RARE DISEASE
Idiopathic aplastic anemia
Idiopathic aplastic anemia
Idiopathic aplastic anemia
Synonyms: Idiopathic bone marrow failure
Synonyms: Idiopathic bone marrow failure
Synonyms: Idiopathic bone marrow failure
Drug discovery
4
drugs
With orphan designations
Overview
Idiopathic aplastic anemia (IAA) is a rare, immune-mediated bone marrow failure disorder characterized by pancytopenia and hypocellular bone marrow without an identifiable cause. It arises from cytotoxic T-cell destruction of hematopoietic stem cells, leading to impaired blood cell production. Diagnosis requires exclusion of inherited and secondary causes. Management depends on disease severity, age, and donor availability, with immunosuppressive therapy (IST) or hematopoietic stem cell transplantation (HSCT) as cornerstone treatments [1][3][8][17][19].
Therapies
First-line: HSCT for severe cases (ages <40) with matched donors (5-year survival: 80–90%); IST (anti-thymocyte globulin + cyclosporine) ± eltrombopag for older adults or non-transplant candidates [1][3][7][8][12].
Refractory disease: Salvage therapies include alternative donor HSCT, thrombopoietin agonists (eltrombopag), or combination IST [4][7][15].
Categories: rare hematological diseases, rare transplant-related disorders
Research Papers
201 drug discovery papers about Idiopathic aplastic anemia, with 2 first-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
201 drug discovery papers about Idiopathic aplastic anemia, with 2 first-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
2026-08-01 | Idiopathic AA Renal Amyloidosis Treated with Tocilizumab — A Case Report
Background Amyloidosis represents a heterogeneous group of disorders characterized by the deposition of misfolded protein. Among its subtypes, serum amyloid A (AA) amyloidosis arises when persistent systemic inflammation drives hepatic production of SAA. Excess SAA misfolds into insoluble fibrils that deposit in organs such as the kidneys, causing progressive proteinuria, loss of renal function and eventual renal failure. Standard management aims to control the underlying inflammation to reduce SAA production and halt further amyloid deposition. For example, interleukin-6 blockade with tocilizumab, which suppresses SAA production, has shown encouraging results in small series of patients with secondary amyloidosis. [1,2] In rare cases without an identifiable source of inflammation, however, no targeted therapies have been shown to be effective. Case Report We report a 48-year-old female first found incidentally at the age of 37 to have renal impairment. Over the past decade, her eGFR had declined to 12mL/min/1.73m 2 , with 3g/L of proteinuria. Renal biopsy in 2020 confirmed widespread AA amyloid deposits with severe interstitial fibrosis and 11/16 glomeruli globally sclerosed, with SAA concentrations exceeding 16,000ng/mL. Throughout her entire course, she had no infectious, rheumatologic or constitutional symptoms. Other than a RF titer of 30 IUx103/L, extensive investigations were negative, including bone marrow biopsy, rheumatologic markers (CCP, ANA, dsDNA, C3/C4, ANCA, ESR, CRP) and infectious serologies (hepatitis, syphilis, schistosoma, strongyloides, whipples, tuberculosis, HIV, HTLV, filariasis). A thorough genetic investigation was also negative including sequencing for the SAA1 gene, panels containing genes associated with hereditary amyloidosis and autoinflammatory diseases, as well as a research exome. A brief trial of colchicine was initially trialed but stopped due to worsening kidney function. Tocilizumab 160mg SC every 2 weeks was initiated in December 2024 in an attempt to suppress hepatic SAA production. A year after, SAA levels fell from >16,000 to 5,427ng/mL. Her GFR stabilized at 13 mL/min/1.73m 2 , and dialysis has not yet been required at the time of writing. Conclusion Idiopathic AA amyloidosis is a therapeutic challenge due to the condition’s rarity and lack of standard therapies. While there are numerous publications demonstrating the efficacy of tocilizumab in secondary AA amyloidosis,[1-3] our case is the first we are aware of, describing this therapeutic effect in idiopathic AA amyloidosis. This case underscores the central role of interleukin-6 in SAA production and may form the foundation for future studies with more patients over a longer duration to further explore the disease-modifying benefits of tocilizumab in idiopathic AA amyloidosis. References [1.] Okuda Y. Mod Rheumatol 2019;29:268-74. [2.] Courties A. Amyloid 2015;22:84-92. [3.] Yamagata A. BMC Nephrol 2017;18:377.
2026-07-30 | Living Donor Liver Transplantation as Salvage Therapy for Steroid-Refractory Chronic Hepatic GVHD Following Allogeneic Hematopoietic Stem Cell Transplantation.
Hepatic graft-versus-host disease (GVHD) is a rare but life-threatening complication following allogeneic hematopoietic stem cell transplantation (HSCT). Steroid-refractory hepatic GVHD carries a high risk of mortality with limited salvage options. Liver transplantation after HSCT poses unique challenges due to dual alloimmunity and is rarely pursued, particularly in pediatric patients. We report a pediatric patient with steroid-refractory chronic hepatic GVHD successfully treated with living donor liver transplantation (LDLT) from a parental donor. A 13-year-old male underwent allogeneic HSCT for idiopathic severe aplastic anemia and developed progressive chronic hepatic GVHD characterized by ductopenia and severe cholestasis. His disease was refractory to corticosteroids and multiple additional therapies, including ruxolitinib, mesenchymal stromal cells, infliximab, sirolimus, anti-thymocyte globulin, alpha-1 antitrypsin, cyclophosphamide, and emapalumab. His course was complicated by Epstein-Barr virus (EBV) associated post-transplant lymphoproliferative disorder (PTLD), successfully treated with rituximab. Fifteen months after HSCT, and seven months after clearance of EBV viremia, he underwent LDLT from a parental donor. Donor selection preserved the option for future donor-derived EBV-specific cytotoxic T lymphocyte therapy if needed. Eleven months following LDLT, the patient remains clinically well, with normal liver function, sustained full donor chimerism, and no evidence of recurrent GVHD or PTLD. LDLT may represent a viable option in select pediatric patients with irreversible chronic hepatic GVHD. This case underscores the importance of early multidisciplinary planning and highlights the absence of liver-specific biomarkers or targeted therapies to guide management. Dedicated research into preventive and regenerative strategies is warranted.
2026-05-01 | C29-13 Somatic Variants Rescue Bone Marrow Failure, But Not Lung Disease in Patients With Germline Variants in TINF2
Abstract Introduction Telomeres are essential for genome maintenance. Rare variants disrupting telomere maintenance genes cause a spectrum of short telomere syndromes, with idiopathic pulmonary fibrosis (IPF) as the most common phenotype. Variants in the shelterin component TIN2 typically cause severe pediatric dyskeratosis congenita (DC) and bone marrow failure. However, a subset of patients with germline TINF2 variants presents with less severe, adult-onset disease, including IPF, suggesting the presence of modifying factors. Methods We identified three patients with pathogenic germline TINF2 hotspot variants evaluated at the University of Pittsburgh Medical Center for lung transplantation. Two had a family history of DC; one had a sporadic presentation. Clinical genetic testing and deep exome sequencing was used to identify somatic variants. We validated the function of somatic variants in vitro using CRISPR/Cas9-mediated genome editing in multiple cell lines and in patient-derived induced pluripotent stem cells (iPSCs). Results All three patients had somatic variants in the DNA-binding domain of POT1 where tumor-specific variants have been previously reported. Edited cells showed no signs of DNA damage, genomic instability, or altered growth kinetics. Telomeres progressively lengthened in all edited lines. iPSCs reprogrammed from patient PBMCs, which harbored both the germline TINF2 and somatic POT1 variants, also demonstrated gradual telomere lengthening, verifying the POT1 variants are functional and sufficient for telomere elongation, even in the context of the pathogenic TINF2 variant. Conclusions Somatic POT1 variants appear to rescue hematopoietic function and cause telomere lengthening in vitro. POT1 editing is efficient and does not appear to influence genome stability. We propose that genetically modifying POT1 may be an effective therapy to rescue the bone marrow of patients with short telomeres. This abstract is funded by: NA
2026-05-01 | Two Sides of the Same Coin, Pathogenic RTEL1 Mutations
Background Regulator of telomere elongation helicase 1 (RTEL1) is a gene that encodes a DNA helicase crucial for DNA repair and telomere maintenance. It belongs to a family of genes associated with telomere biology disorders (TBD). The phenotypic spectrum of RTEL1 mutations is highly variable, encompassing bone marrow failure, idiopathic pulmonary fibrosis, cirrhosis, and malignancy due to excessive telomere erosion. Objective We describe two patients at opposite ends of age, both with pathogenic variants in RTEL1. Clinical Case Descriptions Patient 1 was admitted to the hospital at 3 years of age due to failure to thrive and chronic diarrhea, found to have refractory CMV colitis and CMV and HHV6 viremia. She developed toxic megacolon secondary to CMV, requiring a subtotal colectomy. She underwent a reduced-intensity conditioning matched sibling donor bone marrow transplant (BMT) and ileorectal anastomosis. She was referred at age 4 for immune evaluation due to severe inflammatory bowel disease (IBD)-like gastrointestinal (GI) disease with suboptimal response to steroids and vedolizumab. She was treated with danazol with resolution of her diarrhea and, interestingly, hair regrowth. Patient 2 is a 61-year-old female with leukopenia and recurrent fevers without infection. She was previously healthy with no history of autoimmunity or recurrent infections. She had atypical CD8-positive T cell lymphoid infiltration on liver biopsy with elevated inflammatory cytokines and was referred for immune dysregulation. Investigations Patient 1 had an elevated fecal calprotectin of 889 ug/g and had a homozygous pathogenic RTEL1 variant c.2869C>T (P.Arg957Trp). Patient 2 had an expanded CD8+ T cell population (74%) with a low CD4/CD8 ratio (0.19) and reduced B cells (1%), while immunoglobulin levels remained within the normal range. Cytokine studies showed elevated IFN-y (49pg/mL), elevated CXCL9 (32, 454pg/mL), and elevated soluble IL-2 receptor (5,899 pg/mL) with no evidence of hemophagocytic lymphohistiocytosis (HLH) or granulomas on bone marrow biopsy. She had a heterozygous pathogenic RTEL1 variant: c.3791G>A (p.Arg1264His). Discussion These two cases highlight the phenotypically distinct presentations of pathogenic RTEL1 across the age spectrum. When discovered in older adults, it raises the question of somatic mutations, though germline heterozygous pathogenic variants have been described. Danazol and BMT are being considered in patient 2. Figure 1. Schematic representation of RTEL1 variants with respect to functional protein domains. Variants in P1 and P2 with respect to the RTEL1 function protein domains. HD, N-terminal helicase domain; HHD1 and HHD2, harmonin homology domains 1 and 2; PIP, PCNA-interacting protein box; RING, C-terminal C4C4 type RING domain. Reference transcript NM_001283009.2, adapted from (1). Created using Biorender.com.
2026-04-30 | Efficacy and safety of cyclosporine plus luspatercept versus cyclosporine in newly diagnosed non-transfusion-dependent non-severe aplastic anemia: A prospective randomized trial.
The clinical need for treating anemia in aplastic anemia (AA) patients remains unmet. Luspatercept has been shown to be effective in myelodysplastic neoplasms (MDS). Patients who were newly diagnosed with non-transfusion-dependent non-severe AA (NTD-NSAA) were randomly assigned to receive either cyclosporine (CsA) combined with luspatercept or CsA monotherapy at a 1:1 ratio. This study (ClinicalTrials.gov NCT05399732) aimed to compare their treatment responses, safety, disease progression, and outcomes. In total, 58 patients participated in the final analysis, with 29 receiving CsA+luspatercept and 29 receiving CsA monotherapy. With a median follow-up of 12 months (range: 6-25) and 12 months (range: 7-25), respectively, the overall response rates (ORRs) were 69.0% vs. 37.9% (p = 0.018) at the 3rd month, 79.3% vs. 51.7% (p = 0.027) at the 6th month, and 72.4% vs. 51.7% (p = 0.104) at the end of follow-up. Patients receiving CsA+luspatercept had a shorter time to achieve a positive response than those receiving CsA alone (p = 0.004). A post hoc subgroup analysis based on age (< 60 vs. ≥60 years) showed no significant difference in ORRs for those < 60 years old. However, for patients ≥ 60 years old receiving CsA+luspatercept, a significantly greater ORR was demonstrated at both the 3rd month (p = 0.032) and 6th month (p = 0.046) compared with CsA monotherapy. Compared with CsA monotherapy, the combination of CsA and luspatercept resulted in a higher response rate and a shorter time to response for patients with NTD-NSAA, with an acceptable safety profile. The benefit of CsA+luspatercept was most pronounced in older patients.
2026-08-01 | Idiopathic AA Renal Amyloidosis Treated with Tocilizumab — A Case Report
Background Amyloidosis represents a heterogeneous group of disorders characterized by the deposition of misfolded protein. Among its subtypes, serum amyloid A (AA) amyloidosis arises when persistent systemic inflammation drives hepatic production of SAA. Excess SAA misfolds into insoluble fibrils that deposit in organs such as the kidneys, causing progressive proteinuria, loss of renal function and eventual renal failure. Standard management aims to control the underlying inflammation to reduce SAA production and halt further amyloid deposition. For example, interleukin-6 blockade with tocilizumab, which suppresses SAA production, has shown encouraging results in small series of patients with secondary amyloidosis. [1,2] In rare cases without an identifiable source of inflammation, however, no targeted therapies have been shown to be effective. Case Report We report a 48-year-old female first found incidentally at the age of 37 to have renal impairment. Over the past decade, her eGFR had declined to 12mL/min/1.73m 2 , with 3g/L of proteinuria. Renal biopsy in 2020 confirmed widespread AA amyloid deposits with severe interstitial fibrosis and 11/16 glomeruli globally sclerosed, with SAA concentrations exceeding 16,000ng/mL. Throughout her entire course, she had no infectious, rheumatologic or constitutional symptoms. Other than a RF titer of 30 IUx103/L, extensive investigations were negative, including bone marrow biopsy, rheumatologic markers (CCP, ANA, dsDNA, C3/C4, ANCA, ESR, CRP) and infectious serologies (hepatitis, syphilis, schistosoma, strongyloides, whipples, tuberculosis, HIV, HTLV, filariasis). A thorough genetic investigation was also negative including sequencing for the SAA1 gene, panels containing genes associated with hereditary amyloidosis and autoinflammatory diseases, as well as a research exome. A brief trial of colchicine was initially trialed but stopped due to worsening kidney function. Tocilizumab 160mg SC every 2 weeks was initiated in December 2024 in an attempt to suppress hepatic SAA production. A year after, SAA levels fell from >16,000 to 5,427ng/mL. Her GFR stabilized at 13 mL/min/1.73m 2 , and dialysis has not yet been required at the time of writing. Conclusion Idiopathic AA amyloidosis is a therapeutic challenge due to the condition’s rarity and lack of standard therapies. While there are numerous publications demonstrating the efficacy of tocilizumab in secondary AA amyloidosis,[1-3] our case is the first we are aware of, describing this therapeutic effect in idiopathic AA amyloidosis. This case underscores the central role of interleukin-6 in SAA production and may form the foundation for future studies with more patients over a longer duration to further explore the disease-modifying benefits of tocilizumab in idiopathic AA amyloidosis. References [1.] Okuda Y. Mod Rheumatol 2019;29:268-74. [2.] Courties A. Amyloid 2015;22:84-92. [3.] Yamagata A. BMC Nephrol 2017;18:377.
2026-07-30 | Living Donor Liver Transplantation as Salvage Therapy for Steroid-Refractory Chronic Hepatic GVHD Following Allogeneic Hematopoietic Stem Cell Transplantation.
Hepatic graft-versus-host disease (GVHD) is a rare but life-threatening complication following allogeneic hematopoietic stem cell transplantation (HSCT). Steroid-refractory hepatic GVHD carries a high risk of mortality with limited salvage options. Liver transplantation after HSCT poses unique challenges due to dual alloimmunity and is rarely pursued, particularly in pediatric patients. We report a pediatric patient with steroid-refractory chronic hepatic GVHD successfully treated with living donor liver transplantation (LDLT) from a parental donor. A 13-year-old male underwent allogeneic HSCT for idiopathic severe aplastic anemia and developed progressive chronic hepatic GVHD characterized by ductopenia and severe cholestasis. His disease was refractory to corticosteroids and multiple additional therapies, including ruxolitinib, mesenchymal stromal cells, infliximab, sirolimus, anti-thymocyte globulin, alpha-1 antitrypsin, cyclophosphamide, and emapalumab. His course was complicated by Epstein-Barr virus (EBV) associated post-transplant lymphoproliferative disorder (PTLD), successfully treated with rituximab. Fifteen months after HSCT, and seven months after clearance of EBV viremia, he underwent LDLT from a parental donor. Donor selection preserved the option for future donor-derived EBV-specific cytotoxic T lymphocyte therapy if needed. Eleven months following LDLT, the patient remains clinically well, with normal liver function, sustained full donor chimerism, and no evidence of recurrent GVHD or PTLD. LDLT may represent a viable option in select pediatric patients with irreversible chronic hepatic GVHD. This case underscores the importance of early multidisciplinary planning and highlights the absence of liver-specific biomarkers or targeted therapies to guide management. Dedicated research into preventive and regenerative strategies is warranted.
2026-05-01 | C29-13 Somatic Variants Rescue Bone Marrow Failure, But Not Lung Disease in Patients With Germline Variants in TINF2
Abstract Introduction Telomeres are essential for genome maintenance. Rare variants disrupting telomere maintenance genes cause a spectrum of short telomere syndromes, with idiopathic pulmonary fibrosis (IPF) as the most common phenotype. Variants in the shelterin component TIN2 typically cause severe pediatric dyskeratosis congenita (DC) and bone marrow failure. However, a subset of patients with germline TINF2 variants presents with less severe, adult-onset disease, including IPF, suggesting the presence of modifying factors. Methods We identified three patients with pathogenic germline TINF2 hotspot variants evaluated at the University of Pittsburgh Medical Center for lung transplantation. Two had a family history of DC; one had a sporadic presentation. Clinical genetic testing and deep exome sequencing was used to identify somatic variants. We validated the function of somatic variants in vitro using CRISPR/Cas9-mediated genome editing in multiple cell lines and in patient-derived induced pluripotent stem cells (iPSCs). Results All three patients had somatic variants in the DNA-binding domain of POT1 where tumor-specific variants have been previously reported. Edited cells showed no signs of DNA damage, genomic instability, or altered growth kinetics. Telomeres progressively lengthened in all edited lines. iPSCs reprogrammed from patient PBMCs, which harbored both the germline TINF2 and somatic POT1 variants, also demonstrated gradual telomere lengthening, verifying the POT1 variants are functional and sufficient for telomere elongation, even in the context of the pathogenic TINF2 variant. Conclusions Somatic POT1 variants appear to rescue hematopoietic function and cause telomere lengthening in vitro. POT1 editing is efficient and does not appear to influence genome stability. We propose that genetically modifying POT1 may be an effective therapy to rescue the bone marrow of patients with short telomeres. This abstract is funded by: NA
2026-05-01 | Two Sides of the Same Coin, Pathogenic RTEL1 Mutations
Background Regulator of telomere elongation helicase 1 (RTEL1) is a gene that encodes a DNA helicase crucial for DNA repair and telomere maintenance. It belongs to a family of genes associated with telomere biology disorders (TBD). The phenotypic spectrum of RTEL1 mutations is highly variable, encompassing bone marrow failure, idiopathic pulmonary fibrosis, cirrhosis, and malignancy due to excessive telomere erosion. Objective We describe two patients at opposite ends of age, both with pathogenic variants in RTEL1. Clinical Case Descriptions Patient 1 was admitted to the hospital at 3 years of age due to failure to thrive and chronic diarrhea, found to have refractory CMV colitis and CMV and HHV6 viremia. She developed toxic megacolon secondary to CMV, requiring a subtotal colectomy. She underwent a reduced-intensity conditioning matched sibling donor bone marrow transplant (BMT) and ileorectal anastomosis. She was referred at age 4 for immune evaluation due to severe inflammatory bowel disease (IBD)-like gastrointestinal (GI) disease with suboptimal response to steroids and vedolizumab. She was treated with danazol with resolution of her diarrhea and, interestingly, hair regrowth. Patient 2 is a 61-year-old female with leukopenia and recurrent fevers without infection. She was previously healthy with no history of autoimmunity or recurrent infections. She had atypical CD8-positive T cell lymphoid infiltration on liver biopsy with elevated inflammatory cytokines and was referred for immune dysregulation. Investigations Patient 1 had an elevated fecal calprotectin of 889 ug/g and had a homozygous pathogenic RTEL1 variant c.2869C>T (P.Arg957Trp). Patient 2 had an expanded CD8+ T cell population (74%) with a low CD4/CD8 ratio (0.19) and reduced B cells (1%), while immunoglobulin levels remained within the normal range. Cytokine studies showed elevated IFN-y (49pg/mL), elevated CXCL9 (32, 454pg/mL), and elevated soluble IL-2 receptor (5,899 pg/mL) with no evidence of hemophagocytic lymphohistiocytosis (HLH) or granulomas on bone marrow biopsy. She had a heterozygous pathogenic RTEL1 variant: c.3791G>A (p.Arg1264His). Discussion These two cases highlight the phenotypically distinct presentations of pathogenic RTEL1 across the age spectrum. When discovered in older adults, it raises the question of somatic mutations, though germline heterozygous pathogenic variants have been described. Danazol and BMT are being considered in patient 2. Figure 1. Schematic representation of RTEL1 variants with respect to functional protein domains. Variants in P1 and P2 with respect to the RTEL1 function protein domains. HD, N-terminal helicase domain; HHD1 and HHD2, harmonin homology domains 1 and 2; PIP, PCNA-interacting protein box; RING, C-terminal C4C4 type RING domain. Reference transcript NM_001283009.2, adapted from (1). Created using Biorender.com.
2026-04-30 | Efficacy and safety of cyclosporine plus luspatercept versus cyclosporine in newly diagnosed non-transfusion-dependent non-severe aplastic anemia: A prospective randomized trial.
The clinical need for treating anemia in aplastic anemia (AA) patients remains unmet. Luspatercept has been shown to be effective in myelodysplastic neoplasms (MDS). Patients who were newly diagnosed with non-transfusion-dependent non-severe AA (NTD-NSAA) were randomly assigned to receive either cyclosporine (CsA) combined with luspatercept or CsA monotherapy at a 1:1 ratio. This study (ClinicalTrials.gov NCT05399732) aimed to compare their treatment responses, safety, disease progression, and outcomes. In total, 58 patients participated in the final analysis, with 29 receiving CsA+luspatercept and 29 receiving CsA monotherapy. With a median follow-up of 12 months (range: 6-25) and 12 months (range: 7-25), respectively, the overall response rates (ORRs) were 69.0% vs. 37.9% (p = 0.018) at the 3rd month, 79.3% vs. 51.7% (p = 0.027) at the 6th month, and 72.4% vs. 51.7% (p = 0.104) at the end of follow-up. Patients receiving CsA+luspatercept had a shorter time to achieve a positive response than those receiving CsA alone (p = 0.004). A post hoc subgroup analysis based on age (< 60 vs. ≥60 years) showed no significant difference in ORRs for those < 60 years old. However, for patients ≥ 60 years old receiving CsA+luspatercept, a significantly greater ORR was demonstrated at both the 3rd month (p = 0.032) and 6th month (p = 0.046) compared with CsA monotherapy. Compared with CsA monotherapy, the combination of CsA and luspatercept resulted in a higher response rate and a shorter time to response for patients with NTD-NSAA, with an acceptable safety profile. The benefit of CsA+luspatercept was most pronounced in older patients.
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Drug Discovery Landscape
4 orphan drug designations for Idiopathic aplastic anemia, including 1 approved therapy.
4 orphan drug designations for Idiopathic aplastic anemia, including 1 approved therapy.
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
cord blood derived T regulatory cells | cell therapies | FDA | 2025-04-04 | — | Cellenkos Inc |
adult hemogenic endothelial cells | antibodies | FDA | 2015-05-27 | — | HemoGenyx LLC |
eltrombopag [Promacta] | small molecules | FDA | 2013-11-08 | 2014-08-26 | Novartis Pharmaceuticals Corp. |
Interleukin-1 alpha, human recombinant | proteins | FDA | 1991-06-17 | — | Immunex Corporation |
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