Evidence map›Paper›PMID 39292928›Full record

Trial reportThe New England journal of medicine2024

Pomalidomide for Epistaxis in Hereditary Hemorrhagic Telangiectasia.

Hanny Al-Samkari, Raj S Kasthuri, Vivek N Iyer, Allyson M Pishko, Jake E Decker, Clifford R Weiss, Kevin J Whitehead, Miles B Conrad, Marc S Zumberg, Jenny Y Zhou and 8 more

Registry-linked trialAbstract readClinical Trial, Phase IIMulticenter StudyRandomized Controlled Trial
PubMed Full text at PMC Publisher
In one paragraph

Trial report in The New England journal of medicine, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03910244 (Pomalidomide for the Treatment of Bleeding in Hereditary Hemorrhagic Telangiectasia), which is not on this map. Cited by 25 papers.

0numbers the graph read from it
0cells of the map it votes in
25citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT03910244 phase2completednot on this map

Pomalidomide for the Treatment of Bleeding in Hereditary Hemorrhagic Telangiectasia

TypeinterventionalSponsorThe Cleveland ClinicRan2019 to 2023Enrolled145ConditionsTelangiectasia, Hereditary HemorrhagicArmsPomalidomide Oral Product, Placebo oral capsule
3 · Its place in the literature

Who cites it

25 citing papers in PubMed.

  1. Trial
  2. Observational
  3. Article
  4. Article
  5. Article
  6. Article
  7. Article
  8. Article
  9. Observational
  10. Review
  11. Article
  12. Article
  13. Article
  14. What's new in hereditary hemorrhagic telangiectasia?Hematology. American Society of Hematology. Education Program · 2025
    Review
  15. Article
  16. Article
  17. Review
  18. Article
  19. Review
  20. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

18 authors.

Hanny Al-SamkariFrom Massachusetts General Hospital, Boston (H.A.-S.); University of North Carolina, Chapel Hill (R.S.K.), and RTI International, Research Triangle Park (D.M., L.B., B.A.C., S.M.T.) - both in North Carolina; Mayo Clinic, Rochester, MN (V.N.I.); University of Pennsylvania, Philadelphia (A.M.P.); Medical College of Wisconsin, Milwaukee (J.E.D.); Johns Hopkins University, Baltimore (C.R.W.), and CureHHT, Monkton (M.C.) - both in Maryland; University of Utah, Salt Lake City (K.J.W.); University of California, San Francisco, San Francisco (M.B.C.); University of Florida, Gainesville (M.S.Z.); University of California, San Diego, La Jolla (J.Y.Z.); and Taussig Cancer Center and Lerner Research Institute (K.R.M.), Cleveland Clinic (J.P., L.W., K.R.M.), Cleveland.ORCID 0000-0001-6175-1383
Raj S KasthuriFrom Massachusetts General Hospital, Boston (H.A.-S.); University of North Carolina, Chapel Hill (R.S.K.), and RTI International, Research Triangle Park (D.M., L.B., B.A.C., S.M.T.) - both in North Carolina; Mayo Clinic, Rochester, MN (V.N.I.); University of Pennsylvania, Philadelphia (A.M.P.); Medical College of Wisconsin, Milwaukee (J.E.D.); Johns Hopkins University, Baltimore (C.R.W.), and CureHHT, Monkton (M.C.) - both in Maryland; University of Utah, Salt Lake City (K.J.W.); University of California, San Francisco, San Francisco (M.B.C.); University of Florida, Gainesville (M.S.Z.); University of California, San Diego, La Jolla (J.Y.Z.); and Taussig Cancer Center and Lerner Research Institute (K.R.M.), Cleveland Clinic (J.P., L.W., K.R.M.), Cleveland.ORCID 0000-0001-6672-3082
Vivek N IyerFrom Massachusetts General Hospital, Boston (H.A.-S.); University of North Carolina, Chapel Hill (R.S.K.), and RTI International, Research Triangle Park (D.M., L.B., B.A.C., S.M.T.) - both in North Carolina; Mayo Clinic, Rochester, MN (V.N.I.); University of Pennsylvania, Philadelphia (A.M.P.); Medical College of Wisconsin, Milwaukee (J.E.D.); Johns Hopkins University, Baltimore (C.R.W.), and CureHHT, Monkton (M.C.) - both in Maryland; University of Utah, Salt Lake City (K.J.W.); University of California, San Francisco, San Francisco (M.B.C.); University of Florida, Gainesville (M.S.Z.); University of California, San Diego, La Jolla (J.Y.Z.); and Taussig Cancer Center and Lerner Research Institute (K.R.M.), Cleveland Clinic (J.P., L.W., K.R.M.), Cleveland.
Allyson M PishkoFrom Massachusetts General Hospital, Boston (H.A.-S.); University of North Carolina, Chapel Hill (R.S.K.), and RTI International, Research Triangle Park (D.M., L.B., B.A.C., S.M.T.) - both in North Carolina; Mayo Clinic, Rochester, MN (V.N.I.); University of Pennsylvania, Philadelphia (A.M.P.); Medical College of Wisconsin, Milwaukee (J.E.D.); Johns Hopkins University, Baltimore (C.R.W.), and CureHHT, Monkton (M.C.) - both in Maryland; University of Utah, Salt Lake City (K.J.W.); University of California, San Francisco, San Francisco (M.B.C.); University of Florida, Gainesville (M.S.Z.); University of California, San Diego, La Jolla (J.Y.Z.); and Taussig Cancer Center and Lerner Research Institute (K.R.M.), Cleveland Clinic (J.P., L.W., K.R.M.), Cleveland.
Jake E DeckerFrom Massachusetts General Hospital, Boston (H.A.-S.); University of North Carolina, Chapel Hill (R.S.K.), and RTI International, Research Triangle Park (D.M., L.B., B.A.C., S.M.T.) - both in North Carolina; Mayo Clinic, Rochester, MN (V.N.I.); University of Pennsylvania, Philadelphia (A.M.P.); Medical College of Wisconsin, Milwaukee (J.E.D.); Johns Hopkins University, Baltimore (C.R.W.), and CureHHT, Monkton (M.C.) - both in Maryland; University of Utah, Salt Lake City (K.J.W.); University of California, San Francisco, San Francisco (M.B.C.); University of Florida, Gainesville (M.S.Z.); University of California, San Diego, La Jolla (J.Y.Z.); and Taussig Cancer Center and Lerner Research Institute (K.R.M.), Cleveland Clinic (J.P., L.W., K.R.M.), Cleveland.
Clifford R WeissFrom Massachusetts General Hospital, Boston (H.A.-S.); University of North Carolina, Chapel Hill (R.S.K.), and RTI International, Research Triangle Park (D.M., L.B., B.A.C., S.M.T.) - both in North Carolina; Mayo Clinic, Rochester, MN (V.N.I.); University of Pennsylvania, Philadelphia (A.M.P.); Medical College of Wisconsin, Milwaukee (J.E.D.); Johns Hopkins University, Baltimore (C.R.W.), and CureHHT, Monkton (M.C.) - both in Maryland; University of Utah, Salt Lake City (K.J.W.); University of California, San Francisco, San Francisco (M.B.C.); University of Florida, Gainesville (M.S.Z.); University of California, San Diego, La Jolla (J.Y.Z.); and Taussig Cancer Center and Lerner Research Institute (K.R.M.), Cleveland Clinic (J.P., L.W., K.R.M.), Cleveland.ORCID 0000-0003-2180-1731
Kevin J WhiteheadFrom Massachusetts General Hospital, Boston (H.A.-S.); University of North Carolina, Chapel Hill (R.S.K.), and RTI International, Research Triangle Park (D.M., L.B., B.A.C., S.M.T.) - both in North Carolina; Mayo Clinic, Rochester, MN (V.N.I.); University of Pennsylvania, Philadelphia (A.M.P.); Medical College of Wisconsin, Milwaukee (J.E.D.); Johns Hopkins University, Baltimore (C.R.W.), and CureHHT, Monkton (M.C.) - both in Maryland; University of Utah, Salt Lake City (K.J.W.); University of California, San Francisco, San Francisco (M.B.C.); University of Florida, Gainesville (M.S.Z.); University of California, San Diego, La Jolla (J.Y.Z.); and Taussig Cancer Center and Lerner Research Institute (K.R.M.), Cleveland Clinic (J.P., L.W., K.R.M.), Cleveland.
Miles B ConradFrom Massachusetts General Hospital, Boston (H.A.-S.); University of North Carolina, Chapel Hill (R.S.K.), and RTI International, Research Triangle Park (D.M., L.B., B.A.C., S.M.T.) - both in North Carolina; Mayo Clinic, Rochester, MN (V.N.I.); University of Pennsylvania, Philadelphia (A.M.P.); Medical College of Wisconsin, Milwaukee (J.E.D.); Johns Hopkins University, Baltimore (C.R.W.), and CureHHT, Monkton (M.C.) - both in Maryland; University of Utah, Salt Lake City (K.J.W.); University of California, San Francisco, San Francisco (M.B.C.); University of Florida, Gainesville (M.S.Z.); University of California, San Diego, La Jolla (J.Y.Z.); and Taussig Cancer Center and Lerner Research Institute (K.R.M.), Cleveland Clinic (J.P., L.W., K.R.M.), Cleveland.
Marc S ZumbergFrom Massachusetts General Hospital, Boston (H.A.-S.); University of North Carolina, Chapel Hill (R.S.K.), and RTI International, Research Triangle Park (D.M., L.B., B.A.C., S.M.T.) - both in North Carolina; Mayo Clinic, Rochester, MN (V.N.I.); University of Pennsylvania, Philadelphia (A.M.P.); Medical College of Wisconsin, Milwaukee (J.E.D.); Johns Hopkins University, Baltimore (C.R.W.), and CureHHT, Monkton (M.C.) - both in Maryland; University of Utah, Salt Lake City (K.J.W.); University of California, San Francisco, San Francisco (M.B.C.); University of Florida, Gainesville (M.S.Z.); University of California, San Diego, La Jolla (J.Y.Z.); and Taussig Cancer Center and Lerner Research Institute (K.R.M.), Cleveland Clinic (J.P., L.W., K.R.M.), Cleveland.
Jenny Y ZhouFrom Massachusetts General Hospital, Boston (H.A.-S.); University of North Carolina, Chapel Hill (R.S.K.), and RTI International, Research Triangle Park (D.M., L.B., B.A.C., S.M.T.) - both in North Carolina; Mayo Clinic, Rochester, MN (V.N.I.); University of Pennsylvania, Philadelphia (A.M.P.); Medical College of Wisconsin, Milwaukee (J.E.D.); Johns Hopkins University, Baltimore (C.R.W.), and CureHHT, Monkton (M.C.) - both in Maryland; University of Utah, Salt Lake City (K.J.W.); University of California, San Francisco, San Francisco (M.B.C.); University of Florida, Gainesville (M.S.Z.); University of California, San Diego, La Jolla (J.Y.Z.); and Taussig Cancer Center and Lerner Research Institute (K.R.M.), Cleveland Clinic (J.P., L.W., K.R.M.), Cleveland.
Joseph ParambilFrom Massachusetts General Hospital, Boston (H.A.-S.); University of North Carolina, Chapel Hill (R.S.K.), and RTI International, Research Triangle Park (D.M., L.B., B.A.C., S.M.T.) - both in North Carolina; Mayo Clinic, Rochester, MN (V.N.I.); University of Pennsylvania, Philadelphia (A.M.P.); Medical College of Wisconsin, Milwaukee (J.E.D.); Johns Hopkins University, Baltimore (C.R.W.), and CureHHT, Monkton (M.C.) - both in Maryland; University of Utah, Salt Lake City (K.J.W.); University of California, San Francisco, San Francisco (M.B.C.); University of Florida, Gainesville (M.S.Z.); University of California, San Diego, La Jolla (J.Y.Z.); and Taussig Cancer Center and Lerner Research Institute (K.R.M.), Cleveland Clinic (J.P., L.W., K.R.M.), Cleveland.
Derek MarshFrom Massachusetts General Hospital, Boston (H.A.-S.); University of North Carolina, Chapel Hill (R.S.K.), and RTI International, Research Triangle Park (D.M., L.B., B.A.C., S.M.T.) - both in North Carolina; Mayo Clinic, Rochester, MN (V.N.I.); University of Pennsylvania, Philadelphia (A.M.P.); Medical College of Wisconsin, Milwaukee (J.E.D.); Johns Hopkins University, Baltimore (C.R.W.), and CureHHT, Monkton (M.C.) - both in Maryland; University of Utah, Salt Lake City (K.J.W.); University of California, San Francisco, San Francisco (M.B.C.); University of Florida, Gainesville (M.S.Z.); University of California, San Diego, La Jolla (J.Y.Z.); and Taussig Cancer Center and Lerner Research Institute (K.R.M.), Cleveland Clinic (J.P., L.W., K.R.M.), Cleveland.
Marianne ClancyFrom Massachusetts General Hospital, Boston (H.A.-S.); University of North Carolina, Chapel Hill (R.S.K.), and RTI International, Research Triangle Park (D.M., L.B., B.A.C., S.M.T.) - both in North Carolina; Mayo Clinic, Rochester, MN (V.N.I.); University of Pennsylvania, Philadelphia (A.M.P.); Medical College of Wisconsin, Milwaukee (J.E.D.); Johns Hopkins University, Baltimore (C.R.W.), and CureHHT, Monkton (M.C.) - both in Maryland; University of Utah, Salt Lake City (K.J.W.); University of California, San Francisco, San Francisco (M.B.C.); University of Florida, Gainesville (M.S.Z.); University of California, San Diego, La Jolla (J.Y.Z.); and Taussig Cancer Center and Lerner Research Institute (K.R.M.), Cleveland Clinic (J.P., L.W., K.R.M.), Cleveland.
Lauren BradleyFrom Massachusetts General Hospital, Boston (H.A.-S.); University of North Carolina, Chapel Hill (R.S.K.), and RTI International, Research Triangle Park (D.M., L.B., B.A.C., S.M.T.) - both in North Carolina; Mayo Clinic, Rochester, MN (V.N.I.); University of Pennsylvania, Philadelphia (A.M.P.); Medical College of Wisconsin, Milwaukee (J.E.D.); Johns Hopkins University, Baltimore (C.R.W.), and CureHHT, Monkton (M.C.) - both in Maryland; University of Utah, Salt Lake City (K.J.W.); University of California, San Francisco, San Francisco (M.B.C.); University of Florida, Gainesville (M.S.Z.); University of California, San Diego, La Jolla (J.Y.Z.); and Taussig Cancer Center and Lerner Research Institute (K.R.M.), Cleveland Clinic (J.P., L.W., K.R.M.), Cleveland.
Lisa WisniewskiFrom Massachusetts General Hospital, Boston (H.A.-S.); University of North Carolina, Chapel Hill (R.S.K.), and RTI International, Research Triangle Park (D.M., L.B., B.A.C., S.M.T.) - both in North Carolina; Mayo Clinic, Rochester, MN (V.N.I.); University of Pennsylvania, Philadelphia (A.M.P.); Medical College of Wisconsin, Milwaukee (J.E.D.); Johns Hopkins University, Baltimore (C.R.W.), and CureHHT, Monkton (M.C.) - both in Maryland; University of Utah, Salt Lake City (K.J.W.); University of California, San Francisco, San Francisco (M.B.C.); University of Florida, Gainesville (M.S.Z.); University of California, San Diego, La Jolla (J.Y.Z.); and Taussig Cancer Center and Lerner Research Institute (K.R.M.), Cleveland Clinic (J.P., L.W., K.R.M.), Cleveland.
Benjamin A CarperFrom Massachusetts General Hospital, Boston (H.A.-S.); University of North Carolina, Chapel Hill (R.S.K.), and RTI International, Research Triangle Park (D.M., L.B., B.A.C., S.M.T.) - both in North Carolina; Mayo Clinic, Rochester, MN (V.N.I.); University of Pennsylvania, Philadelphia (A.M.P.); Medical College of Wisconsin, Milwaukee (J.E.D.); Johns Hopkins University, Baltimore (C.R.W.), and CureHHT, Monkton (M.C.) - both in Maryland; University of Utah, Salt Lake City (K.J.W.); University of California, San Francisco, San Francisco (M.B.C.); University of Florida, Gainesville (M.S.Z.); University of California, San Diego, La Jolla (J.Y.Z.); and Taussig Cancer Center and Lerner Research Institute (K.R.M.), Cleveland Clinic (J.P., L.W., K.R.M.), Cleveland.
Sonia M ThomasFrom Massachusetts General Hospital, Boston (H.A.-S.); University of North Carolina, Chapel Hill (R.S.K.), and RTI International, Research Triangle Park (D.M., L.B., B.A.C., S.M.T.) - both in North Carolina; Mayo Clinic, Rochester, MN (V.N.I.); University of Pennsylvania, Philadelphia (A.M.P.); Medical College of Wisconsin, Milwaukee (J.E.D.); Johns Hopkins University, Baltimore (C.R.W.), and CureHHT, Monkton (M.C.) - both in Maryland; University of Utah, Salt Lake City (K.J.W.); University of California, San Francisco, San Francisco (M.B.C.); University of Florida, Gainesville (M.S.Z.); University of California, San Diego, La Jolla (J.Y.Z.); and Taussig Cancer Center and Lerner Research Institute (K.R.M.), Cleveland Clinic (J.P., L.W., K.R.M.), Cleveland.
Keith R McCraeFrom Massachusetts General Hospital, Boston (H.A.-S.); University of North Carolina, Chapel Hill (R.S.K.), and RTI International, Research Triangle Park (D.M., L.B., B.A.C., S.M.T.) - both in North Carolina; Mayo Clinic, Rochester, MN (V.N.I.); University of Pennsylvania, Philadelphia (A.M.P.); Medical College of Wisconsin, Milwaukee (J.E.D.); Johns Hopkins University, Baltimore (C.R.W.), and CureHHT, Monkton (M.C.) - both in Maryland; University of Utah, Salt Lake City (K.J.W.); University of California, San Francisco, San Francisco (M.B.C.); University of Florida, Gainesville (M.S.Z.); University of California, San Diego, La Jolla (J.Y.Z.); and Taussig Cancer Center and Lerner Research Institute (K.R.M.), Cleveland Clinic (J.P., L.W., K.R.M.), Cleveland.ORCID 0000-0001-7340-475X

Funding

1/2 Pomalidomide for Bleeding in Patients with Hereditary Hemorrhagic Telangiectasia (HHT)UH3HL140097 · NHLBI · CLEVELAND CLINIC LERNER COM-CWRU · PI MCCRAE, KEITH R. · 2020 to 2023
$3.8M
2/2 Pomalidomide for Bleeding in Patients with Hereditary Hemorrhagic Telangiectasia (HHT) Bleeding Data Coordinating CenterU24HL140090 · NHLBI · RESEARCH TRIANGLE INSTITUTE · PI THOMAS, SONIA M · 2019 to 2023
$2.1M
Antiangiogenic Therapy to Reduce Bleeding and Improve Health-Related Quality of Life in Hereditary Hemorrhagic TelangiectasiaK23HL159313 · NHLBI · MASSACHUSETTS GENERAL HOSPITAL · PI Hanny T Al-Samkari · 2022 to 2026
$999k
1/2 Pomalidomide for Bleeding in Patients with Hereditary Hemorrhagic Telangiectasia (HHT)UG3HL140097 · NHLBI · CLEVELAND CLINIC LERNER COM-CWRU · PI MCCRAE, KEITH R. · 2019 to 2019
$876k
NHLBI NIH HHS K23 HL159313NHLBI NIH HHS U24 HL140090NHLBI NIH HHS U24HL140090 (to ST)NHLBI NIH HHS UG3 HL140097NHLBI NIH HHS UG3HL140097 (to KRM)NHLBI NIH HHS UH3 HL140097
6 · The paper itself

Abstract

backgroundHereditary hemorrhagic telangiectasia (HHT) is characterized by extensive telangiectasias and arteriovenous malformations. The primary clinical manifestation is epistaxis that results in iron-deficiency anemia and reduced health-related quality of life.

methodsWe conducted a randomized, placebo-controlled trial to evaluate the safety and efficacy of pomalidomide for the treatment of HHT. We randomly assigned patients, in a 2:1 ratio, to receive pomalidomide at a dose of 4 mg daily or matching placebo for 24 weeks. The primary outcome was the change from baseline through week 24 in the Epistaxis Severity Score (a validated bleeding score in HHT; range, 0 to 10, with higher scores indicating worse bleeding). A reduction of 0.71 points or more is considered clinically significant. A key secondary outcome was the HHT-specific quality-of-life score (range, 0 to 16, with higher scores indicating more limitations).

resultsThe trial was closed to enrollment in June 2023 after a planned interim analysis met a prespecified threshold for efficacy. A total of 144 patients underwent randomization; 95 patients were assigned to receive pomalidomide and 49 to receive placebo. The baseline mean (±SD) Epistaxis Severity Score was 5.0±1.5, a finding consistent with moderate-to-severe epistaxis. At 24 weeks, the mean difference between the pomalidomide group and the placebo group in the change from baseline in the Epistaxis Severity Score was -0.94 points (95% confidence interval [CI], -1.57 to -0.31; P = 0.004). The mean difference in the changes in the HHT-specific quality-of-life score between the groups was -1.4 points (95% CI, -2.6 to -0.3). Adverse events that were more common in the pomalidomide group than in the placebo group included neutropenia, constipation, and rash.

conclusionsAmong patients with HHT, pomalidomide treatment resulted in a significant, clinically relevant reduction in epistaxis severity. No unexpected safety signals were identified. (Funded by the National Heart, Lung, and Blood Institute; PATH-HHT Clinicaltrials.gov number, NCT03910244).

Indexed as

EpistaxisTelangiectasia, Hereditary HemorrhagicThalidomideAgedAngiogenesis InhibitorsConstipationDouble-Blind MethodDrug EruptionsFemaleHumansMaleMiddle AgedNeutropeniaQuality of LifeSeverity of Illness IndexTreatment OutcomeAngiogenesis InhibitorspomalidomideThalidomide

Identifiers

PMID39292928
PMCPMC11412318

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