Clinical trial · Interventional
Donor Stem Cell Transplant With or Without Chemotherapy in Treating Children With Primary Myelodysplastic Syndrome
Prospective Study of the Diagnosis and Treatment of Myelodysplastic Syndromes (MDS) in Childhood
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
RATIONALE: Giving chemotherapy before a donor stem cell transplant helps stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. It is not yet known whether donor stem cell transplant is more effective with or without chemotherapy in treating primary myelodysplastic syndrome. PURPOSE: This phase III trial is studying how well donor stem cell transplant given with chemotherapy works and compares it with donor stem cell transplant without chemotherapy in treating children with primary myelodysplastic syndrome.
Conditions
Conditions (2)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Leukemia | Leukemia | ONTOLOGY_EXACT | 0.90 |
| Myelodysplastic/Myeloproliferative Neoplasms | Myelodysplastic/Myeloproliferative Neoplasm | CURATED_BROADER | 0.80 |
Interventions
Interventions (6)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| allogeneic bone marrow transplantation | Procedure | — | UNRESOLVED |
| biopsy | Procedure | — | UNRESOLVED |
| cytarabine | Drug | Cytarabine | ALIAS |
| laboratory biomarker analysis | Other | — | UNRESOLVED |
| mercaptopurine | Drug | Mercaptopurine | ALIAS |
| peripheral blood stem cell transplantation | Procedure | — | UNRESOLVED |
Design
Arms and outcomes
Arms (0)
[]Primary outcomes (1)
- measure
- Patient numbers in the different FAB subtypes
Secondary outcomes (2)
- measure
- Survival
- measure
- Event-free survival
Eligibility
Eligibility (as posted)
- Sex
- All
- Maximum age
- 18 Years
Show eligibility criteria text
DISEASE CHARACTERISTICS: * Morphologically confirmed primary myelodysplastic syndromes (MDS) * Diagnosed between July 1, 1998 and June 30, 2002 * No prior aplastic anemia * No prior congenital bone marrow failure syndrome, such as: * Fanconi's anemia * Kostmann syndrome * Shwachman syndrome * Dyskeratosis congenital * Amegakaryocytic thrombocytopenia * Diamond-Blackfan anemia * No Down syndrome * None of the following cytogenetic or molecular abnormalities: * t(8;21)(q22;q22) * t(15;17)(q22;q12) * inv(16)(p13;q22) * No typical clinical and cytogenetic features of acute myeloid leukemia FAB M7 (i.e., acute megakaryocytic leukemia) with fewer than 30% blasts in bone marrow or peripheral blood PATIENT CHARACTERISTICS: Age * Under 19 Performance status * Not specified Life expectancy * Not specified Hematopoietic * See Disease Characteristics Hepatic * Not specified Renal * Not specified Other * No other concurrent illness that would preclude study PRIOR CONCURRENT THERAPY: Biologic therapy * Not specified Chemotherapy * No prior chemotherapy for MDS Endocrine therapy * Not specified Radiotherapy * No prior radiotherapy for MDS Surgery * Not specified
References
Publications (3)
- DERIVEDDrexler B, Schwarz-Furlan S, Baumann I, Rudelius M, Nollke P, Lebrecht D, Ramamoorthy S, Rotari N, Karow A, Hirabayashi S, Beier F, Behrens YL, Gohring G, Kalb R, Wlodarski MW, Strahm B, Erlacher M, Niemeyer CM, Yoshimi A. Long-term outcomes of patients with refractory cytopenia of childhood under observation only. Blood Adv. 2025 Aug 26;9(16):4279-4285. doi: 10.1182/bloodadvances.2025016136. PMID 40554414
- DERIVEDPastor VB, Sahoo SS, Boklan J, Schwabe GC, Saribeyoglu E, Strahm B, Lebrecht D, Voss M, Bryceson YT, Erlacher M, Ehninger G, Niewisch M, Schlegelberger B, Baumann I, Achermann JC, Shimamura A, Hochrein J, Tedgard U, Nilsson L, Hasle H, Boerries M, Busch H, Niemeyer CM, Wlodarski MW. Constitutional SAMD9L mutations cause familial myelodysplastic syndrome and transient monosomy 7. Haematologica. 2018 Mar;103(3):427-437. doi: 10.3324/haematol.2017.180778. Epub 2017 Dec 7. PMID 29217778
- DERIVEDGohring G, Michalova K, Beverloo HB, Betts D, Harbott J, Haas OA, Kerndrup G, Sainati L, Bergstraesser E, Hasle H, Stary J, Trebo M, van den Heuvel-Eibrink MM, Zecca M, van Wering ER, Fischer A, Noellke P, Strahm B, Locatelli F, Niemeyer CM, Schlegelberger B. Complex karyotype newly defined: the strongest prognostic factor in advanced childhood myelodysplastic syndrome. Blood. 2010 Nov 11;116(19):3766-9. doi: 10.1182/blood-2010-04-280313. Epub 2010 Aug 27. PMID 20802024