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Post-transplant lymphoproliferative disorder (PTLD) is a polyclonal (benign) or clonal (malignant) proliferation of lymphoid cells that develops as a consequence of immunosuppression in a recipient of a solid organ or bone marrow allograft. PTLDs comprise a spectrum ranging from early, Epstein-Barr virus (EBV)-driven polyclonal lymphoid proliferations to EBV-positive or EBV- negative lymphomas of predominantly B-cell or less often T-cell type. (WHO, 2001)
Biomarker and diagnostic research for post-transplant lymphoproliferative disease has been reported in the published literature.
No approved treatments are currently available for post-transplant lymphoproliferative disease. An additional 2 compounds hold orphan drug designation.
While no drugs are FDA-approved specifically for post-transplant lymphoproliferative disease, 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 post-transplant lymphoproliferative disease. Orphan designation reflects regulatory interest and does not indicate approval for treatment.
Brand Name | Generic Name | Sponsor |
|---|
Estimated prevalence: 1-5 in 10,000 (Uncommon).
19 clinical trials registered, 6 recruiting. Interventions under study include biologic therapy, drug therapy, procedural interventions, and other interventions. Pipeline includes 2 PHASE3, 4 PHASE2, 8 PHASE1. 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 5 of 12 sources · Last updated Sep 19, 2026, 5:33 AM UTC
European rare disease database
Genetic and Rare Diseases Info Center
Designated
Exclusivity End |
|---|
Designation Status |
|---|
nanatinostat and valganciclovir | nanatinostat and valganciclovir | Viracta Therapeutics, Inc. | 2019 | — | Designated |
autologous Epstein-Barr specific T-cells | autologous Epstein-Barr specific T-cells | BCM Center for Cell and Gene Therapy | 2017 | — | Designated |
Gene therapy approaches for post-transplant lymphoproliferative disease have been reported in the published literature.
19 trials found
Registry of Older Patients With Cancer |
— |
UNC Lineberger Comprehensive Cancer Center |
RECRUITING |
[NCT06672705](https://clinicaltrials.gov/study/NCT06672705) | Epcoritamab for the Treatment of Relapsed or Refractory Post Transplant Lymphoproliferative Disorders | PHASE1 | Timothy Voorhees | RECRUITING |
[NCT03394365](https://clinicaltrials.gov/study/NCT03394365) | A Phase 3 Study of Tabelecleucel for Participants With Epstein-Barr Virus-Associated Post-Transplant Lymphoproliferative Disease After Failure With Rituximab or Rituximab and Chemotherapy | PHASE3 | Pierre Fabre Medicament | 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 |
[NCT06723457](https://clinicaltrials.gov/study/NCT06723457) | Epcoritamab and Lenalidomide in Treating Patients With Refractory or Relapsed Immunodeficiency-Related Large B-Cell Lymphoma | PHASE2 | Reem Karmali | RECRUITING |
156 publications have been identified in PubMed for post-transplant lymphoproliferative disease. Research spans Case Report / Case Series (42%), Review / Meta-Analysis (20%), and Epidemiology / Natural History (15%).
Research Type | Count | % of Total |
|---|---|---|
Patient case studies | 66 | 42% |
Research summaries | 31 | 20% |
Disease patterns and progression | 23 | 15% |
Clinical study results | 12 | 8% |
Laboratory research | 9 | 6% |
New treatment approaches | 7 | 4% |
Testing and diagnosis research | 6 | 4% |
Other research | 2 | 1% |
Sun Q (2026). [PMID: 41676774](https://pubmed.ncbi.nlm.nih.gov/41676774/). *Hepatobiliary Surg Nutr*. [Other]
Surma J (2026). [PMID: 42181437](https://pubmed.ncbi.nlm.nih.gov/42181437/). *Cureus*. [Case Report / Case Series]
Shimogawa T (2026). [PMID: 42144298](https://pubmed.ncbi.nlm.nih.gov/42144298/). *Transplant Proc*. [Case Report / Case Series]
Rosen R (2026). [PMID: 41343951](https://pubmed.ncbi.nlm.nih.gov/41343951/). *Am J Otolaryngol*. [Epidemiology / Natural History]
Lombardi AF (2026). [PMID: 40952491](https://pubmed.ncbi.nlm.nih.gov/40952491/). *Abdom Radiol (NY)*. [Review / Meta-Analysis]
Khandker SS (2026). [PMID: 42194828](https://pubmed.ncbi.nlm.nih.gov/42194828/). *J Clin Med*. [Review / Meta-Analysis]
Camargo JF (2026). [PMID: 41642712](https://pubmed.ncbi.nlm.nih.gov/41642712/). *J Antimicrob Chemother*. [Epidemiology / Natural History]
Desbaillets N (2026). [PMID: 41993175](https://pubmed.ncbi.nlm.nih.gov/41993175/). *Front Immunol*. [Case Report / Case Series]
Morihisa Y (2026). [PMID: 41697538](https://pubmed.ncbi.nlm.nih.gov/41697538/). *Clin J Gastroenterol*. [Case Report / Case Series]
Unknown (2026). [PMID: 41989797](https://pubmed.ncbi.nlm.nih.gov/41989797/). *Blood*. [Clinical Trial Publication]
AI-curated news mentioning post-transplant lymphoproliferative disease
Updated Jun 15, 2026
A national retrospective study examines the clinicopathological landscape of pediatric monomorphic post-transplant lymphoproliferative disorder (PTLD). This research provides insights into the disease's characteristics and may inform future treatment strategies.
A recent case study highlights an uncommon presentation of post-transplant lymphoproliferative disorder in a child following liver transplantation, characterized by massive splenomegaly. This finding contributes to the understanding of rare disease manifestations in pediatric transplant patients.
A rare case report details gastrointestinal-localized polymorphic post-transplant lymphoproliferative disorder following kidney transplantation after allogeneic hematopoietic stem cell transplantation. This case highlights the complexities and potential complications in post-transplant care.