Disease · MONDO_0100280

Waldenstrom macroglobulinemia

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. Waldenstrom macroglobulinemia. Diseases, record MONDO_0100280. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0100280

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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: Waldenstrom macroglobulinemia; Open Targets
Open Targets ID: MONDO_0100280; Open Targets
Other names: Waldenstrom's macroglobulinemia, Waldenström Macroglobulinemia, lymphoplasmacytic lymphoma with IgM gammopathy; Open Targets
Disease Ontology: DOID:0060901; Disease Ontology
MeSH: D008258; MeSH
NCI Thesaurus: C80307; NCI Thesaurus
Orphanet: ORPHA:33226; Orphanet
Highest stage of its drugs: Approval, for 1 of its 84 drugs, from EMA Human Drugs and PMDA; Open Targets

Drugs84

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
ZANUBRUTINIB	CHEMBL3936761	Small molecule	Approval	EMA Human Drugs and PMDA	21	18	18	Drugs and targets
RITUXIMAB	CHEMBL1201576	Antibody	Phase 3	ClinicalTrials.gov	49	49	49	Drugs and targets
BORTEZOMIB	CHEMBL325041	Small molecule	Phase 3	ClinicalTrials.gov	23	23	23	Drugs and targets
IBRUTINIB	CHEMBL1873475	Small molecule	Phase 3	ClinicalTrials.gov	21	21	21	Drugs and targets
DEXAMETHASONE	CHEMBL384467	Small molecule	Phase 3	ClinicalTrials.gov	17	17	17	Drugs and targets
CYCLOPHOSPHAMIDE	CHEMBL1200796	Small molecule	Phase 3	ClinicalTrials.gov	10	10	10	Drugs and targets
FLUDARABINE	CHEMBL1568	Small molecule	Phase 3	ClinicalTrials.gov	3	3	3	Drugs and targets
CHLORAMBUCIL	CHEMBL515	Small molecule	Phase 3	ClinicalTrials.gov	2	2	2	Drugs and targets
FLUDARABINE PHOSPHATE	CHEMBL1096882	Small molecule	Phase 3	ClinicalTrials.gov	2	2	2	Drugs and targets
BENDAMUSTINE	CHEMBL487253	Small molecule	Phase 2	ClinicalTrials.gov	9	9	9	Drugs and targets

1 to 10 of 84

See all 84 drugs

Targets147

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
BTK	Tyrosine-protein kinase BTK	7	Tyrosine-protein kinase BTK inhibitor	Approval	https://conductscience.org/library/genes/695	Drugs and targets
MS4A1	B-lymphocyte antigen CD20	5	B-lymphocyte antigen CD20 binding agent	Phase 3	https://conductscience.org/library/genes/931	Drugs and targets
ADRM1	26S proteasome	3	26S proteasome inhibitor	Phase 3	https://conductscience.org/library/genes/11047	Drugs and targets
BCL2	Apoptosis regulator Bcl-2	3	Apoptosis regulator Bcl-2 inhibitor; Bcl-2 mRNA antisense inhibitor	Phase 2	https://conductscience.org/library/genes/596	Drugs and targets
CRBN	CRL4(CRBN) E3 ubiquitin ligase	3	CRL4(CRBN) E3 ubiquitin ligase inhibitor; CRL4(CRBN) E3 ubiquitin ligase modulator	Phase 2	https://conductscience.org/library/genes/51185	Drugs and targets
CUL4A	CRL4(CRBN) E3 ubiquitin ligase	3	CRL4(CRBN) E3 ubiquitin ligase inhibitor; CRL4(CRBN) E3 ubiquitin ligase modulator	Phase 2	https://conductscience.org/library/genes/8451	Drugs and targets
DDB1	CRL4(CRBN) E3 ubiquitin ligase	3	CRL4(CRBN) E3 ubiquitin ligase inhibitor; CRL4(CRBN) E3 ubiquitin ligase modulator	Phase 2	https://conductscience.org/library/genes/1642	Drugs and targets
PIK3CD	PI3-kinase class I	3	PI3-kinase class I inhibitor; PI3-kinase p110-delta subunit inhibitor	Phase 2	https://conductscience.org/library/genes/5293	Drugs and targets
POLA1	DNA polymerase (alpha/delta/epsilon)	3	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 3	https://conductscience.org/library/genes/5422	Drugs and targets
POLA2	DNA polymerase (alpha/delta/epsilon)	3	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 3	https://conductscience.org/library/genes/23649	Drugs and targets

1 to 10 of 147

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Trials140

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
NCT02343120	Study of the Safety and Pharmacokinetics of BGB-3111 in Subjects With B-Cell Lymphoid Malignancies	Phase 1/2	385	2022-04-27	B-cell Malignancies	ZANUBRUTINIB	Its record names the disease	ClinicalTrials.gov
NCT01799889	Study to Evaluate Efficacy, Safety, Tolerability, and Pharmacodynamics of Entospletinib in Adults With Relapsed or Refractory Hematologic Malignancies	Phase 2	326	2020-09-23	Chronic Lymphocytic Leukemia, Mantle Cell Lymphoma, Diffuse Large B-cell Lymphoma, Non-FL Indolent Non-Hodgkin's Lymphoma, Follicular Lymphoma	ENTOSPLETINIB	Its record names the disease	ClinicalTrials.gov
NCT03053440	A Study Comparing BGB-3111 and Ibrutinib in Participants With Waldenström's Macroglobulinemia (WM)	Phase 3	201	2023-06-09	Waldenström's Macroglobulinemia	IBRUTINIB, ZANUBRUTINIB	Open Targets' reading, confirmed by NLM's indexing of its papers	ClinicalTrials.gov
NCT02165397	Ibrutinib With Rituximab in Adults With Waldenström's Macroglobulinemia	Phase 3	181	2020-11-16	Waldenström's Macroglobulinemia	IBRUTINIB, RITUXIMAB	Open Targets' reading, confirmed by NLM's indexing of its papers	ClinicalTrials.gov
NCT00481871	Study of Pralatrexate & Gemcitabine With B12 & Folic Acid to Treat Relapsed/Refractory Lymphoproliferative Malignancies	Phase 1/2	119	2012-09-18	Relapsed or Refractory Lymphoproliferative Malignancies, Hodgkin's Lymphoma, Peripheral T-cell Lymphoma, B-cell Lymphoma, Waldenstrom's Macroglobulinemia	GEMCITABINE HYDROCHLORIDE, PRALATREXATE	Its record names the disease	ClinicalTrials.gov
NCT02180724	An Open-label, Phase 2 Study of ACP-196 in Subjects With Waldenström Macroglobulinemia	Phase 2	107	2020-04-24	Waldenström Macroglobulinemia (WM)	ACALABRUTINIB	Its record names the disease	ClinicalTrials.gov
NCT03779113	An Open-label, Dose Escalation Trial to Evaluate the Safety and Pharmacokinetics of HMPL-523 in Patients With Lymphoma	Phase 1	69	2026-02-18	Non Hodgkin Lymphoma	SOVLEPLENIB	Open Targets' reading of its record	ClinicalTrials.gov
NCT00422799	Combination Bortezomib and Rituximab in Patients With Waldenstrom's Macroglobulinemia (WM)	Phase 2	63	2018-01-26	Waldenstrom's Macroglobulinemia	BORTEZOMIB, RITUXIMAB	Its record names the disease	ClinicalTrials.gov
NCT01614821	Ibrutinib (PCI-32765) in Waldenstrom's Macroglobulinemia	Phase 2	63	2020-01-21	Waldenstrom's Macroglobulinemia	IBRUTINIB	Its record names the disease	ClinicalTrials.gov
NCT00718419	A Study for Patients That Have Been Previously Been Treated in Waldenstrom's Macroglobulinemia or Multiple Myeloma	Phase 2	56	2020-09-03	Waldenstrom's Macroglobulinemia, Multiple Myeloma	ENZASTAURIN	Its record names the disease	ClinicalTrials.gov

1 to 10 of 140

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Comparisons in its trials17

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
Arm A: Ibrutinib vs Arm B: Zanubrutinib: deaths from any cause	19 of 99	14 of 102	201	NCT03053440	Trial comparisons
Arm A: Ibrutinib vs Arm B: Zanubrutinib: serious adverse events	50 of 98	59 of 101	199	NCT03053440	Trial comparisons
Multiple Myeloma (MM) vs Waldenstrom's Macroglobulinemia (WM): Number of Participants Who Died Due to Progressive Disease During the 30 Days Following Discontinuation From Study Treatment	0 of 14	1 of 42	56	NCT00718419	Trial comparisons
Multiple Myeloma (MM) vs Waldenstrom's Macroglobulinemia (WM): serious adverse events	4 of 14	9 of 42	56	NCT00718419	Trial comparisons
300 mg Ofatumumab, Wk. 1; 1000/2000 mg Ofatumumab, Wk. 2-4/2-5 vs 300 mg Ofatumumab, Wk. 1; 2000 mg Ofatumumab, Wk. 2-5: Number of Participants With OR for Cycle 1 (Excluding the Redosing Cycle), as Assessed by the Investigator	5 of 15	14 of 22	37	NCT00811733	Trial comparisons
300 mg Ofatumumab, Wk. 1; 1000/2000 mg Ofatumumab, Wk. 2-4/2-5 vs 300 mg Ofatumumab, Wk. 1; 2000 mg Ofatumumab, Wk. 2-5: Number of Participants With Overall Response (OR) for Cycle 1 (Including the Redosing Cycle), as Assessed by the Investigator	7 of 15	15 of 22	37	NCT00811733	Trial comparisons
300 mg Ofatumumab, Wk. 1; 1000/2000 mg Ofatumumab, Wk. 2-4/2-5 vs 300 mg Ofatumumab, Wk. 1; 2000 mg Ofatumumab, Wk. 2-5: serious adverse events	5 of 15	7 of 22	37	NCT00811733	Trial comparisons
300 mg Ofatumumab, Wk. 1; 1000/2000 mg Ofatumumab, Wk. 2-4/2-5 vs 300 mg Ofatumumab, Wk. 1; 2000 mg Ofatumumab, Wk. 2-5: Number of Participants With at Least One Confirmed Positive Post-ofatumumab HAHA Result	1 of 11	4 of 20	31	NCT00811733	Trial comparisons
MYD88 Mutated, CXCR4 Wild-type vs MYD88 Mutated, CXCR4 Mutated: Overall Response Rate by MYD88 L265P and CXCR4-WHIM Status	11 of 11	14 of 15	26	NCT02400437	Trial comparisons
MZL: Umbralisib vs WM: Umbralisib: Number of Participants With at Least One Adverse Event (AE)	8 of 8	13 of 13	21	NCT03364231	Trial comparisons

1 to 10 of 17

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Papers from its trials70

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

PMID	Listed as	Trials listing it	Source
32108102	RESULT	1	PubMed
31771394	RESULT	1	PubMed
30089632	RESULT	1	PubMed
29869556	RESULT	1	PubMed
27053773	RESULT	1	PubMed
26284823	RESULT	1	PubMed
26139427	RESULT	1	PubMed
24523121	RESULT	1	PubMed
23287861	RESULT	1	PubMed
21566092	RESULT	1	PubMed

1 to 10 of 70

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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_0100280

GET https://conductscience.org/api/diseases/v1/MONDO_0100280/drugs?page=2

API reference For AI assistants