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Condition (MeSH)

Anemia, Refractory

ClinicalTrials.gov lists 335 trials for Anemia, Refractory or a narrower condition beneath it. National Cancer Institute (NCI) has the most among industry and government lead sponsors, with 27.

MeSH D000753 Hemic and Lymphatic Diseases C15 Listed by Orphanet as a rare disease
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Phase 1 Phase 1 70 Phase 1/2 Phase 1/2 40 Phase 2 Phase 2 143 Phase 2/3 Phase 2/3 6 Phase 3 Phase 3 26 Phase 4 Phase 4 2 Not applicable Not applicable 22 Observational Observational 26 Phase 1 Phase 1 70 Phase 1/2 Phase 1/2 40 Phase 2 Phase 2 143 Phase 2/3 Phase 2/3 6 Phase 3 Phase 3 26 Phase 4 Phase 4 2 Not applicable Not applicable 22 Observational Observational 26

Trials per phase (log scale)

95 of the 335 trials are tagged with Anemia, Refractory itself; the rest are tagged with a narrower condition beneath it.

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The PDF carries up to 50 rows of each section, where the page shows the first few, with the sources listed. Enter a work email and confirm it from the message we send, and the report follows by email.

When did Anemia, Refractory trials start? (35)

Shown in this order: most recent first, 1990 onwards. 2026 is partial: trials registered with a 2026 start date so far, not a full year.

Trials over time, by phase

  • Phase 1
  • Phase 1/2
  • Phase 2
  • Phase 2/3
  • Phase 3
  • Phase 4
  • Not applicable
  • Observational

Top of scale: 22

1992: 1 trials (Phase 2 1) 1993: 3 trials (Phase 3 1, Phase 2 1, Phase 1 1) 1994: 1 trials (Phase 2 1) 1995: 1 trials (Phase 2/3 1) 1996: 8 trials (Observational 1, Phase 3 2, Phase 2 3, Phase 1 2) 1997: 2 trials (Phase 2 2) 1998: 22 trials (Phase 3 4, Phase 2/3 1, Phase 2 11, Phase 1/2 2, Phase 1 4) 1999: 17 trials (Observational 1, Not applicable 1, Phase 3 1, Phase 2 6, Phase 1/2 2, Phase 1 6) 2000: 20 trials (Phase 3 2, Phase 2/3 1, Phase 2 7, Phase 1/2 3, Phase 1 7) 2001: 17 trials (Observational 2, Not applicable 1, Phase 2 10, Phase 1/2 2, Phase 1 2) 2002: 14 trials (Not applicable 1, Phase 3 2, Phase 2 4, Phase 1/2 3, Phase 1 4) 2003: 13 trials (Not applicable 2, Phase 2 8, Phase 1/2 1, Phase 1 2) 2004: 16 trials (Observational 1, Not applicable 1, Phase 3 1, Phase 2 7, Phase 1/2 1, Phase 1 5) 2005: 18 trials (Phase 3 2, Phase 2/3 1, Phase 2 7, Phase 1/2 1, Phase 1 7) 2006: 13 trials (Phase 3 1, Phase 2/3 1, Phase 2 6, Phase 1/2 2, Phase 1 3) 2007: 17 trials (Not applicable 1, Phase 4 1, Phase 3 1, Phase 2 6, Phase 1/2 2, Phase 1 6) 2008: 8 trials (Observational 1, Not applicable 1, Phase 2 4, Phase 1/2 1, Phase 1 1) 2009: 13 trials (Observational 1, Phase 2 8, Phase 1/2 1, Phase 1 3) 2010: 13 trials (Phase 3 1, Phase 2 7, Phase 1/2 2, Phase 1 3) 2011: 8 trials (Observational 1, Phase 3 1, Phase 2 4, Phase 1/2 2) 2012: 8 trials (Observational 1, Not applicable 1, Phase 2 5, Phase 1 1) 2013: 10 trials (Observational 3, Not applicable 3, Phase 3 1, Phase 2 2, Phase 1/2 1) 2014: 7 trials (Not applicable 1, Phase 2 3, Phase 1/2 2, Phase 1 1) 2015: 5 trials (Observational 1, Phase 3 2, Phase 2 2) 2016: 5 trials (Observational 1, Not applicable 1, Phase 2 1, Phase 1 2) 2017: 4 trials (Phase 2 2, Phase 1/2 1, Phase 1 1) 2018: 9 trials (Phase 3 1, Phase 2 4, Phase 1/2 2, Phase 1 2) 2019: 6 trials (Phase 3 3, Phase 2 1, Phase 1/2 1, Phase 1 1) 2020: 9 trials (Observational 3, Not applicable 1, Phase 2/3 1, Phase 2 3, Phase 1 1) 2021: 3 trials (Not applicable 1, Phase 2 2) 2022: 6 trials (Observational 2, Not applicable 1, Phase 2 2, Phase 1 1) 2023: 3 trials (Not applicable 1, Phase 2 2) 2024: 4 trials (Observational 1, Phase 2 1, Phase 1/2 1, Phase 1 1) 2025: 14 trials (Not applicable 2, Phase 2 3, Phase 1/2 6, Phase 1 3)

Trials registered with a start date in each year, 1990 onward, stacked by phase (complete years only; the current partial year is in the table's notes). The table below lists the same counts, most recent years first.

Trials started per year
Year Trials started
2026 (partial year) 15
2025 14
2024 4
2023 3
2022 6
2021 3
2020 9
2019 6
2018 9
2017 4

Showing 10 of 35 earlier years.

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Forecast of the full-year count, planned starts included, not rows of this table: about 15 trials starting in 2026 and about 13 in 2027, the average of the 24 months through September, counted as registered and carried forward. The rows of this table count trials registered so far with a start date in the year, planned starts included; the forecast looks ahead to the count for the whole year, so it is not the same measure. In backtests on conditions of this size, forecasts made part-way through the year were typically off by about 28 percent and ran about 20 percent low against the count later registered; forecasts made before the year began were off by about 39 percent and ran about 37 percent low against the count later registered. These figures rest on a few years of history (target years from 2018 to 2025, varying by figure), place each condition as it is mapped today rather than as it was then, and score against a count for the newest year that was still being revised. Only condition series were scored.

Prediction, not a registry value: median of 36 months from start to primary completion across 18 trials still running at the 2026-10-09 snapshot; registered median of 49 months across 245 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.

What phase are Anemia, Refractory trials in? (8)

Shown in this order: phase order.

Show the trials by phase as a table
Trials by phase
Phase Trials
Phase 1 70
Phase 1/2 40
Phase 2 143
Phase 2/3 6
Phase 3 26
Phase 4 2
Not applicable 22
Observational 26

How many Anemia, Refractory trials are recruiting? (7)

Shown in this order: the order a trial moves through, from not yet recruiting to unknown.

Trials by recruitment status
Status Trials
Not yet recruiting 7
Recruiting 37
Active, not recruiting 15
Completed 207
Terminated 46

Showing 5 of 7 statuses.

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Status as registered on ClinicalTrials.gov. Each trial is counted once.

Which lead sponsors run the most Anemia, Refractory trials? (4)

Shown in this order: most trials first. Lead sponsors the registry classes as industry, NIH, US federal, other government or network. A hospital or university the registry files as government can appear here. Sponsors it classes as academic or other are shown on the full page, because that class cannot separate an institution from a named investigator.

Lead sponsors
Sponsor Trials
National Cancer Institute (NCI) 27
European Organisation for Research and Treatment of Cancer - EORTC 7
National Heart, Lung, and Blood Institute (NHLBI) 7
Traws Pharma, Inc. 4

Which drugs are being tested for Anemia, Refractory? (2)

Shown in this order: most trials first. One row per drug, matched to a single molecule under all its names, ranked by number of trials.

Drugs tested
Drug Trials
Cytoxan 66
Busulfan 46

Interventions as named in the registry (13)

Shown in this order: most trials first.

Interventions as named in the registry
Drug Trials
SQ Fludarabine 76
Cyclosporine 0.05% 47
Mofetil Mycophenolate 44
Peripheral blood stem cell transplantation 40
Total Body Irradiation 38

Showing 5 of 13 interventions.

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These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.

Which serious adverse events do Anemia, Refractory trials report? (10)

Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.

Serious adverse events in this condition's trials
Adverse event Subjects affected Trials
Thrombocytopenia 724 7
Leukopenia 707 1
Neutropenia 692 6
Anemia 550 8
Infection w/ Grade 3 or 4 Neutropenia 352 1

Showing 5 of 10 adverse events.

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Which publications cover Anemia, Refractory? (32)

Shown in this order: major-topic papers first, then newest, then most linked to trials.

Publications
Publication Journal Year Major topic
Efficacy and safety of romiplostim in refractory aplastic anaemia: a Phase II/III, multicentre, open-label study. British journal of haematology 2021 Yes
Successful treatment with daratumumab for post-HSCT refractory hemolytic anemia. Pediatric blood & cancer 2020 Yes
Romiplostim is effective for eltrombopag-refractory aplastic anemia: results of a retrospective study. International journal of hematology 2020 Yes
Radiofrequency ablation for patients with refractory symptomatic anaemia secondary to gastric antral vascular ectasia. United European gastroenterology journal 2019 Yes
Multicentre, open-label, randomised, parallel-group, superiority study to compare the efficacy of octreotide therapy 40 mg monthly versus standard of care in patients with refractory anaemia due to gastrointestinal bleeding from small bowel angiodysplasias: a protocol of the OCEAN trial. BMJ open 2016 Yes

Showing 5 of 32 publications.

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Prevalence (3)

Shown in this order: jurisdiction, then disease name. Orphanet observation for the mapped ORPHA concept; the mapping relation column says whether that concept is exactly this condition or broader or narrower.

Prevalence
Orphanet disease Type Class Geography Jurisdiction Mapping relation
Myelodysplastic neoplasm with low blasts Point prevalence Unknown Worldwide Not stated Exact
Myelodysplastic neoplasm with low blasts Point prevalence Unknown Worldwide Not stated Unspecified
Myelodysplastic neoplasm with low blasts Point prevalence Unknown Worldwide Not stated Exact

Rare-disease identity (2)

Shown in this order: exact, broader, narrower, other.

Rare-disease identity
Orphanet disease ORPHA code Relation Source
Myelodysplastic neoplasm with low blasts 98826 Exact Orphanet Rare Disease Ontology (ORDO)
Myelodysplastic neoplasm with low blasts 98826 Exact Orphadata Science knowledge files, Product 1

Sub-conditions in MeSH (1)

MeSH descendants one level down with at least one trial, most trials first.

Not on this page

Not measured: delivery record.

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