 20, auto = 5) 2 y OS after SCT = 60% |
| Pemmaraju et al30 1999-2020 | Retrospective | Mixed ALL-type = 35 SL-401 = 37 Others = 28 | 80% 59% 43% | Median OS After ALL-type Rx= 28.3 mo After SL-401 = 13.7 mo After other Rx = 22.8 mo | % responders proceeding to allo-SCT in CR1 15/28 (54%) 13/22 (59%) 4/12 (33%) SCT specific survival data N/A |
| Pemmaraju et al71 2014-2019 | Prospective clinical trial (long-term follow-up, 34 mo) | Targeted SL-401 (F/L) = 65§ | 75% | 2 y OS = 40% | SCT = 19‡ 2 y OS after SCT = 66% 2 y OS without SCT ≈ 30% |
| Author and study period . | Type of study . | Therapy details∗ . | Response rates [(N)/%]† . | OS dynamics . | SCT-specific outcomes . |
|---|---|---|---|---|---|
| Roos-Weil et al33 (EBMT analysis; 2000-2009) | Retrospective (only patients receiving transplant included) | Nontargeted AML/ALL-type = 27 NHL-type = 7 | N/A | Allo-SCT in CR1 = 19 Allo-SCT ≥ CR1 = 15 3 y OS = 43% | |
| Laribi et al121 2001-2017 | Retrospective | Nontargeted AML-type = 53 ALL-type = 96 NHL-type = 150 | AML-type Rx f/b SCT = 14/16 (88%) ALL-type Rx f/b SCT = 31/33 (94%) NHL-type Rx f/b SCT = 12/12 (100%) | Median OS = 18 mo | 5 years OS AML-type therapy f/b allo-SCT = 59.5% ALL-type therapy f/b allo-SCT = 47.3% NHL-type therapy f/b allo-SCT = 71.1% No allo-SCT = 9.6% for leukemia-type therapy, 8.3% for lymphoma-type therapy |
| Yun et al72 2001-2019 | Retrospective | Mixed AML-like = 1 ALL-type = 11 NHL-like = 10 SL-401 = 12 | N/A 11 (100) 9 (90) 9 (75) | mOS with SCT = NR mOS without SCT ≈ 3 y | |
| Pagano et al10 2005-2011 | Retrospective | Nontargeted AML-type = 26 ALL-type = 15 | 7/16 (44) 10/15 (67) | Median OS = 8.7 mo After AML-type Rx = 7.1 mo After ALL-type Rx = 12.3 mo | SCT = 6 mOS with SCT = 22.7 mo mOS without SCT= 7.1 mo |
| Aoki et al31 (JSHCT analysis; 2002-2015) | Retrospective (only patients receiving transplant included) | Nontargeted AML-type =4 ALL-type = 10 NHL-type = 11 | N/A | 4 y OS = 65% After AML-type Rx = 67% After ALL-type Rx = 70% After NHL-type Rx = 62% | 4 y OS After auto-SCT = 82% After allo-SCT = 69% |
| Taylor et al24 2000-2017 | Retrospective | Nontargeted AML-type = 9 ALL-type = 35 Others = 10 | N/A | 2 y OS = 49% | SCT = 25; (allo = 20, auto = 5) 2 y OS after SCT = 60% |
| Pemmaraju et al30 1999-2020 | Retrospective | Mixed ALL-type = 35 SL-401 = 37 Others = 28 | 80% 59% 43% | Median OS After ALL-type Rx= 28.3 mo After SL-401 = 13.7 mo After other Rx = 22.8 mo | % responders proceeding to allo-SCT in CR1 15/28 (54%) 13/22 (59%) 4/12 (33%) SCT specific survival data N/A |
| Pemmaraju et al71 2014-2019 | Prospective clinical trial (long-term follow-up, 34 mo) | Targeted SL-401 (F/L) = 65§ | 75% | 2 y OS = 40% | SCT = 19‡ 2 y OS after SCT = 66% 2 y OS without SCT ≈ 30% |
The boldface text denotes the type of regimen used in the study mentioned in each particular row.
auto-SCT, autologous stem cell transplantation; EBMT, European society for blood and marrow transplantation; f/b, followed by; mOS, median overall survival; N/A, not available; NHL, non-Hodgkin lymphoma; NR, not reached; Rx, therapy; SL-401, tagraxofusp.
Therapy details have been broadly divided as targeted or nontargeted based on treatment with SL-401, which is targeted against CD 123.
Response rates include complete and partial remissions and annotated the best possible extent from available study data.
Includes the 19 patients who underwent SCT after CR + CRc; overall 21 patients underwent SCT.
Patients who received the 12 μg/kg dose of SL-401.