About

Allyson Marie Pishko, MD, MSCE, is an Assistant Professor of Medicine in Hematology-Oncology at the Hospital of the University of Pennsylvania. She serves as an Attending Physician at the same hospital and is the Associate Program Director for Hematology/Oncology at the University of Pennsylvania. Her clinical expertise focuses on bleeding and thrombotic disorders, including Hereditary Hemorrhagic Telangiectasia. Dr. Pishko completed her undergraduate studies at the University of Notre Dame in 2008, earned her MD from the University of Pittsburgh School of Medicine in 2012, and obtained a Master of Science in Clinical Epidemiology from the University of Pennsylvania in 2018.

Research topics

• Medicine
• Internal medicine
• Family medicine
• Oncology
• Intensive care medicine
• Emergency medicine

Selected publications

• Pomalidomide for hereditary hemorrhagic telangiectasia: after trial longitudinal assessment study (PATH-HHT ATLAS)

  Blood Advances · 2026-01-09 · 1 citations

  articleOpen access

  ABSTRACT: Hereditary hemorrhagic telangiectasia (HHT) causes severe recurrent epistaxis, chronic gastrointestinal bleeding, solid organ arteriovenous malformations, and significant anemia. Although it is the second most common inherited bleeding disorder worldwide, no approved therapies exist. The multicenter, US randomized, controlled PATH-HHT trial demonstrated efficacy of short-term pomalidomide treatment of epistaxis in HHT. However, questions regarding long-term safety, effectiveness, and utility for HHT-associated gastrointestinal bleeding remain. The PATH-HHT ATLAS (after trial longitudinal assessment study) was a multicenter US longitudinal observational study evaluating patients enrolled in PATH-HHT who continued pomalidomide after PATH-HHT through a poststudy drug access program, with ongoing close monitoring per PATH-HHT protocol and as mandated by the US Food and Drug Administration. Sixty-two patients treated with pomalidomide for HHT for up to 4.4 years were included. Significant, durable improvements in mean epistaxis severity score (5.55 points [baseline] to 2.80 points [month 12]; P< .0001) and mean hematologic support score (9.11 red cell unit equivalents [RUEs] during 6 months pretreatment to 5.73 RUEs [months 7-12]; P = .0056) were observed. Pomalidomide was less effective for gastrointestinal bleeding than for epistaxis, particularly in patients with high red blood cell transfusion requirements. Thirty-one patients (50%) underwent dose reduction from 4 mg daily, primarily due to neutropenia, but most maintained effectiveness at 2 or 3 mg daily. Over 105.2 patient-years of pomalidomide treatment, 0 patients developed peripheral neuropathy, 1 developed venous thromboembolism, and 5 developed serious infections. In conclusion, pomalidomide was effective for HHT-associated epistaxis over extended durations, albeit with nontrivial potential toxicities, and may be considered for certain patients with HHT. This trial was registered at www.clinicaltrials.gov as NCT07018401.

  PublisherOA PDFDOI

• Characteristics associated with clinical response to pomalidomide in hereditary hemorrhagic telangiectasia

  Blood Advances · 2026-02-20

  articleOpen access

  ABSTRACT: Hereditary hemorrhagic telangiectasia (HHT) results in recurrent severe epistaxis, chronic gastrointestinal bleeding, and other morbid manifestations. Pomalidomide was recently demonstrated to be efficacious for bleeding in HHT in the landmark PATH-HHT trial. Given that the vascular remodeling that results in bleeding improvement may require several months, characteristics associated with treatment response would be invaluable to plan treatment. Using data from PATH-HHT (NCT03910244), we identified characteristics associated with change from baseline in Epistaxis Severity Score (ESS; a well-validated 10-point HHT bleeding scale and the trial's primary end point) using multivariable-adjusted repeated-measures models. Characteristics associated with a better response to pomalidomide at weeks 16 to 24 of the trial included a higher (more severe) baseline ESS (change from baseline, -0.76 [95% confidence interval (CI), -0.99 to -0.54] points per 1-point higher baseline score, P< .001) and concomitant antifibrinolytic therapy (-0.73 [95% CI, -1.48 to 0.01] vs no therapy, P = .05). Characteristics associated with a less robust response were older age (0.29 [95% CI, 0.05-0.53] per 10 years older, P = .02) and underlying ACVRL1 germ line mutation (0.62 [95% CI, -0.04 to 1.27] vs ENG, SMAD4, or mutation not known, P = .06]. Characteristics notably associated with placebo response (placebo effect) were patient-reported outcome measures, such that those experiencing more severe disease at baseline had less placebo effect. In conclusion, several baseline patient and disease characteristics were associated with pomalidomide response, findings that may help guide patient selection for the use of pomalidomide in HHT. This trial was registered at www.Clinicaltrials.gov as NCT03910244.

  PublisherOA PDFDOI

• Pomalidomide for Hereditary Hemorrhagic Telangiectasia: After Trial Longitudinal Assessment Study (PATH-HHT ATLAS).

  UNC Libraries · 2026-03-21

  articleOpen access

  Hereditary hemorrhagic telangiectasia (HHT) causes severe recurrent epistaxis, chronic gastrointestinal bleeding, solid organ arteriovenous malformations, and a profound anemia burden. It is the second most common inherited bleeding disorder worldwide yet remains without approved therapies. The multicenter U.S. randomized, controlled PATH-HHT trial demonstrated efficacy of short-term pomalidomide treatment of epistaxis in HHT. However, many critical questions regarding long-term safety, effectiveness, and utility for HHT-associated gastrointestinal bleeding remain. The PATH-HHT After Trial Longitudinal Assessment Study (NCT07018401) was a multicenter U.S. longitudinal observational study evaluating patients enrolled in PATH-HHT who continued pomalidomide post-PATH-HHT through a post-study drug access program, with ongoing close monitoring per PATH-HHT protocol and as mandated by FDA. Sixty-two patients treated with pomalidomide for HHT for up to 4.4 years were included. Significant, durable improvements in mean Epistaxis Severity Score (5.55 points [baseline] to 2.80 points [month 12], p&lt;0.0001) and mean Hematologic Support Score (9.11 red-cell unit equivalents [RUEs] during 6 months pretreatment to 5.73 RUEs [months 7-12], p=0.0056) were observed. Pomalidomide was less effective for gastrointestinal bleeding than epistaxis, particularly in patients with high RBC transfusion requirements. Thirty-one patients (50%) dose-reduced from 4-mg daily, primarily due to neutropenia, but most maintained effectiveness at 2- or 3-mg daily. Over 105.2 patient-years of pomalidomide treatment, 0 patients developed peripheral neuropathy, 1 developed venous thromboembolism, and 5 developed serious infections. In conclusion, pomalidomide was highly and durably effective for HHT-associated epistaxis, albeit with nontrivial potential toxicities, and may be considered a routine long-term antiangiogenic therapeutic option for certain patients with HHT. NCT07018401.

  PublisherDOI

• Correction: Thrombotic thrombocytopenic purpura: early diagnosis and effective treatment in 2025

  Intensive Care Medicine · 2025-07-22

  erratumOpen access
  PublisherOA PDFDOI

• Validation and clinical application of the hereditary hemorrhagic telangiectasia-specific quality of life scale

  Journal of Thrombosis and Haemostasis · 2025-10-15 · 1 citations

  articleOpen access

  BACKGROUND: Hereditary hemorrhagic telangiectasia (HHT) is the second most common inherited bleeding disorder worldwide and results in substantial medical and psychosocial morbidity due to recurrent, often severe epistaxis, chronic gastrointestinal bleeding, hematologic support-dependent iron deficiency anemia, and serious bleeding and nonbleeding complications of visceral arteriovenous malformations. With disease-modifying therapeutics entering clinical trials, disease-specific health-related quality of life (HRQoL) instruments capable of longitudinal assessment of HHT-specific manifestations are essential. OBJECTIVES: To externally validate and define the features relevant to clinical application of the HHT-specific Quality of Life scale. METHODS: The HHT-specific Quality of Life (HHT-QoL) scale was developed as an easily administered, 4-item instrument assessing physical, social, personal, and work impacts due to HHT longitudinally. Utilizing comprehensive, longitudinal data from pomalidomide for the treatment of HHT, the largest HHT randomized clinical trial to date, we evaluated internal consistency and external validity of the HHT-QoL and defined essential characteristics for clinical application. RESULTS: The HHT-QoL demonstrated good internal consistency and external validity, including directionally and clinically consistent correlations with the Epistaxis Severity Score, bleeding duration as assessed by epistaxis diary, and multiple well-validated generic HRQoL instruments measuring emotional distress, social roles and activities, and fatigue. Development of an HHT HRQoL severity classification utilizing HHT-QoL total scores was successful (0 as no limitation, 1-3 as mild, 4-7 as moderate, and 8-16 points as severe limitation), and the minimal clinically important difference was calculated to be 1.1 points (best rounded to 1 point as the scores are integers) utilizing anchor-based and distribution-based methods. CONCLUSION: This study successfully externally validates the HHT-QoL. The HHT-QoL is a validated disease-specific HRQoL instrument with defined severity classification and minimal clinically important difference, making it ideal for use in clinical trials of bleeding in HHT.

  PublisherDOI

• Evaluation of a screening protocol for telomere biology disorders in individuals with interstitial lung disease undergoing lung transplant evaluation

  ERJ Open Research · 2025-02-20 · 2 citations

  articleOpen access

  Background: Because of the potential for extrapulmonary disease, it is important for lung transplant programmes to identify telomere biology disorder (TBD)-related interstitial lung disease (ILD). The aim of this study was to evaluate a TBD phenotype screen among ILD patients undergoing lung transplant evaluation, including the sensitivity and specificity of individual phenotype screening questions and the characteristics of patients with TBD identified outside of the screening protocol. Methods: This was a retrospective cohort study of adults with ILD who underwent lung transplant evaluation from 1 January 2018 to 28 February 2023. The TBD phenotype screen included early greying, family history of ILD and unexplained liver disease, cytopenias or macrocytosis. TBD screen-positive patients underwent telomere length (TL) testing. Results: Among 383 patients evaluated, 92 (24.0%) had a positive phenotype screen. 58 (63.0%) had early greying, 39 (42.4%) had a first-degree relative with ILD and 29 (31.5%) had unexplained macrocytosis or cytopenias. Using granulocyte and lymphocyte TL <10th percentile, 51 (55.4%) patients met criteria for a TBD. Early greying had the highest sensitivity for TBD (72.5%) with specificity increasing with the number of positive screening questions. Among the 23 patients who underwent TL testing outside of the screening protocol, most commonly because of an early age of onset of ILD, eight (34.8%) had TL between the 1st and 10th percentile. Conclusions: Lymphocyte and granulocyte TL <10th is common among TBD phenotype screen-positive ILD patients undergoing transplant evaluation. Inclusion of additional screening questions related to age of onset of ILD could improve the sensitivity of the protocol for short TL.

  PublisherDOI

• Risk factors for silent cerebral infarction in immune‐mediated thrombotic thrombocytopenic survivors

  British Journal of Haematology · 2025-12-08

  articleOpen access

  Immune-mediated thrombotic thrombocytopenic purpura (iTTP) survivors are predisposed to silent cerebral infarctions (SCI) defined as radiological evidence of brain ischaemia without focal symptoms. This study examined risk factors associated with SCI burden in iTTP survivors during remission. We included the first 39 iTTP survivors enrolled in the prospective neurological sequelae of TTP (NeST) study. Participants underwent brain magnetic resonance imaging during clinical remission. SCI burden was quantified using the modified age-related white matter changes (ARWMC) score. Multivariate linear regression models identified the predictors of SCI burden. Of 39 participants, 20 (51.3%) had SCI. On univariate analysis, higher SCI burden was associated with older age, elevated level of peak serum creatinine and peak lactate dehydrogenase (LDH) during the index iTTP episode, comorbidities including diabetes mellitus, prior stroke, coronary artery disease (CAD) and family history of cerebrovascular disease. In the multivariate model, higher SCI burden was significantly associated with older age (p < 0.001), diabetes mellitus (p = 0.007), prior stroke (p = 0.01), CAD (p = 0.02) and elevated peak LDH (p = 0.046). Total days of thrombocytopenia (surrogate for 'days with active iTTP') were not associated with SCI burden. Both modifiable and non-modifiable factors contribute to SCI in iTTP survivors. Targeted management of cardiovascular comorbidities during remission may reduce long-term neurological sequelae. Validation in larger cohorts is needed.

  PublisherOA PDFDOI

• Immune Thrombotic Thrombocytopenic Purpura

  JAMA · 2025-05-19 · 17 citations

  article1st authorCorresponding

  Importance: Immune thrombotic thrombocytopenic purpura (iTTP) is a life-threatening thrombotic microangiopathy that presents with microangiopathic hemolytic anemia (MAHA) and thrombocytopenia. Worldwide annual incidence of iTTP is 2 cases per million to 6 cases per million. Observations: Immune TTP is caused by an autoantibody to a disintegrin and metallopeptidase with thrombospondin type 1 motif 13 (ADAMTS13), an enzyme that cleaves von Willebrand factor (vWF). With severely low ADAMTS13 activity (<10%), large multimers of vWF accumulate and bind platelets, forming microvasculature thromboses that cause ischemic organ injury (eg, myocardial infarction and stroke). The incidence of iTTP is higher in adults than children (incident rate ratio [IRR], 31.62 per million person-years [95% CI, 14.68-68.10]), females than males (IRR, 3.19 [95% CI, 2.65-3.85]), and Black compared with non-Black individuals (IRR, 7.09 [95% CI, 6.05-8.31]). Common presenting symptoms are neurologic (eg, headache, confusion, or seizures [39%-80%]) and abdominal pain (35%-39%). For patients presenting with MAHA and thrombocytopenia, clinical prediction scores for iTTP using laboratory data, such as platelet count less than 30 × 109/L and creatinine level less than 2.0 mg/dL (176.8 μmol/L), can help guide empirical treatment initiation for iTTP before ADAMTS13 results are available. Prompt initiation of therapy with therapeutic plasma exchange, corticosteroids, and rituximab improves survival with iTTP from almost zero to approximately 93%. Caplacizumab, a synthetic small antibody (nanobody) that blocks platelet binding to vWF, administered concurrently with immunosuppression and therapeutic plasma exchange and continued until ADAMTS13 recovery, reduces the time to normalization of platelet count and decreases the risk of early recurrence (defined as within 30 days of completing therapeutic plasma exchange) compared with placebo (risk difference [RD], -29% [95% CI, -42 to -14%]) but increases bleeding risk (RD, 17% [95% CI, 4%-30%]). After obtaining clinical remission (defined as at least 30 days of sustained normalization of platelet count, decreased serum lactate dehydrogenase level, and absence of new or progressive ischemic organ injury without therapeutic plasma exchange or caplacizumab), 16% of patients have at least 1 relapse of iTTP. Regular monitoring of ADAMTS13 activity in remission and administration of rituximab when ADAMTS13 activity is less than 20% reduces risk of relapse (odds ratio, 0.09 [95% CI, 0.04-0.24]). Conclusions and Relevance: Immune TTP is a rare immune-mediated disorder that presents with thrombocytopenia and MAHA and may cause life-threatening thrombosis. Treatment with therapeutic plasma exchange, corticosteroids, and rituximab is associated with 30-day survival rates of more than 90%. Addition of caplacizumab shortens time to normalization of platelet count and reduces recurrences while receiving the drug but increases bleeding risk. Monitoring ADAMTS13 activity in survivors and initiation of rituximab for those with low ADAMTS13 activity reduces the risk of clinical relapse.

  PublisherDOI

• Critical bleeding in adults and children with immune thrombocytopenia: a multicenter cohort study

  Blood Advances · 2025-04-09 · 3 citations

  articleOpen access

  ABSTRACT: Critical bleeding in patients with immune thrombocytopenia (ITP) is a life-threatening hematologic emergency. This study aimed to describe the frequency, management, and outcomes of critical bleeds among adults and children with ITP. We conducted a retrospective cohort study of patients with ITP who presented to the emergency room with a platelet count <20 × 109/L across 7 centers in the United States and Canada between 2010 and 2019. Of 1226 patients (n = 296 adults; n = 930 children), 28 (2.3%) had critical bleeds (adults, n = 15 [median age, 68 years]; children, n = 13 [median age, 11 years]). Of patients with critical bleeds, 12 adults (80.0%) and 6 children (46.2%) had intracranial hemorrhage (ICH). For adults, the common interventions used to treat critical bleeds were platelet transfusions (n = 11 [73.3%]), corticosteroids (n = 10 [66.7%]), and IV immunoglobulin (n = 8 [53.3%]), and for children, common interventions were IV immunoglobulin (n = 10 [76.9%]), corticosteroids (n = 8 [61.5%]), platelet transfusions (n = 8 [61.5%]), thrombopoietin receptor agonists (n = 4 [30.8%]), and antifibrinolytic agents (n = 4 [30.8%]). For both adults and children, the most common treatment combination was corticosteroids, IV immunoglobulin, and platelet transfusion (n = 6 [40.0%] vs n = 6 [46.2%]). The median time from presentation to first treatment was 6.9 hours for adults and 3.5 hours for children. Overall, 9 patients (32.1%) with critical ITP bleeds died, including 7 adults (46.7%) and 2 children (15.4%). Critical bleeding in patients with ITP was rare but frequently fatal, especially among older adults with ICH and when treatments were delayed.

  PublisherOA PDFDOI

• HTRS2025.P2.90 Evaluation of Patients with Discordant Lupus Anticoagulant testing: A Single Center Retrospective Analysis

  Research and Practice in Thrombosis and Haemostasis · 2025-11-01

  articleOpen access
  PublisherDOI

Frequent coauthors

• Margaret V. Ragni

  University of Pittsburgh

  103 shared

• Margareth C. Ozelo

  Universidade Estadual de Campinas (UNICAMP)

  100 shared

• Roland W. Herzog

  Indiana University – Purdue University Indianapolis

  100 shared

• David Lillicrap

  Queen's University

  100 shared

• Nigel S. Key

  University of North Carolina at Chapel Hill

  100 shared

• Benjamin J. Samelson‐Jones

  Children's Hospital of Philadelphia

  100 shared

• Leonard A. Valentino

  Rush University Medical Center

  100 shared

• Adam Cuker

  University of Pennsylvania

  23 shared

Labs

• Allyson Marie Pishko LabPI

Education

• B.S.

  University of Notre Dame

  2008

• M.D., Medicine

  University of Pittsburgh School of Medicine

  2012

• Other, Clinical Epidemiology

  University of Pennsylvania

  2018