Disease · MONDO_0000159

bone marrow failure syndrome

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. bone marrow failure syndrome. Diseases, record MONDO_0000159. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0000159

On this page

Disease

Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.

Name: bone marrow failure syndrome; Open Targets
Open Targets ID: MONDO_0000159; Open Targets
MeSH: C536572; MeSH
NCI Thesaurus: C165614; NCI Thesaurus
Highest stage of its drugs: Phase 3, for 3 of its 12 drugs, from ClinicalTrials.gov; Open Targets

Drugs12

The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.

Drug	ChEMBL ID	Type	Open Targets' stage	Stage reported by	Reports	Of them trials	Trials shown here	Source
ANTILYMPHOCYTE IMMUNOGLOBULIN (HORSE)	CHEMBL2108981		Phase 3	ClinicalTrials.gov	4	4	0	Drugs and targets
CYCLOPHOSPHAMIDE	CHEMBL1200796	Small molecule	Phase 3	ClinicalTrials.gov	3	3	3	Drugs and targets
FLUDARABINE	CHEMBL1568	Small molecule	Phase 3	ClinicalTrials.gov	3	3	3	Drugs and targets
ALEMTUZUMAB	CHEMBL1201587	Antibody	Phase 2/3	ClinicalTrials.gov	2	2	2	Drugs and targets
BUSULFAN	CHEMBL820	Small molecule	Phase 2/3	ClinicalTrials.gov	1	1	1	Drugs and targets
FLUDARABINE PHOSPHATE	CHEMBL1096882	Small molecule	Phase 2	ClinicalTrials.gov	3	3	3	Drugs and targets
THIOTEPA	CHEMBL671	Small molecule	Phase 2	ClinicalTrials.gov	2	2	2	Drugs and targets
TREOSULFAN	CHEMBL455186	Small molecule	Phase 2	ClinicalTrials.gov	2	2	2	Drugs and targets
FILGRASTIM	CHEMBL1201567	Protein	Phase 1	ClinicalTrials.gov	2	2	1	Drugs and targets
PLERIXAFOR	CHEMBL18442	Small molecule	Phase 1	ClinicalTrials.gov	1	1	1	Drugs and targets

1 to 10 of 12

See all 12 drugs

Targets17

The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.

Target	Protein	Its drugs that act on it	Mechanisms	Highest stage of those drugs	Gene in Genes	Source
CSF3R	Granulocyte colony-stimulating factor receptor	3	Granulocyte colony stimulating factor receptor agonist	Phase 1	https://conductscience.org/library/genes/1441	Drugs and targets
CD52	CAMPATH-1 antigen	1	CAMPATH-1 antigen inhibitor	Phase 2/3	https://conductscience.org/library/genes/1043	Drugs and targets
CXCR4	C-X-C chemokine receptor type 4	1	C-X-C chemokine receptor type 4 partial agonist	Phase 1	https://conductscience.org/library/genes/7852	Drugs and targets
POLA1	DNA polymerase (alpha/delta/epsilon)	1	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/5422	Drugs and targets
POLA2	DNA polymerase (alpha/delta/epsilon)	1	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/23649	Drugs and targets
POLD1	DNA polymerase (alpha/delta/epsilon)	1	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/5424	Drugs and targets
POLD2	DNA polymerase (alpha/delta/epsilon)	1	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/5425	Drugs and targets
POLD3	DNA polymerase (alpha/delta/epsilon)	1	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/10714	Drugs and targets
POLD4	DNA polymerase (alpha/delta/epsilon)	1	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/57804	Drugs and targets
POLE	DNA polymerase (alpha/delta/epsilon)	1	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/5426	Drugs and targets

1 to 10 of 17

See all 17 targets

Trials7

Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs. 1 more is not shown, since the checks do not confirm it.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT00919503	Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders	Phase 2	98	2021-08-13	Non-Neoplastic Hematologic and Lymphocytic Disorder	FLUDARABINE PHOSPHATE, TREOSULFAN	Open Targets' reading of its record	ClinicalTrials.gov
NCT00004143	Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes	Phase 2	2	2013-11-11	Sickle Cell Anemia, Severe Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria (PNH), Pure Red Cell Aplasia	ALEMTUZUMAB, CYCLOPHOSPHAMIDE, FLUDARABINE	Its record names the disease	ClinicalTrials.gov
NCT00774527	Comparison of Cy-Atg Vs Cy-Flu-Atg for the Conditioning Therapy in Allo-HCT	Phase 3	82		Bone Marrow Failure Syndromes	CYCLOPHOSPHAMIDE, FLUDARABINE	Its record names the disease	ClinicalTrials.gov
NCT03980769	Donor Stem Cell Transplant With Treosulfan, Fludarabine, and Thiotepa in Treating Patients With Non-malignant Disorders	Phase 2	40		Non-Neoplastic Hematopoietic and Lymphoid Cell Disorder	FLUDARABINE PHOSPHATE, THIOTEPA, TREOSULFAN	Open Targets' reading of its record	ClinicalTrials.gov
NCT01019876	Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases	Phase 2/3	38		Bone Marrow Failure, Osteopetrosis, Fanconi Anemia, Severe Combined Immunodeficiency	ALEMTUZUMAB, BUSULFAN, CYCLOPHOSPHAMIDE, FLUDARABINE	Open Targets' reading of its record	ClinicalTrials.gov
NCT00295971	Donor Stem Cell Transplant in Treating Young Patients With Myelodysplastic Syndrome, Leukemia, Bone Marrow Failure Syndrome, or Severe Immunodeficiency Disease	Phase 1	21		Congenital Amegakaryocytic Thrombocytopenia, Leukemia, Myelodysplastic Syndromes, Severe Congenital Neutropenia	FLUDARABINE PHOSPHATE, THIOTEPA	Its record names the disease	ClinicalTrials.gov
NCT07585136	Stem Cell Mobilization and Apheresis for Life-threatening Blood Disorders	Phase 1	12		Bone Marrow Failure Syndrome	FILGRASTIM, PLERIXAFOR	Its record names the disease	ClinicalTrials.gov

Comparisons in its trials10

Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.

Comparison	Group 1, with the outcome	Group 2, with the outcome	Participants	Trial	Source
Regimen A (PBSCT and BMT) vs Regimen B (UBCT): Number of Participants With Infections	57 of 84	9 of 14	98	NCT00919503	Trial comparisons
Regimen A (PBSCT and BMT) vs Regimen B (UBCT): deaths from any cause	7 of 84	6 of 14	98	NCT00919503	Trial comparisons
Regimen A (PBSCT and BMT) vs Regimen B (UBCT): Overall Survival	80 of 84	9 of 14	98	NCT00919503	Trial comparisons
Regimen A (PBSCT and BMT) vs Regimen B (UBCT): Non-relapse Mortality	2 of 84	5 of 14	98	NCT00919503	Trial comparisons
Regimen A (PBSCT and BMT) vs Regimen B (UBCT): serious adverse events	17 of 84	5 of 14	98	NCT00919503	Trial comparisons
Regimen A (PBSCT and BMT) vs Regimen B (UBCT): Number of Patients With Grade II-IV Acute Graft-versus-host Disease	44 of 84	8 of 12	96	NCT00919503	Trial comparisons
Regimen A (PBSCT and BMT) vs Regimen B (UBCT): Disease Response at One Year Following Hematopoietic Cell Transplantation	78 of 84	8 of 11	95	NCT00919503	Trial comparisons
Regimen A (PBSCT and BMT) vs Regimen B (UBCT): Preliminary Efficacy	83 of 84	10 of 11	95	NCT00919503	Trial comparisons
Regimen A (PBSCT and BMT) vs Regimen B (UBCT): Number of Patients With of Chronic Graft-versus-host Disease	29 of 84	5 of 11	95	NCT00919503	Trial comparisons
Regimen A (PBSCT and BMT) vs Regimen B (UBCT): Immune Reconstitution Following Hematopoietic Cell Transplantation	39 of 73	4 of 9	82	NCT00919503	Trial comparisons

Papers from its trials0

The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.

None in this release.

Data dates

The date of each source this release read.

Open Targets: 2026-09-18; Open Targets
Drug links and their checks, from Drugs and targets: 2026-09-18; Drugs and targets
ClinicalTrials.gov, from Trials: 2026-11-12; Trials
Comparisons, from Trial comparisons: 2026-03-23; Trial comparisons
PubMed, for the checks: 2026-11-06; PubMed
MeSH, for the checks: 2026-11-10; MeSH

Sources 6

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ChEMBL, through Open TargetsCC BY-SA 3.0
ClinicalTrials.govNo license stated
NLM's MeSHNLM's terms
Open TargetsCC0 1.0
PubMedNLM's terms

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
ClinicalTrials.govnone stated
NLM's MeSHNLM's
Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
PubMedNLM's terms

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this disease from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/diseases/v1/MONDO_0000159

GET https://conductscience.org/api/diseases/v1/MONDO_0000159/drugs

API reference For AI assistants