Evidence map›Paper›PMID 21478921›Full record

ReviewBone marrow transplantation2012

Treosulfan-based conditioning before hematopoietic SCT: more than a BU look-alike.

I Danylesko, A Shimoni, A Nagler

2 registry-linked trialsAbstract readReview
PubMed Publisher
In one paragraph

Review in Bone marrow transplantation, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 33 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
33citing papers in PubMed, 1 pooled it
–field-weighted citation impact
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.

NCT03538899 phase1 / phase2recruitingnot on this mapstarted 2018, after this paper: background citation

Phase I/II Safety and Efficacy Study of Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency (ART-SCID) in Newly Diagnosed Patients Using Self-Inactivating Lentiviral Vector (AProArt) to Transduce Autologous CD34 Hematopoietic Cells

TypeinterventionalSponsorUniversity of California, San FranciscoRan2018 to 2038Enrolled24ConditionsSevere Combined ImmunodeficiencyArmsAProArt-CD34, CliniMACS® CD34 Reagent System cell sorter device, Busulfan
NCT07232953 phase2active not recruitingnot on this mapstarted 2022, after this paper: background citation

Prospective Phase II Study on Safety and Efficacy of Fludarabine Plus Treosulfan (14g) (FT14) Conditioning Regimen for Allogeneic Stem Cell Transplantation (Allo-SCT) in Acute Myeloid Leukemia (AML) Patients (≥40 <65years) (FT14-Trial)

TypeinterventionalSponsorAzienda Socio Sanitaria Territoriale degli Spedali Civili di BresciaRan2022 to 2027Enrolled82ConditionsAcute Myeloid Leukaemia (AML), Hematopoietic Stem Cell Transplant (HSCT)ArmsFludarabine + Treosulfan
3 · Its place in the literature

Who cites it

33 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Trial
  3. Hematopoietic Cell Transplantation for Paroxysmal Nocturnal Hemoglobinuria in the Age of Eculizumab.Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2019
    Trial
  4. Trial
  5. Trial
  6. Review
  7. Article
  8. Article
  9. Article
  10. Article
  11. Article
  12. Article
  13. Article
  14. Review
  15. Review
  16. Article
  17. Article
  18. Observational
  19. Article
  20. Treosulfan-based conditioning for inborn errors of immunity.Therapeutic advances in hematology · 2021
    Review
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

3 authors.

I DanyleskoHematology and Bone Marrow Transplantation Division, Department of Hematology and Bone Marrow Transplantation, Laboratory of Molecular Immunobiology, Chaim Sheba Medical Center, Tel Hashomer, Israel.
A Shimoni
A Nagler

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Allogeneic hematopoietic SCT is a curative treatment for a variety of hematological malignancies and genetic diseases. There is a continuous search for novel conditioning regimens that will reduce SCT-related toxicity while retaining maximal antimalignancy effect. Treosulfan (L-threitol-1,4-bis-methanesulfonate; dihydroxybusulfan) was initially used in the treatment of certain solid tumors. Preclinical studies showed that it has a myeloablative effect on committed and non-committed stem cells. It has potent immunosuppressive characteristics, more prominent than its related chemotherapy agent BU, which makes it an attractive candidate for the use in conditioning regimens before allo-SCT. It is also associated with a favorable toxicity profile with little extramedullary toxicity. The combination of fludarabine and treosulfan was explored in several studies in patients not eligible for standard myeloablative conditioning, and data are rapidly emerging. This regimen is associated with consistent engraftment. A limited non-relapse mortality (NRM) rate in the range of 9-28% was observed. This rate is promising considering the patients selected and results from low rates of organ toxicity as well as acute and chronic GVHD. The regimen was also associated with low relapse rates of 5-30% depending on disease status at SCT. Together with low NRM rate, this resulted in favorable survival in the range of 40-80%. Promising results were seen in myelodysplastic syndrome (survival 36-70%) and leukemia in remission (60-70%). Randomized prospective studies will be needed to better define the role of treosulfan-based regimens in SCT.

Indexed as

Hematopoietic Stem Cell TransplantationAntineoplastic Agents, AlkylatingBusulfanDisease-Free SurvivalGraft vs Host DiseaseHumansLeukemiaMyelodysplastic SyndromesSurvival RateTransplantation ConditioningTransplantation, HomologousAntineoplastic Agents, AlkylatingBusulfantreosulfan

Identifiers

PMID21478921

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

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.