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Disease · HP_0005528

Bone marrow hypocellularity

6 sourcesdiseases-2026-09-18-0.1.0

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ConductScience Library. Bone marrow hypocellularity. Diseases, record HP_0005528. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/HP_0005528

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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 hypocellularity
Open Targets ID
HP_0005528
Other names
Bone marrow failure, Bone marrow hypoplasia, Hypoplastic bone marrow, Myeloid hypoplasia
Highest stage of its drugs
Phase 2/3, for 3 of its 22 drugs, from ClinicalTrials.gov

Source Open Targets

Drugs22

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.

DrugChEMBL IDTypeOpen Targets' stageStage reported byReportsOf them trialsTrials shown hereSource
FLUDARABINE PHOSPHATECHEMBL1096882Small moleculePhase 2/3ClinicalTrials.gov222Drugs and targets
ANTILYMPHOCYTE IMMUNOGLOBULIN (HORSE)CHEMBL2108981Phase 2/3ClinicalTrials.gov110Drugs and targets
BUSULFANCHEMBL820Small moleculePhase 2/3ClinicalTrials.gov111Drugs and targets
ALEMTUZUMABCHEMBL1201587AntibodyPhase 2ClinicalTrials.gov444Drugs and targets
CYCLOSPORINECHEMBL160ProteinPhase 2ClinicalTrials.gov332Drugs and targets
FLUDARABINECHEMBL1568Small moleculePhase 2ClinicalTrials.gov333Drugs and targets
AUTOLOGOUS CORD BLOODCHEMBL4594461Phase 2ClinicalTrials.gov220Drugs and targets
ELTROMBOPAGCHEMBL461101Small moleculePhase 2ClinicalTrials.gov222Drugs and targets
RITUXIMABCHEMBL1201576AntibodyPhase 2ClinicalTrials.gov222Drugs and targets
TACROLIMUS ANHYDROUSCHEMBL269732Small moleculePhase 2ClinicalTrials.gov222Drugs and targets

1 to 10 of 22

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Targets25

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.

TargetProteinIts drugs that act on itMechanismsHighest stage of those drugsGene in GenesSource
CSF3RGranulocyte colony-stimulating factor receptor3Granulocyte colony stimulating factor receptor agonistPhase 1/2https://conductscience.org/library/genes/1441Drugs and targets
FKBP1APeptidyl-prolyl cis-trans isomerase FKBP1A2FK506-binding protein 1A inhibitorPhase 2https://conductscience.org/library/genes/2280Drugs and targets
RRM1Ribonucleoside-diphosphate reductase RR12Ribonucleoside-diphosphate reductase RR1 inhibitorPhase 2/3https://conductscience.org/library/genes/6240Drugs and targets
RRM2Ribonucleoside-diphosphate reductase RR12Ribonucleoside-diphosphate reductase RR1 inhibitorPhase 2/3https://conductscience.org/library/genes/6241Drugs and targets
RRM2BRibonucleoside-diphosphate reductase RR12Ribonucleoside-diphosphate reductase RR1 inhibitorPhase 2/3https://conductscience.org/library/genes/50484Drugs and targets
ARAndrogen receptor1Androgen Receptor agonistPhase 1/2https://conductscience.org/library/genes/367Drugs and targets
CD52CAMPATH-1 antigen1CAMPATH-1 antigen inhibitorPhase 2https://conductscience.org/library/genes/1043Drugs and targets
IMPDH1Inosine-5'-monophosphate dehydrogenase (IMPDH)1Inosine-5'-monophosphate dehydrogenase (IMPDH) inhibitorPhase 2https://conductscience.org/library/genes/3614Drugs and targets
IMPDH2Inosine-5'-monophosphate dehydrogenase (IMPDH)1Inosine-5'-monophosphate dehydrogenase (IMPDH) inhibitorPhase 2https://conductscience.org/library/genes/3615Drugs and targets
MPLThrombopoietin receptor1Thrombopoietin receptor agonistPhase 2https://conductscience.org/library/genes/4352Drugs and targets

1 to 10 of 25

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Trials13

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.

TrialTitlePhaseParticipantsResults first postedConditionsDrugsLinked bySource
NCT01328587Eltrombopag for Moderate Aplastic AnemiaPhase 2342019-04-23Moderate Aplastic Anemia, Unilineage Bone Marrow Failure DisordersELTROMBOPAGIts record names the diseaseClinicalTrials.gov
NCT00058825Stem Cell Transplant for Hematologic DiseasesPhase 1/2272015-10-22Hematologic MalignanciesALEMTUZUMAB, CYCLOSPORINE, FLUDARABINE, TACROLIMUS, TACROLIMUS ANHYDROUSIts record names the diseaseClinicalTrials.gov
NCT00176878Stem Cell Transplant for Bone Marrow Failure SyndromesPhase 2/3102009-08-06Diamond-Blackfan Anemia, Kostmann's Neutropenia, Shwachman-Diamond SyndromeBUSULFAN, FLUDARABINE PHOSPHATEIts record names the diseaseClinicalTrials.gov
NCT05794425Clinical Study of UCB Combined With UC-MSCs in the Treatment of Bone Marrow Failure DisordersPhase 1/2100Bone Marrow Failure DisordersCYCLOSPORINEIts record names the diseaseClinicalTrials.gov
NCT03579875Alpha/Beta TCD HCT in Patients With Inherited BMF DisordersPhase 248
Fanconi Anemia, Severe Aplastic Anemia, Myelodysplastic Syndromes, T Cell Receptor Alpha/Beta Depletion, Telomere Biology Disorder, Bone Marrow Failure, Dyskeratosis Congenita, Telomere Biology Disorders
ALEMTUZUMAB, RITUXIMABIts record names the diseaseClinicalTrials.gov
NCT01659606Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis CongenitaPhase 240Dyskeratosis Congenita, Hoyeraal Hreidarsson Syndrome, Revesz Syndrome, Aplastic AnemiaALEMTUZUMAB, FLUDARABINE, MYCOPHENOLATE MOFETIL, TACROLIMUS ANHYDROUSIts record names the diseaseClinicalTrials.gov
NCT03710356Evaluating Efficacy and Safety of Danazol in Severe Hematologic or Pulmonary Disease Related to TelomeropathyPhase 1/240Telomere Shortening, Telomere Length, Mean LeukocyteDANAZOLIts record names the diseaseClinicalTrials.gov
NCT04965597Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)Phase 240
Bone Marrow Failure Syndrome, Congenital Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Hereditary Sideroblastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Shwachman-Diamond Syndrome, Hematologic Neoplasm With Germline GATA2 Mutation, Hematologic Neoplasm With Germline SAMD9 Mutation, Hematologic Neoplasm With Germline SAMD9L Mutation
FLUDARABINE PHOSPHATE, TREOSULFANIts record names the diseaseClinicalTrials.gov
NCT01720147Quercetin in Children With Fanconi Anemia; a Pilot StudyPhase 130Fanconi AnemiaQUERCETINIts record names the diseaseClinicalTrials.gov
NCT03206086Eltrombopag for Fanconi AnemiaPhase 225Fanconi AnemiaELTROMBOPAGIts record names the diseaseClinicalTrials.gov

1 to 10 of 13

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Comparisons in its trials1

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.

ComparisonGroup 1, with the outcomeGroup 2, with the outcomeParticipantsTrial
Isolex vs CLINIMACs: Number of Patients Who Engrafted With the Isolex/CLINIMACs System15 of 197 of 827NCT00058825

Papers from its trials4

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

PMIDListed asTrials listing itSource
39820512DERIVED1PubMed
34086408DERIVED1PubMed
32330244DERIVED1PubMed
29958797DERIVED1PubMed

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/HP_0005528

GET https://conductscience.org/api/diseases/v1/HP_0005528/drugs?page=2