About

Michael Allen Pulsipher, MD, is the Division Chief of Pediatric Hematology at Intermountain Primary Children’s Hospital and Oncology, the Director of the Children’s and Adolescent Cancer Initiative at Huntsman Cancer Institute, and holds a Presidential Chair in Pediatric Oncology and Hematology at the University of Utah. His research interests include allogeneic transplantation and cell therapy for acute leukemias, especially ALL, where he is running national research trials aimed at testing haploidentical approaches and assessing the role of NGS-MRD in identifying patients able to undergo less intensive BMT approaches. Dr. Pulsipher’s work in cell therapy contributed to the FDA approval of the first CART cell treatment, tisagenlecleucel, and he continues to run trials in CAR T, NK, and Viral Specific T-cell therapies. He has also done extensive work in reduced toxicity approaches to transplantation for both malignant and non-malignant disorders, and has led national protocols for patients with immunodeficiencies, HLH, and bone marrow failure. Additionally, he has conducted large trials assessing the safety and quality of life of pediatric bone marrow donors and is currently leading a national trial on the psychological well-being of donor and recipient families during the transplant process.

Research topics

• Medicine
• Internal medicine
• Oncology
• Immunology
• Pediatrics
• Gastroenterology

Selected publications

• Factors Associated With Pre-donation Health-related Quality-of-Life Among Pediatric Sibling Hematopoietic Cell Donors: A DonorKids QL Study

  Transplantation and Cellular Therapy · 2026-04-01

  articleOpen accessSenior author

  Due to limited published data assessing pediatric hematopoietic cell donor experiences, we previously conducted one of the largest quantitative investigations of pediatric donor experiences and health-related quality-of-life (HRQoL) at the time (RDSafe). Findings from RDSafe demonstrated that a subset of pediatric HC donors experienced very poor HRQoL; unfortunately, that dataset addressed only a limited number of factors, and key associations explaining this poor HRQoL were not found. In this study, our goal was to address that deficit by describing pre-donation donor HRQoL in detail and identifying factors across five key domains that were associated with donor HRQoL. We conducted a prospective study involving 29 centers in the US (31 enrolled, 29 contributed data). After consent at the local center, data were collected via telephone interviews with donors, recipients, other siblings, and parents, and via a web-based survey from transplant centers. Family data (donors, recipients, sibling, parents) were collected before donation and at four weeks, six months, and one-year post-donation. Domains assessed included sociodemographics, general and donation-related psychosocial, clinical, and transplant center characteristics. Data presented here are from the 133 donor-parent pairs who completed the pre-donation interview, 64 recipients, 59 non-donor/non-recipient siblings in these related donor families, and 78 comparison siblings of patients receiving unrelated marrow, PBSC, or cord blood transplantation. Important percentages of pediatric donors reported very poor psychosocial (20%) and overall (13%) HRQoL and parents overestimated their donor child's HRQoL. Donors had significantly better HRQoL than recipients and worse HRQoL than parent proxy reports. Multiple donor, recipient, parent and transplant center characteristics were associated with HRQoL in bivariate analyses. Multivariable analyses by donor age group suggested that donor HRQoL was significantly negatively associated with donor self-reported anxiety, depression and parental education and significantly positively associated with family cohesion, understanding of donation and proxy reports of donor HRQoL. The link between donor HRQoL and recipient and parent health and well-being suggests that variations in donor HRQoL do not occur in isolation but affect and/or are affected by the overall functioning and well-being of other family members and the family as a whole. Particularly striking is the association of donor HRQoL with perceived understanding of the donation process-improved donor education is a potential pathway to improving donor HRQoL. Assessment of-and Interventions to mitigate-risks to donors should involve the functioning of the family as a whole and not just the donor.

  PublisherDOI

• Evaluation of in vivo CAR transgene levels in tisagenlecleucel-treated patients with relapsed/refractory B-ALL and DLBCL

  Blood Advances · 2025-06-24 · 1 citations

  articleOpen access

  ABSTRACT: Tisagenlecleucel is a CD19-directed autologous chimeric antigen receptor (CAR) T-cell therapy. Quantitative polymerase chain reaction assays are highly sensitive in defining in vivo kinetics by measuring CAR transgene in peripheral blood. This study aimed to identify clinically meaningful CAR T-cell blood levels that correlated with response/relapse. In pediatric/young adult patients with relapsed/refractory (R/R) B-cell acute lymphoblastic leukemia (B-ALL), maximum CAR T-cell blood levels were higher in patients with ongoing complete remission and in patients with CD19- relapse relative to those with CD19+ relapse. In adult patients with R/R diffuse large B-cell lymphoma (DLBCL), no apparent association between in vivo kinetics and response was noted, with a wide range of transgene levels at relapse. In B-ALL, patients with B-cell aplasia sustained >6 months had higher CAR T-cell expansion relative to those with early B-cell recovery (BCR) (<6 months after infusion); however, a definitive cutoff for BCR-associated expansion level could not be identified. In most patients with B-ALL, BCR >6 months maintained favorable responses. However, early BCR could not be confirmed as a potential indicator of relapse due to high censoring from transplant, following presumed risk of relapse. However, allografting in these patients may potentially mitigate the poor prognosis related to early BCR. In DLBCL, BCR was not associated with relapse. These findings suggest that blood CAR transgene levels may be associated with long-term responses; however, they lack robust predictive potential for relapses. As reported earlier, next-generation sequencing for minimal residual disease appears to be a reliable biomarker predictive of relapse for B-ALL.

  PublisherOA PDFDOI

• Highly efficient collection and manufacture of autologous HSC gene therapy cell product for patients with sickle cell disease using a lentiviral vector containing a shmir targeting BCL11a

  Blood · 2025-11-03 · 1 citations

  article

  Abstract Achieving an adequate dose of genetically modified hematopoietic stem cells (HSCs) for gene therapy in patients with sickle cell disease (SCD) remains a challenge due to limitations related to stem cell mobilization using plerixafor alone, reduced apheresis collection efficiency, and losses during ex vivo cell manipulation. To date, 39 SCD patients were enrolled in NHLBI-funded Phase I (NCT03282656; PMC7962145) and NHLBI/CIRM-funded multi-site Phase II (NCT05353647) gene therapy trials to receive autologous CD34+ HSCs transduced with a lentiviral vector encoding a short hairpin RNA embedded in a microRNA (shmiR) targeting BCL11A. To collect HSCs as the starting material for drug product manufacture (minimum 4×10⁶ cells/kg), apheresis (minimum 4 blood volumes or up to 8 hours) was performed within 3 hours of daily plerixafor for 2 consecutive days. A third day of collection was used to generate a back-up product as needed. Mean mobilized, pre-apheresis peripheral blood (PB) CD34+ count was 40 cells/μL (range 7–126). Among 35 evaluable patients (4 excluded: 1 pending release, 3 withdrew), 74% (26/35) reached target CD34+ collection in a single mobilization cycle (mean 2.6 procedures, range 2–6). Manufacturing was achieved with a mean of 11.92×10⁶ CD34+ cells/kg collected resulting in a drug product of 7.04×10⁶ CD34+ cells/kg and a net cell recovery from apheresis to drug product of 62% (range 38–91%). Drug products were successfully generated for all patients with a mean product vector copy number of 4.04 copies/cell (range 1-7). The time interval from first collection cycle to completion of product testing was a median of 39 days (mean 56) for all patients, and a median of 37 days (mean 38) for those collected in 1 cycle. Preparative transfusions were given before collections to bridge patients after stopping hydroxyurea or to mitigate stress erythropoiesis. We examined the impact of preparative transfusion on mobilization and apheresis efficiency in 16 evaluable patients at a single site. All underwent ≥ 3 months of preparative transfusion with a HbS target of ≤30% before mobilization using simple transfusion or automated red cell exchange. The mean HbS% at mobilization was 10.8% (range 2.9–21.5%). Overall, the preparative transfusion regimen reduced PB reticulocyte percentage by 50% to a mean of 7.2% (range 3.7–12.3), indicating decreased stress erythropoiesis. Resting PB CD34+ counts before and after 3-months of transfusion were 10.8 cells/μL (range 2–21) and 6.2 cells/μL (range 1–13), respectively. A total of 39 mobilization/collection procedures were performed in 16 patients with a mean post-plerixafor CD34+ count of 43 cells/μL (range 13–85) with collections performed between the two darkest color preferences of the manufacturer. Cell collection preference was monitored using real-time intraprocedural CD34+ cell sampling. A mean of 4.3 total blood volumes was processed (range 2.5–6.4), with mean collection efficiency (CE) (CD34+ cells collected ÷ [pre-apheresis CD34+ count × total blood processed]) of 48% (range 8.2–107.1). The mean reticulocyte percent in patients with CE &amp;lt;30% (11.4%, range 2.5 –18.9) was significantly higher than in those with CE &amp;gt;30% (6.2%, range 1.8–11.9, p=0.0026). Two patients had a history of delayed hemolytic transfusion reactions and/or multiple RBC alloantibodies before study entry that limited preparative transfusions to reach a HbS of 30% for 3 months. They were successfully collected, and products were generated after simple transfusions with a single limited-volume red cell exchange immediately prior to collection. The success of this study was driven by optimizing apheresis strategies, including suppression of stress erythropoiesis through transfusion, real-time instrument adjustments, and efficient manufacturing that allowed a collection target of nearly half the CD34+ cells recommended in the FDA-approved gene product. Strikingly, the highly efficient ex vivo manipulation platform described here enables successful mobilization and manufacturing of gene therapy products for SCD in several months, easing the path to autologous gene therapy for SCD.

  PublisherDOI

• Post-Transplant Chronic Granulomatous Disease Patient Follow-Up: A PIDTC Survey

  Journal of Human Immunity · 2025-04-25

  articleOpen access

  Background Chronic granulomatous disease (CGD) is an inborn error of immunity caused by defects in NADPH oxidase, which causes phagocyte dysfunction. CGD is characterized by recurrent infections and autoimmunity. Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative therapy. However, data regarding patient satisfaction with post-HSCT state of health and quality of life are lacking. Methods A Primary Immune Deficiency Treatment Consortium (PIDTC) working group designed online surveys to assess post-HSCT quality of life and satisfaction with transplantation. Surveys were distributed from November 2023 to March 2024 to all members of the CGD Association of America (CGDAA). Adults who personally underwent or parents of children who underwent HSCT for CGD were asked to participate. Two surveys were offered: one for participants who were 18 years or older and one for parents of patients. Results Complete surveys representing 54 unique patients were included for analysis. Forty-four were from parents whose children were a median of 12 years old and a median of 6 years post-transplant. Nine were returned from patients who were a median of 30 years old and a median of 4 years post-transplant (Table 1). Table 1. Seventy-eight percent of patient and parent respondents reported that quality of life was better after transplant and 89% felt that the transplant improved their physical health (Figure 1). Figure 1. (A) Do you feel quality of life is better now post-transplant than it was pre-transplant? (B) Do you feel that the transplant has been beneficial from a medical perspective? PROMIS Global Health scoring tool revealed 84% of patients had mental health scores within 1 SE of the mean. Only a few mental health diagnoses were self-reported (Figure 2). Figure 2. (A) PROMIS Mental Health Report Scores. (B) Mental health condition diagnosed by a physician. After transplant, there were no self-reported cases of autoimmune disease or malignancy. Three participants were able to conceive children. One respondent used sperm banking; two did not use fertility preservation or treatments. Discussion Our data demonstrate that the majority of patients who undergo allogeneic HSCT for definitive treatment of CGD experience improvements in physical and/or mental health and enjoy an improved quality of life after transplant. Overall, this study supports positive outcomes for patients treated with allogeneic HSCT.

  PublisherDOI

• Correction: Impact of autologous blood transfusion after bone marrow harvest on unrelated donor’s health and outcome: a CIBMTR analysis

  UNC Libraries · 2025-06-26

  articleOpen access
  PublisherDOI

• Harmonized immune recovery monitoring after HCT: evidence and practical guidance from the Westhafen Intercontinental Group

  Blood Advances · 2025-09-03 · 5 citations

  articleOpen access

  ABSTRACT: Allogeneic hematopoietic cell transplantation (allo-HCT) is a curative option for patients with high-risk malignancies and nonmalignant disorders. Long-term survival depends on robust immune reconstitution (IR), which governs overall immune homeostasis and risks of infection, graft-versus-host disease, and relapse. However, despite its centrality to posttransplant outcomes, IR is not consistently monitored across transplant centers, limiting ability to generate meaningful, comparable, and translatable data. This review synthesizes current knowledge on numerical and functional IR milestones after allo-HCT, with a primary focus on flow cytometry-based monitoring of key immune cell subsets. Importantly, early CD4+ T-cell recovery (achieving >50 cells per μL by day 100 after transplant), is supported by strong clinical evidence and correlates with improved outcomes. Although emerging data suggest that additional subsets (CD8+ T cells, natural killer cells, B cells, naïve and recent thymic emigrant T cells, and γδ T cells) may also influence clinical trajectories, further harmonized, multicenter studies are needed to validate prognostic relevance across transplant settings. We propose practical, evidence-based guidelines for IR monitoring, including recommended time points, preferred assays, and flow cytometry panel components. Additionally, we highlight modifiable factors (eg, immunosuppressive drug exposures, graft manipulation) offering interventional opportunities for influencing IR. Harmonized monitoring strategies will support robust correlation between IR and clinical outcomes, guide real-time risk stratification, and facilitate the development of targeted, individualized transplant approaches. Standardization efforts led by consortia and registries are essential for advancing knowledge and optimizing care. We provide a roadmap for implementing uniform IR monitoring to improve outcomes and quality of life for allo-HCT recipients.

  PublisherOA PDFDOI

• Outcomes following matched sibling donor transplantation for severe combined immunodeficiency: a report from the PIDTC

  Blood Advances · 2025-11-25

  articleOpen access

  ABSTRACT: The Primary Immune Deficiency Treatment Consortium 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 patients with SCID 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 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 patients with leaky SCID or Omenn syndrome. 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 affected by development of grade 2 to 4 or 3 to 4 acute GVHD. Conditioning did not affect T- or B-cell reconstitution. Our data suggest omitting conditioning and GVHD prophylaxis for patients with typical SCID did not negatively affect 5-year outcomes after MSD HCT, but the data are insufficient to recommend this approach for best long-term outcomes. This trial was registered at www.clinicaltrials.gov as #NCT01186913 and #NCT01346150.

  PublisherOA PDFDOI

• Harmonized Immune Recovery Monitoring after HCT:Evidence & Practical Guidance from the Westhafen Intercontinental Group

  Utrecht University Repository (Utrecht University) · 2025-12-09

  articleOpen access

  Allogeneic hematopoietic cell transplantation (allo-HCT) is a curative option for patients with high-risk malignancies and nonmalignant disorders. Long-term survival depends on robust immune reconstitution (IR), which governs overall immune homeostasis and risks of infection, graft-versus-host disease, and relapse. However, despite its centrality to posttransplant outcomes, IR is not consistently monitored across transplant centers, limiting ability to generate meaningful, comparable, and translatable data. This review synthesizes current knowledge on numerical and functional IR milestones after allo-HCT, with a primary focus on flow cytometry-based monitoring of key immune cell subsets. Importantly, early CD4+ T-cell recovery (achieving >50 cells per μL by day 100 after transplant), is supported by strong clinical evidence and correlates with improved outcomes. Although emerging data suggest that additional subsets (CD8+ T cells, natural killer cells, B cells, naïve and recent thymic emigrant T cells, and γδ T cells) may also influence clinical trajectories, further harmonized, multicenter studies are needed to validate prognostic relevance across transplant settings. We propose practical, evidence-based guidelines for IR monitoring, including recommended time points, preferred assays, and flow cytometry panel components. Additionally, we highlight modifiable factors (eg, immunosuppressive drug exposures, graft manipulation) offering interventional opportunities for influencing IR. Harmonized monitoring strategies will support robust correlation between IR and clinical outcomes, guide real-time risk stratification, and facilitate the development of targeted, individualized transplant approaches. Standardization efforts led by consortia and registries are essential for advancing knowledge and optimizing care. We provide a roadmap for implementing uniform IR monitoring to improve outcomes and quality of life for allo-HCT recipients.

  Publisher

• Clinical spectrum of Wiskott-Aldrich syndrome carriers: Self-reported survey of 193 carriers

  Clinical Immunology · 2025-12-18 · 1 citations

  articleOpen access
  PublisherOA PDFDOI

• Identification of Distinct Subtypes in Immune Tolerance after Hematopoietic Cell Transplantation Using the Prospective ABLE1.0 Pediatric Study Cohort

  Transplantation and Cellular Therapy · 2025-10-01

  articleOpen access

  • There appears to be more than one distinct biologic subtype or pathway to achieve primary and secondary immune tolerance after HCT. • Acute GvHD induces some changes in immune tolerance development after HCT, especially in the timing of reconstitution of some immune cell populations. • Pubertal impact on thymic function, MMP3, Phosphatidylcholine metabolism, and NK and NKT cells appear to impact on different distinctive patterns of tolerance. The lack of immune tolerance after hematopoietic cell transplantation (HCT) can result in chronic graft-versus-host-disease (cGvHD), which is the primary non-relapse limitation on a successful HCT. To date, immune tolerance has been considered as a single biologic entity, but we hypothesized that post-HCT immune tolerance could develop through multiple pathways. Using the ABLE network database, which comprises measurements of 75 cell populations, 10 cytokines and chemokines, lymphocyte population telomere length, KREC and TREC, and 132 metabolites from the largest pediatric cGvHD cohort (N = 241), we applied clustering analysis to the primary immune tolerance (PIT; no acute GvHD or cGvHD) and secondary immune tolerance (SIT; previous acute GvHD and no cGvHD) patients to test whether subtypes could be identified. Evaluation of PIT found three subtypes. PIT-1, associated with post-pubertal age and lower thymic output, and increased ST2 compared to PIT-2 and PIT-3, was effector memory T cell-predominant. PIT-2, associated with prepubertal age, normal thymic output, increased B cell development, longer lymphocyte telomeres, had a naïve T cell-predominant pattern. PIT-3, associated with post-puberty, higher thymic output, and malignancy, was dominated by increased PD1 + T reg and helper T cells and decreased long chain acylcarnitine. We partially replicated these PIT subtypes using metabolomic data from a separate pediatric cohort of the COG trial ASCT0031 (24 PIT patients). Previously resolved acute GvHD had minimal impact on the overall patterns of SIT-1 and SIT-2 compared to PIT-1 and PIT-2, except for time delays in expansion of some immune cells. PIT-3 and SIT-3 were dominated by a late increase in phosphatidylcholines (lysophosphatidylcholines precursors) and long chain lysophosphatidylcholines (LYSOC20:4 and LYSOC16:2), respectively. This is the first time that distinct biologic patterns of immune reconstitution after HCT are identified, which upon validation, could potentially aid future strategies for tolerance induction.

  PublisherDOI

Recent grants

• Randomized study of low versus moderate dose busulfan in transplant for severe combined immunodeficiency

  NIH · $8.5M · 2017–2024

• NIH Grant U10HL069254

  NIH · $839k · 2017

• Unrelated Donor BMT vs. Immune Suppression for Newly Diagnosed Severe Aplastic Anemia in Children and Young Adults: BMT CTN Core Center Renewal for the Pediatric Blood and Marrow Transplant Consortium

  NIH · $1.3M · 2001–2024

• KIR-FAVORABLE HAPLOIDENTICAL TRANSPLANTATION IN CHILDREN

  NIH · $1.8M · 2014–2021

• NIH Grant R13HL127918

  NIH · $5k · 2016

Frequent coauthors

• Brenda M. Sandmaier

  Fred Hutch Cancer Center

  690 shared

• Rainer Storb

  University of Washington

  673 shared

• David G. Maloney

  University of Washington

  659 shared

• Thomas R. Chauncey

  Fred Hutch Cancer Center

  648 shared

• Barry E. Storer

  635 shared

• Michael B. Maris

  Sarah Cannon

  588 shared

• Edward Agura

  438 shared

• Richard T. Maziarz

  Oregon Health & Science University

  429 shared

Labs

• Pediatric Hematology & Oncology at Primary Children's Hospital and Huntsman Cancer InstitutePI

Education

• Fellowship, Pediatric Hematology/Oncology

  Dana Farber Cancer Institute

  1996

• Pediatric Residency, Pediatrics

  Children's Hospital of Philadelphia

  1993

Awards & honors

• PTCTC Lifetime Achievement Award in 2020