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488 citation(s) found for "Abdel, MP".

Incidence of subsequent surgical decompression following minimally invasive approaches to treat lumbar spinal stenosis: A retrospective review
Shahzad, Hania , Hussain, Nasir , D’Souza, Ryan S. , Bhatti, Nazihah , Orhurhu, Vwaire , Simopoulos, Thomas , Essandoh, Michael , Khan, Safdar N. , Weaver, Tristan, Simopoulos, Thomas , D'Souza, Ryan S , Abdel‐Rasoul, Mahmoud

Pain Practice | November 13, 2023 | Volume: 24 | Issue: 3 | Pages: 431-439

10.1111/papr.13315 | PMID 37955267 Get It

Abstract Background Context Surgical decompression is the definitive treatment for managing symptomatic lumbar spinal stenosis; however, select patients are poor surgical candidates. Consequently, minimally invasive procedures have gained popularity, but there exists the potential for failure of therapy necessitating eventual surgical decompression. Purpose To evaluate the incidence and characteristics of patients who require surgical decompression following minimally invasive procedures to treat lumbar spinal stenosis. Study Design/Setting Retrospective review. Patient Sample Patients who underwent minimally invasive procedures for lumbar spinal stenosis (Percutaneous Image‐guided Lumbar Decompression [PILD] or interspinous spacer device [ISD]) and progressed to subsequent surgical decompression within 5 years. Outcome Measures The primary outcome was the rate of surgical decompression within 5 years following the minimally invasive approach. Secondary outcomes included demographic and comorbid factors associated with increased odds of requiring subsequent surgery. Methods Patient data were collected using the PearlDiver‐Mariner database. The rate of subsequent decompression was described as a percentage while univariable and multivariable regression analysis was used for the analysis of predictors. Results A total of 5278 patients were included, of which 3222 (61.04%) underwent PILD, 1959 (37.12%) underwent ISD placement, and 97 (1.84%) had claims for both procedures. Overall, the incidence of subsequent surgical decompression within 5 years was 6.56% (346 of 5278 patients). Variables associated with a significantly greater odds ratio (OR) [95% confidence interval (CI)] of requiring subsequent surgical decompression included male gender and a prior history of surgical decompression by 1.42 ([1.14, 1.77], p = 0.002) and 2.10 times ([1.39, 3.17], p < 0.001), respectively. In contrast, age 65 years and above, a diagnosis of obesity, and a Charlson Comorbidity Index score of three or greater were associated with a significantly reduced OR [95% CI] by 0.64 ([0.50, 0.81], p < 0.001), 0.62 ([0.48, 0.81], p < 0.001), and 0.71 times ([0.56, 0.91], p = 0.007), respectively. Conclusions Minimally invasive procedures may provide an additional option to treat symptomatic lumbar spinal stenosis in patients who are poor surgical candidates or who do not desire open decompression; however, there still exists a subset of patients who will require subsequent surgical decompression. Factors such as gender and prior surgical decompression increase the likelihood of subsequent surgery, while older age, obesity, and a higher Charlson Comorbidity Index score reduce it. These findings aid in selecting suitable surgical candidates for better outcomes in the elderly population with lumbar spinal stenosis.
Hidden risk of gadolinium-based contrast agents during interventional pain medicine procedures: a retrospective chart review
Eldrige, Jason S. , Provenzano, David , Abdel-Latif, Tesneem, Klasová, Johana , Furtado-Pessoa-de-Mendonca, Laura , Yan, Dan , Hunt, Christine , Hallo-Carrasco, Alejandro, Eldrige, Jason, Provenzano, David Anthony , Hunt, Christine , Hallo‐Carrasco, Alejandro , Gonzalez‐Estrada, Alexei

Regional Anesthesia & Pain Medicine | November 11, 2023 | Volume: 49 | Issue: 10 | Pages: 751-756

10.1136/rapm-2023-104952 | PMID 37951601 Get It

Introduction Epidural steroid injections and epidural blood patches commonly involve the injection of a small amount of radiocontrast media under fluoroscopy to properly identify the target tissue or anatomic space and prevent off-target or intravascular delivery of therapeutic or diagnostic drugs. Iodinated low osmolar non-ionic contrast media is the standard preparation used as it is considered safe and cost-effective, but gadolinium-based preparations have been used as an alternative for patients with an ‘iodine’-related or radiocontrast media allergy label to prevent hypersensitivity reactions. The risk of neurotoxic events when gadolinium is inadvertently injected into the intrathecal space has been reported in recent years, raising concerns when gadolinium-based contrast media is used in lieu of iodinated low osmolar non-ionic contrast media. Methods A retrospective review was conducted of patients who received gadolinium-based contrast media for procedures with risk of inadvertent intrathecal access from January 1, 2019 to May 1, 2022. Information on patient demographics, allergy label information, and procedure description was documented for all patients who received gadolinium-based contrast media for axial spine procedures (including epidural steroid injections, epidural blood patch procedures, and selective nerve root blocks), and all side effects reported within 1 month of the procedure were recorded. Saved fluoroscopy images of all procedures for which there was concern for possible gadolinium-based contrast media-related side effect were reviewed for evidence of inadvertent intrathecal gadolinium-based contrast media administration. Descriptive statistical analysis was performed using REDCap and IBM SPSS Statistics V.28. Results We identified 508 patients who received gadolinium-based contrast media during a fluoroscopically guided axial spine procedure. These patients underwent 697 epidural procedures and 23 patients were identified as experiencing an adverse event that could be consistent with possible, probable, or clear signs of exposure to intrathecal gadolinium. Our calculated adverse event rate was 3.3%. Ten patients required additional medical evaluation or treatment. Discussion Almost all patients in our cohort had an allergy label on their chart that guided the provider to switch to gadolinium-based contrast media, but most were incomplete, ill-defined, or related to allergy to iodine but not iodinated contrast media. Such practice is not recommended based on current guidelines. The current study raises concern regarding the use of gadolinium-based contrast media in axial spine procedures, with the risk of potential severe adverse events, without evidence-based need for avoiding iodinated contrast media.
Midterm outcomes of total hip arthroplasty after internal hemipelvectomy and iliofemoral arthrodesis
Dittman, Lauren E. , Houdek, Matthew T. , Rose, Peter S. , Sim, Franklin H. , Lewallen, David G. , Couch, Cory G., Houdek, Matthew T , Abdel, Matthew P.

Journal of Surgical Oncology | November 9, 2023 | Volume: 129 | Issue: 3 | Pages: 609-616

10.1002/jso.27504 | PMID 37942700 Get It

AbstractBackground and ObjectivesRecent studies have reported acceptable outcomes after arthrodesis takedown and conversion to total hip arthroplasty (THA); however, there are no reports on outcomes after oncologic resection, which are inherently complex and may portend poorer outcomes. The purpose of this study was to examine the surgical and functional outcomes of patients who underwent prior hemipelvectomy for tumor resection and were later converted to THA.MethodsAll patients who had prior iliofemoral arthrodesis after oncologic resection that were later converted to THA at a single institution were examined. Charts were reviewed for demographic information, operative information, functional outcomes, and complications/reoperations.ResultsAll three patients in this study were males who underwent internal hemipelvectomies for chondrosarcoma. Patients were converted to THA at a mean of 26 years after arthrodesis. Mean follow‐up after conversion to THA was 7.4 years. During this follow‐up period, two of the three patients required revision surgery. At last follow‐up, the mean Harris Hip Score was 81 and the mean Mayo Hip Score was 67, and all patients were ambulatory without significant pain.ConclusionsOverall, patients who undergo iliofemoral arthrodesis after oncologic hemipelvectomy and are later converted to THA can expect to have a reasonable outcome, despite a high rate of complications and revision surgery.
Phage Therapy as a Novel Therapeutic for the Treatment of Bone and Joint Infections
Suh, Gina A. , Ferry, Tristan , Abdel, Matthew P.

Clinical Infectious Diseases | November 7, 2023 | Volume: 77 | Issue: Supplement_5 | Pages: S407-S415

10.1093/cid/ciad533 | PMID 37932115 Get It

Abstract Solutions for bone and joint infection (BJI) are needed where conventional treatments are inadequate. Bacteriophages (phages) are naturally occurring viruses that infect bacteria and have been harnessed for refractory bone and joint infections (BJI) in many case reports. Here we examine the safety and efficacy of English-language published cases of BJI since 2010 with phage therapy. From 33 reported cases of BJI treated with phage therapy, 29 (87%) achieved microbiological or clinical success, 2 (5.9%) relapsed with the same organisms, and 2 (5.9%) with a different organism. Of these 4 relapses, all but 1 had eventual clinical resolution with additional surgery or phage treatments. Eight out of 33 cases (24%) reported mild, transient adverse events with no serious events reported. Further work is needed to understand the true efficacy of phages and the role of phages in BJI. Opportunities lay ahead for thoughtfully designed clinical trials adapted to individualized therapies.
S1498 Gender Disparities in Hepatocellular Carcinoma Incidence Among 40 Years and Above Age Groups: Need for a Targeted Intervention?
Karki, Bibek , Belbase, Samjhana, Kandel, Pujan , Ghimire, Calvin, Abdelgadir, Deena M., Elbedawi, Mamoon, Bachuwa, Ghassan , Abdel-Gadir, Deena

American Journal of Gastroenterology | October 18, 2023 | Volume: 118 | Issue: 10S | Pages: S1136-S1136

10.14309/01.ajg.0000955632.53846.b8 Get It

Factors Associated With Otolaryngologists Performing Tracheotomy
Soliman, Shady , Panuganti, Bharat , Francis, David O. , Klebaner, Dasha, Asturias, Alicia , Alattar, Ali , Wood, Samuel , Terry, M. C. , Bryson, Paul C. , Tipton, Courtney B. , Zhao, Elise E. , Maria, Chloe Santa , Sung, C. Kwang , Lao, Wilson P. , Thompson, Jordan M. , Crawley, Brianna K. , Rosen, Sarah P. , Berezovsky, Anna , Hennesy, Theresa B. , Joseph, Ian T. , Sarhadi, Kamron, Kuhn, Maggie A. , Abdel‐Aty, Yassmeen , Kennedy, Maeve M. , Weissbrod, Philip A. , Abdel-Aty, Yassmeen, Soliman, Shady I , Sung, C K , Weissbrod, Philip A , Pang, John , Terry, Morgan, O'Rourke, Ashli , Grimm, David R. , Kupfer, Robbi A. , Clary, Matthew S. , Lott, David G.

JAMA Otolaryngology–Head & Neck Surgery | October 5, 2023 | Volume: 149 | Issue: 12 | Pages: 1066

10.1001/jamaoto.2023.2698 | PMID 37796485 Get It

ImportanceTracheotomies are frequently performed by nonotolaryngology services. The factors that determine which specialty performs the procedure are not defined in the literature but may be influenced by tracheotomy approach (open vs percutaneous) and other clinicodemographic factors.ObjectiveTo evaluate demographic and clinical characteristics associated with tracheotomies performed by otolaryngologists compared with other specialists and to differentiate those factors from factors associated with use of open vs percutaneous tracheotomy.Design, Setting, and ParticipantsThis multicenter, retrospective cohort study included patients aged 18 years or older who underwent a tracheotomy for cardiopulmonary failure at 1 of 8 US academic institutions between January 1, 2013, and December 31, 2016. Data were analyzed from September 2022 to July 2023.ExposureTracheotomy.Main Outcomes and MeasuresThe primary outcome was factors associated with an otolaryngologist performing tracheotomy. The secondary outcome was factors associated with use of the open tracheotomy technique.ResultsA total of 2929 patients (mean [SD] age, 57.2 [17.2] years; 1751 [59.8%] male) who received a tracheotomy for cardiopulmonary failure (652 [22.3%] performed by otolaryngologists and 2277 [77.7%] by another service) were analyzed. Although 1664 of all tracheotomies (56.8%) were performed by an open approach, only 602 open tracheotomies (36.2%) were performed by otolaryngologists. Most tracheotomies performed by otolaryngologists (602 of 652 [92.3%]) used the open technique. Multivariable regression analysis revealed that self-reported Black race (odds ratio [OR], 1.89; 95% CI, 1.52-2.35), history of neck surgery (OR, 2.71; 95% CI, 2.06-3.57), antiplatelet and/or anticoagulation therapy (OR, 1.74; 95% CI, 1.29-2.36), and morbid obesity (OR, 1.54; 95% CI, 1.24-1.92) were associated with greater odds of an otolaryngologist performing tracheotomy. In contrast, history of neck surgery (OR, 1.36; 95% CI, 0.96-1.92), antiplatelet and/or anticoagulation therapy (OR, 0.80; 95% CI, 0.56-1.14), and morbid obesity (OR, 0.94; 95% CI, 0.74-1.19) were not associated with undergoing open tracheotomy when performed by any service, and Black race (OR, 0.56; 95% CI, 0.44-0.71) was associated with lesser odds of an open approach being used. Age-adjusted Charlson Comorbidity Index score greater than 4 was associated with greater odds of both an otolaryngologist performing tracheotomy (OR, 1.26; 95% CI, 1.03-1.53) and use of the open tracheotomy technique (OR, 1.48, 95% CI, 1.21-1.82).Conclusions and RelevanceIn this study, otolaryngologists were significantly more likely than other specialists to perform a tracheotomy for patients with history of neck surgery, morbid obesity, and ongoing anticoagulation therapy. These findings suggest that patients undergoing tracheotomy performed by an otolaryngologist are more likely to present with complex and challenging clinical characteristics.
Global Cardio Oncology Registry (G-COR): Registry Design, Primary Objectives, and Future Perspectives of a Multicenter Global Initiative
Teske, Arco J. , Moudgil, Rohit , López‐Fernández, Teresa , Barac, Ana , Brown, Sherry, Deswal, Anita , Neilan, Tomas G. , Ganatra, Sarju , Qadir, Husam Abdel, Menon, Venu , Sverdlov, Aaron L. , Cheng, Richard K. , Makhoul, Silvia , Ghosh, Arjun K. , Szmit, Sebastian , Addison, Daniel , Zhang, Lili , Herrmann, Joerg , Chong, Jun Hua , Agarwala, Vivek , Iakobishvili, Zaza , Guerrero, Patricia A., Leja, Monika, Guha, Avirup , Okwuosa, Tochukwu, Silva, Carolina Carvalho, DeCara, Jeanne M. , Bauer, Brenton , Lenneman, Carrie , Sadler, Diego , Zaha, Vlad G , Addison, Daniel , Agarwala, Vivek , Iakobishvili, Zaza , Yang, Eric , Akhter, Nausheen , Guha, Avirup , Okwuosa, Tochukwu M , Collier, Patrick , DeCara, Jeanne , Bauer, Brenton , Sadler, Diego , Deswal, Anita M , Barac, Ana , López-Fernández, Teresa , Sverdlov, Aaron L , Cheng, Richard , Brown, Sherry‐Ann , Collier, Patrick

Circulation: Cardiovascular Quality and Outcomes | September 13, 2023 | Volume: 16 | Issue: 10 | Pages: No page available

10.1161/circoutcomes.123.009905 | PMID 37702048 Get It

BACKGROUND: Global collaboration in cardio-oncology is needed to understand the prevalence of cancer therapy-related cardiovascular toxicity in different risk groups, practice settings, and geographic locations. There are limited data on the socioeconomic and racial/ethnic disparities that may impact access to care and outcomes. To address these gaps, we established the Global Cardio-Oncology Registry, a multinational, multicenter prospective registry. METHODS: We assembled cardiologists and oncologists from academic and community settings to collaborate in the first Global Cardio-Oncology Registry. Subsequently, a survey for site resources, demographics, and intention to participate was conducted. We designed an online data platform to facilitate this global initiative. RESULTS: A total of 119 sites responded to an online questionnaire on their practices and main goals of the registry: 49 US sites from 23 states and 70 international sites from 5 continents indicated a willingness to participate in the Global Cardio-Oncology Registry. Sites were more commonly led by cardiologists (85/119; 72%) and were more often university/teaching (81/119; 68%) than community based (38/119; 32%). The average number of cardio-oncology patients treated per month was 80 per site. The top 3 Global Cardio-Oncology Registry priorities in cardio-oncology care were breast cancer, hematologic malignancies, and patients treated with immune checkpoint inhibitors. Executive and scientific committees and specific committees were established. A pilot phase for breast cancer using Research Electronic Data Capture Cloud platform recently started patient enrollment. CONCLUSIONS: We present the structure for a global collaboration. Information derived from the Global Cardio-Oncology Registry will help understand the risk factors impacting cancer therapy-related cardiovascular toxicity in different geographic locations and therefore contribute to reduce access gaps in cardio-oncology care. Risk calculators will be prospectively derived and validated.
Efficacy of Milrinone and Dobutamine in Cardiogenic Shock: An Updated Systematic Review and Meta-Analysis
Abdel‐Razek, Omar , Santo, Pietro Di , Jung, Richard G. , Parlow, Simon , Motazedian, Pouya , Visintini, Sarah , Labinaz, Marino , Mathew, Rebecca , Hibbert, Benjamin , Hibbert, Benjamin , Prosperi‐Porta, Graeme , Marbach, Jeffrey A. , Ramirez, F. Daniel , Simard, Trevor

Critical Care Explorations | August 28, 2023 | Volume: 5 | Issue: 9 | Pages: e0962

10.1097/cce.0000000000000962 | PMID 37649849 Get It

OBJECTIVES: Inotropic support is commonly used in patients with cardiogenic shock (CS). High-quality data guiding the use of dobutamine or milrinone among this patient population is limited. We compared the efficacy and safety of these two inotropes among patients with low cardiac output states (LCOS) or CS. DATA SOURCES: MEDLINE, Embase, and Cochrane Central Register of Controlled Trials were searched up to February 1, 2023, using key terms and index headings related to LCOS or CS and inotropes. DATA EXTRACTION: Two independent reviewers included studies that compared dobutamine to milrinone on all-cause in-hospital mortality, length of ICU stay, length of hospital stay, and significant arrhythmias in hospitalized patients. DATA SYNTHESIS: A total of eleven studies with 21,084 patients were included in the meta-analysis. Only two randomized controlled trials were identified. The primary outcome, all-cause mortality, favored milrinone in observational studies only (odds ratio [OR] 1.19 (95% CI, 1.02–1.39; p = 0.02). In-hospital length of stay (LOS) was reduced with dobutamine in observational studies only (mean difference –1.85 d; 95% CI –3.62 to –0.09; p = 0.04). There was no difference in the prevalence of significant arrhythmias or in ICU LOS. CONCLUSIONS: Only limited data exists supporting the use of one inotropic agent over another exists. Dobutamine may be associated with a shorter hospital LOS; however, there is also a potential for increased all-cause mortality. Larger randomized studies sufficiently powered to detect a difference in these outcomes are required to confirm these findings.
Safety and efficacy of immune checkpoint inhibitors in advanced penile cancer: report from the Global Society of Rare Genitourinary Tumors
Zarif, Talal El , Pond, Gregory R. , Zhuang, Tony , Master, Viraj A. , Nazha, Bassel , Niglio, Scot A. , Simon, Nicholas I. , Hahn, Andrew W. , Pettaway, Curtis A. , Tu, Shi‐Ming, Abdel‐Wahab, Noha , Velev, Maud , Flippot, Ronan , Maruzzo, Marco , Yang, Yuanquan , Rodriguez, Pablo Alvarez, Castellano, Daniel , Velasco, Guillermo de , Roviello, Giandomenico , Antonuzzo, Lorenzo , McKay, Rana R. , Hui, Gavin , Drakaki, Alexandra , Glover, Michael , Khaki, Ali Raza , El‐Am, Edward , Patel, Vaibhav G. , Piedra, A. , Gernone, Angela , Davis, Nancy B. , Matthews, Harrison, Giudice, Giulia Claire , Farolfi, Alberto , Milowsky, Matthew I. , Appleman, Leonard J. , Freeman, Dory , Sonpavde, Guru , El Zarif, Talal , Nazha, Bassel , Hahn, Andrew W , Antonuzzo, Lorenzo , Khaki, Ali Raza , El-Am, Edward , Appleman, Leonard , Choueiri, Toni K , Spiess, Philippe E , Necchi, Andrea , Sonpavde, Guru , Pond, Gregory Russell , Nassar, Amin H. , Tu, Shi‐Ming , Buti, Sebastiano , Mittra, Arjun , Gheeya, Jinesh , Vincenzi, Bruno , Cortellini, Alessio , Drakaki, Alexandra , Adra, Nabil , Mouhieddine, Tarek H. , Harrison, Michael R. , Kanesvaran, Ravindran , Barata, Pedro C. , Lee, Jae‐Lyun , Stahlfeld, Charlotte N. , Kim, Joseph W. , Choueiri, Toni K. , Spiess, Philippe E. , Necchi, Andrea , Apolo, Andrea B

JNCI: Journal of the National Cancer Institute | August 11, 2023 | Volume: 115 | Issue: 12 | Pages: 1605-1615

10.1093/jnci/djad155 | PMID 37563779 Get It

Abstract Background Treatment options for penile squamous cell carcinoma are limited. We sought to investigate clinical outcomes and safety profiles of patients with penile squamous cell carcinoma receiving immune checkpoint inhibitors. Methods This retrospective study included patients with locally advanced or metastatic penile squamous cell carcinoma receiving immune checkpoint inhibitors between 2015 and 2022 across 24 centers in the United States, Europe, and Asia. Overall survival and progression-free survival were estimated using the Kaplan-Meier method. Objective response rates were determined per Response Evaluation Criteria in Solid Tumours 1.1 criteria. Treatment-related adverse events were graded per the Common Terminology Criteria for Adverse Events, version 5.0. Two-sided statistical tests were used for comparisons. Results Among 92 patients, 8 (8.7%) were Asian, 6 (6.5%) were Black, and 24 (29%) were Hispanic and/or Latinx. Median (interquartile range) age was 62 (53-70) years. In all, 83 (90%) had metastatic penile squamous cell carcinoma, and 74 (80%) had received at least second-line treatment. Most patients received pembrolizumab monotherapy (n = 26 [28%]), combination nivolumab-ipilimumab with or without multitargeted tyrosine kinase inhibitors (n = 23 [25%]), or nivolumab (n = 16 [17%]) or cemiplimab (n = 15 [16%]) monotherapies. Median overall and progression-free survival were 9.8 months (95% confidence interval = 7.7 to 12.8 months) and 3.2 months (95% confidence interval = 2.5 to 4.2 months), respectively. The objective response rate was 13% (n = 11/85) in the overall cohort and 35% (n = 7/20) in patients with lymph node–only metastases. Visceral metastases, Eastern Cooperative Oncology Group (ECOG) performance status of 1 or higher, and a higher neutrophil/lymphocyte ratio were associated with worse overall survival. Treatment-related adverse events occurred in 27 (29%) patients, and 9.8% (n = 9) of the events were grade 3 or higher. Conclusions Immune checkpoint inhibitors are active in a subset of patients with penile squamous cell carcinoma. Future translational studies are warranted to identify patients more likely to derive clinical benefit from immune checkpoint inhibitors.
Genotype, oxidase status, and preceding infection or autoinflammation do not affect allogeneic HCT outcomes for CGD
Leiding, Jennifer W. , Arnold, Danielle E. , Parikh, Suhag , Logan, Brent R. , Marsh, Rebecca , Griffith, Linda M. , Wu, Ruizhe , Kidd, Sharon A. , Mallhi, Kanwaldeep , Chellapandian, Deepak , Lim, Stephanie Si , Grunebaum, Eyal , Falcone, Emilia Liana , Grossman, Debbi, Prasad, Vinod K. , Heimall, Jennifer , Burroughs, Lauri M. , Bleesing, Jack , Kapoor, Neena , Dara, Jasmeen , Williams, Olatundun , Kapadia, Malika, Oshrine, Benjamin , Rayes, Ahmad , Chong, Hey , Satter, Lisa Forbes , Martinez, Caridad , Lugt, Mark T. Vander , Yu, Lolie C. , Joshi, Avni Y. , Prockop, Susan E. , Aquino, Victor , Madden, Lisa, Milner, Jordan , Rangarajan, Hemalatha G. , Shah, Ami J. , Gillio, Alfred P. , Knutsen, Alan P. , Miller, Holly, Graham, Pamela, Bauchat, Andrea , Bunin, Nancy , Teira, Pierre , Petrović, Aleksandra , Chandra, Sharat , Abdel‐Azim, Hisham , Birbrayer, Olga, Cowan, Morton J. , Dvorak, Christopher C. , Pai, Sung‐Yun , Puck, Jennifer M. , Torgerson, Troy R. , Kang, Elizabeth M. , Si Lim, Stephanie J , Murguia-Favela, Luis , Prasad, Vinod K , Touzot, Fabien , Bednarski, Jeffrey J , Cuvelier, Geoffrey D E , Forbes Satter, Lisa R , Martinez, Caridad , Joshi, Avni, Prockop, Susan E , Dávila Saldaña, Blachy J , Broglie, Larisa A , Ebens, Christen L , Milner, Jordan , Rangarajan, Hemalatha G , Chandra, Sharat , Dorsey, Morna J , Cowan, Morton J , Dvorak, Christopher C , Haddad, Elie , Kohn, Donald B , Notarangelo, Luigi D , Puck, Jennifer M , Pulsipher, Michael A. , Torgerson, Troy R , Malech, Harry L , Kang, Elizabeth M , Parikh, Suhag , Leiding, Jennifer W , Shah, Ami J , Dara, Jasmeen , Cuvelier, Geoff D.E. , Chandrakasan, Shanmuganathan , Saldaña, Blachy J. Dávila , Broglie, Larisa , Ebens, Christen L. , DeSantes, Kenneth B , Moore, Theodore Bruce , Graham, Pamela, Dorsey, Morna J. , Haddad, Élie , Kohn, Donald B. , Malech, Harry L.

Blood | August 10, 2023 | Volume: 142 | Issue: 24 | Pages: 2105-2118

10.1182/blood.2022019586 | PMID 37562003 Get It

Abstract Chronic granulomatous disease (CGD) is a primary immunodeficiency characterized by life-threatening infections and inflammatory conditions. Hematopoietic cell transplantation (HCT) is the definitive treatment for CGD, but questions remain regarding patient selection and impact of active disease on transplant outcomes. We performed a multi-institutional retrospective and prospective study of 391 patients with CGD treated either conventionally (non-HCT) enrolled from 2004 to 2018 or with HCT from 1996 to 2018. Median follow-up after HCT was 3.7 years with a 3-year overall survival of 82% and event-free survival of 69%. In a multivariate analysis, a Lansky/Karnofsky score &lt;90 and use of HLA-mismatched donors negatively affected survival. Age, genotype, and oxidase status did not affect outcomes. Before HCT, patients had higher infection density, higher frequency of noninfectious lung and liver diseases, and more steroid use than conventionally treated patients; however, these issues did not adversely affect HCT survival. Presence of pre-HCT inflammatory conditions was associated with chronic graft-versus-host disease. Graft failure or receipt of a second HCT occurred in 17.6% of the patients and was associated with melphalan-based conditioning and/or early mixed chimerism. At 3 to 5 years after HCT, patients had improved growth and nutrition, resolved infections and inflammatory disease, and lower rates of antimicrobial prophylaxis or corticosteroid use compared with both their baseline and those of conventionally treated patients. HCT leads to durable resolution of CGD symptoms and lowers the burden of the disease. Patients with active infection or inflammation are candidates for transplants; HCT should be considered before the development of comorbidities that could affect performance status. This trial was registered at www.clinicaltrials.gov as #NCT02082353.