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ORPHA:33226

Waldenström macroglobulinemia

low confidenceDisorder

Publications

10,588

Trials

290

Interventional, condition-specific

Researchers

1,197

Distinct authors in sample

Gene link

—

Readiness

2/6

Stages with a signal

Clinical definition (Orphanet)

A rare indolent B-cell non-Hodgkin lymphoma, characterized by the infiltration of monoclonal lymphoplasmacytic cells in the bone marrow and the production of serum immunoglobulin M (IgM) monoclonal protein.

How rare: How common this is has not been clearly measured.

Orphanet entry

Cross-references

Joined from Mondo / Orphanet. MeSH labels may enter searches; UMLS / OMIM / NCIT are stored for reference.

Additional Mondo synonyms (3)

Waldenstrom macroglobulinemia · Waldenstrom's macroglobulinemia · Waldenström Macroglobulinemia

Research stages

Trial readiness signals

Where this condition sits on an open-data research pipeline — not how close a treatment is, and not medical advice. Empty stages often mean “not in these databases under this Mondo ID,” not “impossible.”

2/6 stages with a signal

An interventional trial matched this condition name on ClinicalTrials.gov — see trials below.

  1. Gene identifiedNot found

    No GenCC disease–gene assertion in this build

  2. LiteraturePresent

    10,588 matched papers (4,077 in last 10 years) Source

  3. Phenotype characterisedNot checked

    Not yet enriched from Monarch / HPO

  4. Animal modelNot checked

    Not yet enriched from Monarch / Alliance

  5. Orphan designationNot checked

    FDA/EMA orphan-drug designation not enriched yet

  6. Interventional trialPresent

    290 matched on ClinicalTrials.gov (36 recruiting in sample)

Biology

Genes and phenotypes

Gene–disease validity from GenCC, plus phenotypes and animal models joined from Monarch Initiative via Mondo ID — not a clinical diagnosis aid.

Do we know what causes it?

Not yet — the cause hasn't been pinned down in GenCC.

No strong gene–disease assertion joined for this Orphanet entity.

Phenotypes (Monarch / HPO)

Not enriched in this build — Monarch phenotype joins were not run for this record.

Animal models (Monarch / Alliance)

Not enriched in this build.

Literature

Is anyone studying this?

10,588

10,588 papers — among the better-studied rare conditions, though still a fraction of common-disease literature (breast cancer: over 700,000). Median papers in the last 10 years for a rare disease in this dataset (publications denominator n=5449) is 41.

10,588 papers since the earliest indexed year in this search — median last-10-year count for a rare disease in this dataset is 41 (publications denominator n=5449).

4,077 in the last 10 years · low confidence

Phrase hits: 10,588 · MeSH hits: 0

Open Europe PMC search

Who's working on it?

1,197

Distinct author names in 200 sampled papers — named people below.

Who's working on it?

People publishing on this condition (sampled Europe PMC records). Affiliation is the most recent found in that sample.

  1. 01
    Castillo JJ26 papers · 2026

    Bing Center for Waldenström's Macroglobulinemia, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA.

    Papers in Europe PMC
  2. 02
    Treon SP23 papers · 2026

    Shayna Sarosiek, MD, and Steven P. Treon, MD, PhD, Bing Center for Waldenström Macroglobulinemia, Dana-Farber Cancer Institute, Boston, MA, Department of Medicine, Harvard Medical School, Boston, MA; Andrew R. Branagan, MD, PhD, Department of Medicine, Harvard Medical School, Boston, MA, Center for Multiple Myeloma, Massachusetts General Hospital, Boston, MA; and Jorge J. Castillo, MD, Bing Center for Waldenström Macroglobulinemia, Dana-Farber Cancer Institute, Boston, MA, Department of Medicine, Harvard Medical School, Boston, MA.

    Papers in Europe PMC
  3. 03
    Sarosiek S19 papers · 2026

    Bing Center for Waldenström Macroglobulinemia, Dana-Farber Cancer Institute, Harvard Medical School, Boston, Massachusetts, USA.

    Papers in Europe PMC
  4. 04
    Guijosa A14 papers · 2026

    Bing Center for Waldenström Macroglobulinemia, Dana-Farber Cancer Institute, Boston, MA.

    Papers in Europe PMC
  5. 05
    Hunter ZR13 papers · 2026

    Dana Farber Cancer Institute, Boston, Massachusetts, USA.

    Papers in Europe PMC
  6. 06
    Patterson CJ12 papers · 2026

    Bing Center for Waldenström Macroglobulinemia, Dana-Farber Cancer Institute, Boston, Massachusetts, USA.

    Papers in Europe PMC
  7. 07
    Kastritis E11 papers · 2026

    Department of Clinical Therapeutics, Faculty of Medicine, National and Kapodistrian University of Athens, 80 Vassilisis Sofias Avenue, Athens 11527, Greece.

    Papers in Europe PMC
  8. 08
    Dimopoulos MA10 papers · 2026

    Department of Clinical Therapeutics, National and Kapodistrian University of Athens, School of Medicine, Alexandra Hospital, Athens, Greece.

    Papers in Europe PMC
  9. 09
    Tsakmaklis N10 papers · 2026

    Dana Farber Cancer Institute, Boston, Massachusetts, USA.

    Papers in Europe PMC
  10. 10
    Branagan AR9 papers · 2026

    Shayna Sarosiek, MD, and Steven P. Treon, MD, PhD, Bing Center for Waldenström Macroglobulinemia, Dana-Farber Cancer Institute, Boston, MA, Department of Medicine, Harvard Medical School, Boston, MA; Andrew R. Branagan, MD, PhD, Department of Medicine, Harvard Medical School, Boston, MA, Center for Multiple Myeloma, Massachusetts General Hospital, Boston, MA; and Jorge J. Castillo, MD, Bing Center for Waldenström Macroglobulinemia, Dana-Farber Cancer Institute, Boston, MA, Department of Medicine, Harvard Medical School, Boston, MA.

    Papers in Europe PMC

Clinical research

Is a treatment being tested?

290

interventional trials for this specific condition

290 interventional trials matched this specific condition name; 36 currently recruiting in our sample.

Data as of 27 July 2026

290 interventional trials — more than 73% of diseases in the trials denominator have none at all (5114 of 7003; this disease is at the 99.5th percentile).

low confidence · 99.5th percentile (trials denominator)

Recruiting interventional trials

From the matched ClinicalTrials.gov set

290 interventional trials matched after quoted-phrase search and title/condition post-filter.

Observational and natural-history studies

22 observational studies match this condition. These do not test a treatment and are not counted in the interventional-trial headline, but they are genuine research: natural-history work often defines the endpoints needed for a future rare-disease trial, and families may be able to enroll.

Recruiting or not-yet-recruiting

Open the complete matched search on ClinicalTrials.gov

Where to find support

We do not yet link condition-specific patient organisations. These umbrella groups support undiagnosed and ultra-rare families:

India — NPRD

Last verified 2026-07-26

This ORPHAcode is not on our curated NPRD list (direct or Mondo-parent match). That does not decide clinical eligibility; families in India should ask a notified Centre of Excellence about current coverage.

Hand-curated for this project. ORPHAcode mappings are best-effort and may be incomplete or imprecise for umbrella categories. Parent (Mondo) matches mean the policy lists a broader category — confirm eligibility with a Centre of Excellence. Financial entitlements summarised from public policy statements and may change. This is not official government guidance.

How we counted this

Europe PMC query (preferred label + any corrected label + Orphanet and Mondo exact synonyms, stoplisted; unioned with resolved MeSH labels when available). UMLS / OMIM / NCIT cross-references are stored on the overview but are not added to the query string.

"Waldenström macroglobulinemia" OR "Waldenstrom macroglobulinemia" OR "Waldenstrom's macroglobulinemia"

Run this search on Europe PMC

ClinicalTrials.gov query (quoted phrases + MeSH via query.cond, plus recall-expansion terms when used):

"Waldenström macroglobulinemia" OR "Waldenstrom macroglobulinemia" OR "Waldenstrom's macroglobulinemia"

Interventional trials matched via: phrase (mesh = registered under a MeSH descriptor no name phrase would catch; recall-expansion = gene / selected parent terms used only for trials).

Study-type breakdown: 290 interventional · 22 observational · 2 expanded access. Only interventional studies enter the trial headline.

Query health: ok — strategies attempted: phrase; with hits: phrase

Run this search on ClinicalTrials.gov

Confidence reasoning

  • Preferred label is multi-word and distinctive
  • No synonyms dropped by stoplist
  • No label/synonym collisions with other diseases in this corpus
  • Publication count (10588) is extremely high with unknown/missing prevalence — treat as possible over-matching, not proven research intensity

Ingested 2026-07-26T23:33:37.929Z