Evidence map›Paper›PMID 29720227›Full record

ArticleJournal of hematology & oncology2018

First pediatric experience of SL-401, a CD123-targeted therapy, in patients with blastic plasmacytoid dendritic cell neoplasm: report of three cases.

Weili Sun, Huaying Liu, Young Kim, Nicole Karras, Anna Pawlowska, Debbie Toomey, Wade Kyono, Paul Gaynon, Joseph Rosenthal, Anthony Stein

Open access · goldAbstract readCase Reports
In one paragraph

Article in Journal of hematology & oncology, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 papers.

0numbers the graph read from it
0cells of the map it votes in
24citing papers in PubMed
6.2field-weighted citation impact, top 3% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

24 citing papers in PubMed, 49 citations in OpenAlex.

  1. Review
  2. BPDCN: state of the art.Hematology. American Society of Hematology. Education Program · 2024
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  17. Dendritic Cell Leukemia: a Review.Current oncology reports · 2020
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

10 authors at 4 institutions in 2 countries.

Weili SunDepartment of Pediatrics, City of Hope National Medical Center, 1500 E. Duarte Road, Duarte, CA, 91010, USA. weilisun@coh.org.
Huaying LiuDepartment of Pediatrics, City of Hope National Medical Center, 1500 E. Duarte Road, Duarte, CA, 91010, USA.
Young KimDepartment of Pathology, City of Hope National Medical Center, Duarte, CA, USA.
Nicole KarrasDepartment of Pediatrics, City of Hope National Medical Center, 1500 E. Duarte Road, Duarte, CA, 91010, USA.
Anna PawlowskaDepartment of Pediatrics, City of Hope National Medical Center, 1500 E. Duarte Road, Duarte, CA, 91010, USA.
Debbie ToomeyDepartment of Pediatrics, City of Hope National Medical Center, 1500 E. Duarte Road, Duarte, CA, 91010, USA.
Wade KyonoDepartment of Pediatrics, Kapiolani Medical Center for Women and Children, University of Hawaii, Honolulu, HI, USA.
Paul GaynonChildren's Center for Cancer and Blood Diseases, Children's Hospital Los Angeles, University of Southern California, Los Angeles, CA, USA.
Joseph RosenthalDepartment of Pediatrics, City of Hope National Medical Center, 1500 E. Duarte Road, Duarte, CA, 91010, USA.
Anthony SteinDepartment of Hematology and Hematopoietic Cell Transplantation, City of Hope National Medical Center, Duarte, CA, USA.
City Of Hope National Medical Center · USChildren's Center · USNanfang Hospital · CNUniversity of Hawaiʻi at Mānoa · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundBlastic plasmacytoid dendritic cell neoplasm (BPDCN) is a highly aggressive hematological malignancy with extremely poor outcome. The median overall survival for adult patients is 9-13 months. Pediatric patients are exceedingly rare with an unclear clinical course. Currently, no standardized therapy has been established, although an acute lymphoblastic leukemia type of treatment appears to be more effective in those patients who are able to tolerate aggressive chemotherapy. SL-401 is a targeted therapy directed to CD123, a protein ubiquitously expressed at high level on the surface of BPDCN blasts. In adult phase 2 trials, it has demonstrated efficacy with 90% overall response rate. No pediatric patients with BPDCN using SL-401 have been reported. CASE PRESENTATION: Here, we report the first pediatric experience of three children with BPDCN treated with SL-401 at our institution. All patients tolerated SL-401 without significant toxicities. One patient with multiply relapsed and refractory disease had no response. The other two cases had significant and rapid clinical improvement after the two courses of treatment. However, the response was transient, and growth of soft tissue mass was observed in-between cycles in both patients with large tumor burden.

conclusionsThis is the first report of SL-401 in pediatric patients with BPDCN. Sl-401 was well tolerated and can produce a promising response. Further testing this agent in children is warranted.

Indexed as

AdolescentChildDendritic CellsFemaleHematologic NeoplasmsHumansInterleukin-3 Receptor alpha SubunitRecombinant Fusion ProteinsInterleukin-3 Receptor alpha SubunitRecombinant Fusion ProteinstagraxofuspBPDCNPediatricSL-401Targeted therapy

Identifiers

PMID29720227
PMCPMC5932872
OpenAlexW2806114755

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.