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A mature B- or T- cell leukemia with progressive clinical course. It is characterized by the presence of medium-sized lymphocytes with visible nucleoli (prolymphocytes) in the peripheral blood, bone marrow, and spleen.
No approved treatments are currently available for prolymphocytic leukemia. An additional 1 compound holds orphan drug designation.
While no drugs are FDA-approved specifically for prolymphocytic leukemia, some of the following designated compounds may be used off-label in clinical practice. Treatment decisions should be made in consultation with a specialist familiar with this condition.
The following drugs have received orphan drug designation from the FDA for prolymphocytic leukemia. Orphan designation reflects regulatory interest and does not indicate approval for treatment.
Brand Name | Generic Name | Sponsor |
|---|
25 clinical trials registered, 11 recruiting. Interventions under study include drug therapy, other interventions, biologic therapy, and procedural interventions. Pipeline includes 7 PHASE2, 11 PHASE1, 1 NA. Research is sponsored by a mix of industry and academic institutions.
NCT ID | Title | Phase | Sponsor | Status |
|---|---|---|---|---|
[NCT01137825](https://clinicaltrials.gov/study/NCT01137825) |
Data assembled from 4 of 12 sources · Last updated Sep 18, 2026, 8:32 PM UTC
Genetic and Rare Diseases Info Center
Designated
Exclusivity End |
|---|
Designation Status |
|---|
blinatumomab | blinatumomab | Amgen, Inc. | 2008 | — | Designated |
Gene therapy approaches for prolymphocytic leukemia have been reported in the published literature.
25 trials found
Registry of Older Patients With Cancer |
— |
UNC Lineberger Comprehensive Cancer Center |
RECRUITING |
[NCT01137643](https://clinicaltrials.gov/study/NCT01137643) | Tissue, Blood, and Body Fluid Sample Collection From Patients With Hematologic Cancer | — | UNC Lineberger Comprehensive Cancer Center | RECRUITING |
[NCT04195633](https://clinicaltrials.gov/study/NCT04195633) | Donor Stem Cell Transplant With Treosulfan, Fludarabine, and Total-Body Irradiation for the Treatment of Hematological Malignancies | PHASE2 | Fred Hutchinson Cancer Center | RECRUITING |
[NCT06420076](https://clinicaltrials.gov/study/NCT06420076) | Sequential CAR-T Cells Therapy for CD5/CD7 Positive T-cell Acute Lymphoblastic Leukemia and Lymphoblastic Lymphoma Using CD5/CD7-Specific CAR-T Cells | PHASE1 | Essen Biotech | RECRUITING |
[NCT07311746](https://clinicaltrials.gov/study/NCT07311746) | Phase Ib/II Trial of Cladribine/Ruxolitinib/Venetoclax in Patients With Relapsed/Refractory T-cell Prolymphocytic Leukemia | PHASE1 | M.D. Anderson Cancer Center | RECRUITING |
81 publications have been identified in PubMed for prolymphocytic leukemia. Research spans Case Report / Case Series (47%), Basic Science / Preclinical (21%), and Clinical Trial Publication (16%).
Research Type | Count | % of Total |
|---|---|---|
Patient case studies | 38 | 47% |
Laboratory research | 17 | 21% |
Clinical study results | 13 | 16% |
Research summaries | 5 | 6% |
New treatment approaches | 5 | 6% |
Disease patterns and progression | 2 | 2% |
Other research | 1 | 1% |
Ermann DA (2026). [PMID: 42078476](https://pubmed.ncbi.nlm.nih.gov/42078476/). *EJHaem*. [Clinical Trial Publication]
Shumilov E (2026). [PMID: 42206428](https://pubmed.ncbi.nlm.nih.gov/42206428/). *Haematologica*. [Clinical Trial Publication]
Kadia TM (2026). [PMID: 41536780](https://pubmed.ncbi.nlm.nih.gov/41536780/). *Blood neoplasia*. [Basic Science / Preclinical]
Maffei R (2026). [PMID: 42044618](https://pubmed.ncbi.nlm.nih.gov/42044618/). *Cancer Genet*. [Case Report / Case Series]
Merrill M (2026). [PMID: 41906811](https://pubmed.ncbi.nlm.nih.gov/41906811/). *Leuk Lymphoma*. [Case Report / Case Series]
Kaseb H (2026). [PMID: 31082044](https://pubmed.ncbi.nlm.nih.gov/31082044/). *Unknown Journal*. [Review / Meta-Analysis]
Pramanik D (2026). [PMID: 41871898](https://pubmed.ncbi.nlm.nih.gov/41871898/). *BMJ Case Rep*. [Case Report / Case Series]
Perrone S (2026). [PMID: 41954626](https://pubmed.ncbi.nlm.nih.gov/41954626/). *Ann Hematol*. [Case Report / Case Series]
Parsi M (2026). [PMID: 31082180](https://pubmed.ncbi.nlm.nih.gov/31082180/). *Unknown Journal*. [Case Report / Case Series]
Ravishankar A (2026). [PMID: 41899567](https://pubmed.ncbi.nlm.nih.gov/41899567/). *Cancers (Basel)*. [Review / Meta-Analysis]