Jessie Lee Ann Alexander

All Publications

• Can we eliminate TBI/Busulfan and GVHD in Fanconi anemia transplantation? Results from a prospective haploidentical transplant approach Bertaina, A., Czechowicz, A., Hiroshima, L., Kunte, N., Klinger, E., Saini, G., John, T., Alexander, J., Shyr, D., Shah, A., Klein, O., Boyel, J., Boelens, J., Wlodarsky, M., Shizuru, J., Arreola, V., Parkman, R., Weinberg, K., Porteus, M., Agarwal, R. SPRINGERNATURE. 2026: 122-123

  View details for Web of Science ID 001825215301095

• Phase 1 gene therapy for patients with IPEX syndrome: preliminary in vivo data of the treatment with engineered Treg-like cell CD4<SUP>LVFOXP3</SUP> Alexander, J. L., Agarwal, R., Namjoshi, S., Ramachandran, A., Wang, B., Borna, S., Shah, S., Schaefer, K., Alkayed, A., Kuo, Y., Wani, P., Faseli, F., Kandar, K., Dougall, K., Goodwin, E., Saini, G., Cooper, M., Bucciol, G., Meyts, I., Mendonca, L., Kalil, J., Chong, H., Dunn, C., Riviere, J., Kristal, E., Shendi, H., Roncarolo, M., Bacchetta, R. SPRINGERNATURE. 2026: 48-49

  View details for Web of Science ID 001825215301028

• Autologous stem cell transplantation (ASCT) for Refractory juvenile-onset systemic sclerosis (JSSc). Annals of the rheumatic diseases Torok, K. S., Horvei, P., Li, J., Rosser, F., Alexander, J. L., Rose-Felker, K., Sood, V., Olson, A., Hogue, N., Vandergrift, V., Stanczak, H., Miglioretti, M., Farver, L., Koster, W., Mcguire, D., Havrilla, H., McIntyre, S., Domsic, R. T., Lafyatis, R. A., Szabolcs, P. 2026

  Abstract

  OBJECTIVES: Juvenile-onset systemic sclerosis (JSSc) is a rare autoimmune disease characterised by vasculopathy and multiorgan fibrosis, leading to significant morbidity and early mortality. Autologous stem cell transplantation (ASCT) is an established therapy for severe adult systemic sclerosis, but data in JSSc are limited. We reported outcomes of 8 consecutive patients with treatment-refractory JSSc who received ASCT at a single centre.METHODS: Patients with moderate-to-severe JSSc refractory to ≥ 2 disease-modifying antirheumatic drugs underwent CD34+-selected ASCT using institutional review board- and US Food and Drug Administration-approved protocols (ClinicalTrials.gov: NCT03630211). Conditioning included alemtuzumab, rituximab, antithymocyte globulin, total body irradiation, and thiotepa. Clinical outcomes were assessed at baseline and every 6 months up to 48 months post-ASCT.RESULTS: All patients engrafted successfully (median neutrophil and platelet engraftment: 13 and 22 days, respectively). There were no treatment-related deaths or organ toxicities. Event-free survival (no death, cardiopulmonary failure, or renal crisis) was 100% at a median follow-up of 30 months. Skin scores (modified Rodnan skin score) improved by a median of 93%, and 6-minute walk distance improved by 13%. All patients discontinued immunosuppression for ≥18 months post-ASCT; 1 patient later required reinitiation for disease flare. Pulmonary function stabilised; both patients with pulmonary hypertension showed normalised pulmonary pressures. Gastrointestinal symptoms, including oesophageal motility and reflux, improved in most patients. Musculoskeletal function (Childhood Health Assessment Questionnaire and Childhood Myositis Assessment Scale), vascular symptoms, and quality of life (Scleroderma Health Assessment Questionnaire and performance scores) improved across the cohort.CONCLUSIONS: ASCT was safe and well-tolerated in patients with refractory JSSc and resulted in durable, multisystem improvement. These findings support earlier consideration of ASCT for patients with JSSc with progressive, treatment-resistant disease.

  View details for DOI 10.1016/j.ard.2026.05.022

  View details for PubMedID 42309868

• Refractory infantile IPEX with Treg-restricted FOXP3null expression caused by a novel variant in FOXP3. Journal of human immunity Rayes, A., Ramachandran, A., Pulsipher, M. A., Dolan, J. G., Cho, S., Johnson, A. K., Alexander, J. L., Borna, S., Bacchetta, R. 2026; 2 (3): e20250249

  Abstract

  Immune dysregulation, polyendocrinopathy, enteropathy, X-linked (IPEX) syndrome is a life-threatening monogenic inborn error of immunity caused by pathogenic variants in the FOXP3 gene. In this report, we describe the clinical and immunologic consequences of a novel FOXP3 variant in an infant with refractory IPEX syndrome. Rapid whole-genome sequencing revealed a missense variant c.1050C>G (p.Ile350Met) in the forkhead domain of FOXP3. FOXP3 expression was abrogated in the regulatory T cell (Treg) subset but was maintained at low levels in activated effector T cells. The frequency of Treg-specific demethylated region (TSDR)-demethylated T cells was within normal range at birth, but it increased to pathologically high levels at 2 wk of age, prior to manifestation of disease symptoms. These results support the clinical relevance of the TSDR-demethylated cell percentage in evaluation of disease activity in IPEX. This case highlights a severe form of IPEX syndrome refractory to multiple immunosuppressive agents and the importance of early immunological studies to verify the clinical significance of novel genetic findings.

  View details for DOI 10.70962/jhi.20250249

  View details for PubMedID 41909206

  View details for PubMedCentralID PMC13023379

• Autologous Stem Cell Transplantation for Refractory Juvenile-Onset Systemic Sclerosis Horvei, P., Li, J., Rosser, F., Rose-Felker, K., Alexander, J. L., Hogue, N., Mcintyre, S., Koshy, L., Olson, A., Torok, K., Szabolcs, P. ELSEVIER SCIENCE INC. 2026: S39-S40

  View details for Web of Science ID 001697336900042

• Model-Based ATG Dosing Optimization in Ab Haplo-HSCT: Improved Pre-HSCT Exposure Consistency and Viral Infection Analysis Shyr, D. C., Hiroshima, L., John, T. D., Barbarito, G., Agarwal, R., Alexander, J. L., Shah, A. J., Li, T., Chang, I., Arreola, V., Boelens, J., Bertaina, A. ELSEVIER SCIENCE INC. 2026

  View details for Web of Science ID 001708751400003

• Hematopoietic Cell Transplantation for Wiskott-Aldrich syndrome: A PIDTC Report. Blood advances Alexander, J. L., Dávila Saldaña, B. J., Brazauskas, R., Dammalapati, S. G., Griffith, L. M., Shah, A. J., Shimano, K. A., Ochs, H. D., Bleesing, J., Ebens, C. L., Kapadia, M., Bauchat, A., Kapoor, N., Oved, J. H., Eissa, H., Lust, H., Keller, M. D., Haines, H., Chandrakasan, S., Talano, J. M., Rayes, A., Madden, L. M., Shereck, E. B., Miller, H. K., Forbes Satter, L. R., Martinez, C. A., Rozmus, J., Bednarski, J. J., Yu, L. C., Chellapandian, D., Aquino, V. M., Knutsen, A. P., Chong, H., Chopek, A., Gillio, A. P., Joshi, A., Rangarajan, H. G., Moore, T. B., Andolina, J. R., DeSantes, K., Vander Lugt, M. T., Prockop, S. E., Shyr, D., Sullivan, K. E., Parikh, S. H., Weinacht, K. G., Torgerson, T. R., Marsh, R. A., Dvorak, C. C., Chan, A. Y., Haddad, E., Heimall, J., Pulsipher, M. A., Leiding, J. W., Kohn, D. B., Puck, J. M., Notarangelo, L. D., Rawlings, D. J., Cowan, M. J., Petrovic, A., Pai, S. Y., Burroughs, L. M. 2025

  Abstract

  Wiskott-Aldrich syndrome (WAS), an X-linked disorder characterized by immunodeficiency, thrombocytopenia, autoimmunity, and malignancy, can be effectively treated with allogeneic hematopoietic cell transplantation (HCT). Older age at HCT and mismatched donors are known to impact overall survival (OS). The influence of specific clinical manifestations or WAS variant class on OS and factors associated with event-free survival (EFS) remain incompletely defined. We analyzed outcomes of 308 patients with WAS who underwent HCT at 37 institutions of the Primary Immune Deficiency Treatment Consortium (PIDTC) from 1990-2018. With a median follow-up of 5.3 years, the 5-year OS and EFS were 87.2% and 79.7%, respectively. Age ≥5 years, donor type, and a pre-HCT history of severe infection had a negative impact on OS and EFS, whereas pre-HCT autoimmunity had no impact. Reduced intensity regimens were associated with lower T cell and myeloid donor chimerism, particularly when non-busulfan-based regimens were used. Low myeloid donor chimerism was associated with lower platelet counts. Mixed chimerism was not consistently associated with post-HCT autoimmunity. Patients with class I (exon 1-2 missense and intron 5 hotspot variants) and class II variants (all others) had similar pre-HCT clinical symptom severity and no difference in OS, EFS or platelet recovery post-HCT. In conclusion, our study showed excellent long-term OS and EFS following HCT for WAS, highlighting the importance of early HCT, before the development of severe infections. We confirmed that HCT using busulfan-based conditioning was associated with improved donor chimerism and platelet recovery. This study was registered at www.clinicaltrials.gov as #NCT02064933.

  View details for DOI 10.1182/bloodadvances.2025017662

  View details for PubMedID 41346295

• Outcomes Following Matched Sibling Donor Transplantation for Severe Combined Immunodeficiency: A Report from the PIDTC. Blood advances Rayes, A., Logan, B. R., Liu, X., Dara, J., Buckley, R. H., Oved, J. H., Kapoor, N., Kapadia, M., Chandra, S., Martinez, C. A., Bunin, N., Chandrakasan, S., Chen, K., Bednarski, J. J., Haines, H. L., Eissa, H., Talano, J. M., Keller, M. D., Ebens, C. L., Chaudhury, S., Shereck, E. B., Aquino, V. M., Knutsen, A. P., Alexander, J. L., Gillio, A. P., Chellapandian, D., Shah, A. J., Miller, H. K., Vander Lugt, M. T., Seroogy, C. M., Dorsey, M. J., Mousallem, T., Parrott, R. E., O'Reilly, R. J., Aguayo-Hiraldo, P. I., Prockop, S. E., Dávila Saldaña, B. J., Thakar, M. S., Burroughs, L. M., Torgerson, T. R., Leiding, J. W., Marsh, R. A., Griffith, L. M., Pulsipher, M. A., Kohn, D. B., Notarangelo, L. D., Cowan, M. J., Puck, J. M., Cuvelier, G. D., Heimall, J., Haddad, E., Pai, S. Y., Dvorak, C. C. 2025

  Abstract

  The Primary Immune Deficiency Treatment Consortium (PIDTC) performed a retrospective analysis of 133 patients with severe combined immunodeficiency (SCID) receiving matched sibling donor (MSD) hematopoietic cell transplantation (HCT) between 1980 and 2023 at 30 North American institutions. In this largest cohort of MSD outcomes in SCID patients to date, we examined the impact of conditioning regimen and graft-versus-host disease (GVHD) prophylaxis on survival and immune recovery. Outcomes after MSD HCT for SCID were excellent. Patients without an active infection or failure to thrive (FTT) at the time of HCT had 5-year overall survival (OS) superior to those with infection or FTT. Acute and chronic GVHD outcomes were independent of GVHD prophylaxis, conditioning regimen, SCID type, or presence of maternal engraftment. Patients without active infection at the time of HCT had superior chronic GVHD free event-free survival vs. those with infection. T cell reconstitution at 6 months was less likely achieved with use of GVHD prophylaxis or serotherapy, and in leaky SCID or Omenn syndrome patients. At 6 months, 1 year, and 2-5 years T cell reconstitution was less likely with ADA, DCLRE1C or RAG genotype. B cell reconstitution at 1 year and 2-5 years was negatively impacted by development of grade II-IV or III-IV acute GVHD. Conditioning did not impact T or B cell reconstitution. Our data suggest omitting conditioning and GVHD prophylaxis for patients with typical SCID did not negatively impact 5-year outcomes following MSD HCT, but the data are insufficient to recommend this approach for best long-term outcomes. (ClinicalTrials.gov NCT01186913).

  View details for DOI 10.1182/bloodadvances.2025016812

  View details for PubMedID 41289158

• Marrow-ly we learn along: A needs assessment of the educational needs of pediatric and young adult patients with bone marrow failure Bowman, S., de Jong, J., Alexander, J., Ilonze, C., Rose, M. ELSEVIER. 2025: 2861-2862

  View details for DOI 10.1182/blood-2025-2861

  View details for Web of Science ID 001659007200010

• Improvement Across Multi-organ Domains and Patient Reported Outcomes in Refractory Juvenile-Onset Systemic Sclerosis (jSSc) up to 4 Years After Autologous Stem Cell Transplantation (ASCT) Li, J., Horvei, P., Rosser, F., Rose-Felker, K., Sood, V., Olson, A., Vandergrift, V., Hogue, N., Farver, L., Mcguire, D., Havrilla, H., Alexander, J., McIntyre, S., Szabolcs, P., Torok, K. WILEY. 2024: 4985-4988

  View details for Web of Science ID 001331419107227

• Improvement in Clinical and Patient-Reported Outcomes for Refractory Juvenile-Onset Systemic Sclerosis (jSSc) 6 Months to 2 Years After Autologous Stem Cell Transplantation (ASCT) Torok, K., Horvei, P., Rosser, F., Rose-Felker, K., Sood, V., Olsen, A., Hogue, N., Vandergrift, V., Farver, L., Mcguire, D., Li, J., Havrilla, H., Alexander, J., McIntyre, S., Szabolcs, P. WILEY. 2023: 5204-5206

  View details for Web of Science ID 001190014305212