LASIK is associated with early cataract surgery in healthy patients.

PURPOSE: To evaluate the association between LASIK and early cataract phacoemulsification surgery (PE). METHODS: A matched case-control study was conducted. Cases were otherwise healthy adults with a history of LASIK. Groups were paired according to corrected-distance visual acuity (CDVA), axial length, and cataract grade. RESULTS: A total of 213 patients were included. 85 patients were classified as post-LASIK group and 128 as controls. The mean age at the time of LASIK was 42.32 ± 9.24 years. The mean CDVA before PE was 0.29 ± 0.19 Log MAR in post-LASIK group and 0.34 ± 0.22 Log MAR in controls (p = 0.07). The mean axial length was 23.99 ± 1.78 mm in post-LASIK group and 23.62 ± 0.98 mm in controls (p = 0.085). The mean nuclear cataract grading was 1.36 in post-LASIK group and 1.47 in controls (p = 0.34). The mean age at the time of PE was 60.18 ± 7.46 years in post-LASIK group and 67.35 ± 9.28 in controls (p < 0.0005). The difference between the mean age of LASIK and the mean age of PE was 17.85 ± 5.72 years. There was a positive association between the post-LASIK group and the age of PE ≤ 55 years (OR: 4.917, 95% CI: 2.21-10.90, p < 0.001). CONCLUSION: LASIK may be associated with early PE. Patients with LASIK had a 7-year earlier PE compared to a matched control group.

Ocular Mucous Membrane Pemphigoid: The Effect of Risk Factors at Presentation on Treatment Outcomes.

PURPOSE: Analyze the influence of risk factors at presentation in the long-term immunosuppressive therapy (IMT) outcomes of ocular mucous membrane pemphigoid (OMMP). DESIGN: Retrospective multicenter study. PARTICIPANTS: Patients with OMMP seen at the Duke Eye Center, Tecnologico de Monterrey, and Hospital Clinic of Barcelona from 1990 to 2022. METHODS: Data at presentation on demographics, direct immunofluorescence, ocular findings, sites of extraocular manifestations (EOMs), and previous treatments in patients with a clinical or laboratory diagnosis of OMMP, were analyzed with multivariable analysis and Kaplan-Meier plots to identify factors associated with adverse outcomes. MAIN OUTCOME MEASURES: (1) Inflammatory control (no conjunctival inflammation in both eyes at 3 months on IMT); (2) relapse (new-onset inflammation after absolute control in either eye); (3) progression (≥ 1 cicatrizing stage progression in either eye); and (4) vision loss (≥ 2 Snellen lines). RESULTS: A total of 117 patients (234 eyes), 61% (71/117) of whom were women, with a mean age of 66.6 (SD: 12.4) years (range: 37-97 years) and median follow-up of 34 months (interquartile range: 16-66 months; range: 3-265 months), were enrolled. Inflammatory control was achieved in 57% of patients (67/117), with high-risk EOM (HR-EOM), including esophageal, nasopharyngeal, and/or genital involvement (adjusted odds ratio [aOR]: 12.51; 95% confidence interval [CI]: 2.61-59.99; P = 0.002) and corneal scarring (aOR: 3.06; 95% CI, 1.15-8.14; P = 0.025), as significant risk factors for persistent inflammation. Disease relapse, progression, and vision loss occurred in 20% of patients (23/117), 12% of patients (14/117), and 27% of patients (32/117), respectively. Baseline corneal scarring was a risk factor for relapse (adjusted hazard ratio: 4.14; 95% CI: 1.61-10.62; P = 0.003), progression (aOR: 11.46; 95% CI: 1.78-73.75; P = 0.010), and vision loss (aOR: 3.51; 95% CI: 1.35-9.10; P = 0.010). HR-EOM was associated with stage progression (aOR, 34.57; 95% CI, 6.57-181.89; P<0.001) and vision loss (aOR, 8.42; 95% CI, 2.50-28.42; P = 0.001). No significant differences were found between IMT regimes and relapse (P = 0.169). CONCLUSIONS: Ocular mucous membrane pemphigoid presenting with HR-EOMs and corneal scarring has an increased risk of stage progression and vision loss. Corneal scarring and severe inflammation at baseline were associated with an increased risk of relapse. A disease progression staging system incorporating both the HR-EOMs and corneal involvement is required to predict the visual outcome of OMMP better. FINANCIAL DISCLOSURE(S): Proprietary or commercial disclosure may be found in the Footnotes and Disclosures at the end of this article.

Knowledge and compliance with contact lens care: A population-based study at a referral center in Northeast Mexico.

OBJECTIVE: Despite the increasing prevalence of contact lens (CL) wear, knowledge and compliance with proper care remain suboptimal. This study aims to assess the level of knowledge and compliance with lens care practices in patients attending a third-level institution in Northeast Mexico. METHODS: A cross-sectional study using a self-administered online survey was conducted. Patients at the Instituto Tecnolgico de Monterrey were invited to participate. The questionnaire consisted of three sections: demographic data and CL information, CL practices, and CL care knowledge. Participants responding correctly to 6/7 questions from the second and third sections were classified as having good compliance and good knowledge, respectively. Predictive factors for good compliance and knowledge were calculated using a logistic regression model. RESULTS: A total of 287 users participated in the study. The median age was 25 (14-78) years with a female (n = 221, 77 %) predominance. Good knowledge was observed in 215 (74.9 %), whereas only 42 (14.6 %) presented good compliance. Failing to replace CL as prescribed (n = 199, 69.3 %) and sleeping with the devices (n = 198, 69 %) were the most frequent practices causing non-compliance. Whereas prohibition of swimming with CL was the least known practice (n = 74, 25 %). Users with recent (≤5 years) wearing experience were more likely to present good knowledge (OR 2.19, p = 0.014) and compliance (OR 3.15, p < 00.01). No statistical correlation was established between knowledge and compliance. CONCLUSION: Non-compliance and lack of knowledge of proper CL care were prevalent among in this population. Long-term CL users were at higher risk of non-compliance and lack of knowledge. Moreover, knowledge was not related to compliance; therefore, different strategies must be implemented to reduce CL misconduct.

Underrepresentation of Low- and Middle-Income Nations in Ophthalmology Journals: A Critical Analysis on Diversity, Equity, and Global Representation.

Purpose: Low- to middle-income nations contain more than 80% of the world's population; however, only 4% of articles in ophthalmology journals belong to these countries. We aim to analyze the global diversity of the editorial boards of ophthalmology journals. Methods: Cross-sectional study, including all journals in the Ophthalmology section of the SCImago Journal & Country Rank (SJR). Journals were classified according to the country of origin, SJR interquartile range (Q1-Q4), impact factor, and open-access policy. Global diversity among journals was determined by the country of affiliation of editors-in-chief and editorial board members. Nations were classified by income according to the World Bank's 2022 system. The association between editorial diversity and the journal's metrics and country of origin was analyzed using the χ2 test and the Mann-Whitney U test. Results: A total of 116 journals were included and 83.6% belonged to high-income nations. Only 18 (13.3%) editors-in-chief and 582 (13.5%) board members were affiliated with middle-income nations. The most prevalent middle-income countries in editorial boards were Brazil (n = 184, 4.26%), India (n = 150, 3.47%), Turkey (n = 42, 0.97%), and Iran (n = 36, 0.83%). Only 40 (1.07%) editorial board members of Q1 journals were affiliated with non-high-income nations, most belonging to India (n = 28, 70%). Journals from middle-income nations had a statistically significant lower prevalence in the first- and second-quartile ranking (P < 0.001) and a higher proportion of open-access policies (P = 0.019). Conclusions: A clear underrepresentation of low- to middle-income nations was observed in ophthalmology journals. Promoting editorial diversity and minimizing the possibility of editorial bias could lead to greater exposure to real-world data from resource-constrained settings. Translational Relevance: The documented underrepresentation of low- to middle-income nations in ophthalmology journals highlights the importance of promoting diversity and inclusion.

Challenges in the diagnosis and management of simultaneous, bilateral, toxic anterior segment syndrome after phacorefractive surgery.

A 59-year-old woman presented with a 24-hour history of severe, bilateral, painless vision loss starting 1 day after immediately sequential, bilateral, phacorefractive surgery with multifocal intraocular lens (IOL) implantation at another institution. Best-corrected visual acuity was counting fingers at 1 foot in the right eye and 20/100 in the left eye. Slit-lamp evaluation showed a dense fibrin membrane on the anterior surface of the IOL and significant anterior chamber inflammation in both eyes. B-scan ultrasound revealed bilateral vitreous haze, without membrane formation. The rapid onset, absence of sharp pain, ciliary injection, conjunctival chemosis, eyelid edema, and erythema raised suspicion for bilateral toxic anterior segment syndrome. Significant clinical improvement after high-dose prednisone and hourly prednisolone acetate eye drops supported the diagnosis. After 6 months of tapering anti-inflammatory therapy, optical coherence tomography of the macula showed no pathologic changes, and the patient's best-corrected visual acuity improved to 20/25 in both eyes.

Assessing the Efficacy of Mitoxantrone and Doxorubicin as Frontline Anthracyclines During Induction Therapy of Newly Diagnosed Acute Promyelocytic Leukemia.

BACKGROUND: Therapeutic advances in acute promyelocytic leukemia (APL) have transformed it into today's most curable form of leukemia. However, recommended agents, including arsenic trioxide, idarubicin, or daunorubicin, are not easily available in low-middle-income countries, where outcomes remain suboptimal. We aimed to assess the efficacy and safety of more accessible anthracyclines. METHODS: We conducted a retrospective cohort study including sixty-one patients diagnosed with APL over a 15-year period. Patients received low-dose all-trans retinoic acid (ATRA, 25 mg/m2) with mitoxantrone or doxorubicin as an induction to remission therapy. Groups were compared using the χ2 and Student's t-tests. Kaplan-Meier analysis was used for survival analyses. RESULTS: Thirty (49.18%) patients received mitoxantrone, and 31 (50.82%) received doxorubicin. The median follow-up was 24.6 months (1-146). Twenty-eight (93.3%) patients achieved complete remission (CR) in the mitoxantrone group and 28 (87.1%) in the doxorubicin group (p=0.103), and the median time to CR was 40 and 31 days, respectively. Mitoxantrone had a 6.7% early mortality rate and a 16.7% relapse rate compared with doxorubicin (3.2% and 32.3%, respectively). No differences were found in survival (p = 0.795), hospitalization days (p = 0.261), or adverse events (p = 0.554). CONCLUSIONS: Using mitoxantrone or doxorubicin as induction therapy in newly diagnosed APL is a safe and adequate alternative with comparable outcomes to first-line agents in scenarios where the latter might not be readily available, such as in low-middle-income countries.

Outcomes and survival predictors of Latin American older adults with acute myeloid leukemia: Data from a single center

ABSTRACT Introduction: Acute myeloid leukemia (AML) is most commonly presented in older adults; however, it appears 10 years earlier in Latin American countries. Clinical evolution in older adults from this populations has not been characterized. We analyzed outcomes and survival predictors. Methods: Patients ≥ 55 years old diagnosed with AML at a hematology referral center from 2005 to 2020 receiving intensive chemotherapy (IC), low-dose cytarabine (LDAC) and best supportive care (BSC) were included. Survival analysis included the Kaplan-Meier and Cox models and the cumulative incidence of relapse (CIR). Results: Seventy-five adults were included and the overall survival (OS) was 4.87, 1.67 and 1.16 months, using IC, LDAC and BSC, respectively. The IC led to a higher OS (p < 0.001) and was a protective factor for early death, at a cost of more days spent hospitalized and more non-fatal treatment complications; non-significant differences were found between the LDAC and BSC. Eight (10.7%) patients underwent hematopoietic cell transplantation, with a higher OS (p = 0.013). Twenty (26.7%) patients achieved complete remission; 12 (60%) relapsed with a 6-month CIR of 57.9% in those < 70 years old vs. 86.5% in those ≥ 70 years old, p = 0.034. Multivariate analysis showed the white blood cell count (WBC) and IC had a significant impact on the patient survival, whereas chronological age and the Charlson comorbidity index (CCI) did not. Conclusion: AML in low-middle income countries demands a different approach; the IC improves survival, even with a high incidence of relapse, and should be offered as first-line treatment. Eligibility criteria should include WBC and a multidimensional evaluation. The age per se and the CCI should not be exclusion criteria to consider IC.

Infection-related mortality after HLA-identical and haploidentical hematopoietic cell transplantation using reduced-intensity conditioning in an outpatient setting.

BACKGROUND: Despite the improvements in supportive care for allogeneic-hematopoietic cell transplantation (allo-HCT) recipients, infectious complications and infection-related mortality (IRM) continue to be a major issue for transplantation centers. METHODS: We herein report the infectious complications and IRM of 107 and 89 patients that underwent haploidentical (haplo-HCT) or HLA-identical HCT at a tertiary referral center during 2013-2020. Patients in the haplo-HCT group received post-transplant cyclophosphamide (PT-Cy), and all received reduced-intensity conditioning regimens. RESULTS: More haplo-HCT recipients presented severe infections in the pre-engraftment period (22.4% vs. 6.7%, p = 0.003). Viral (14.9% vs. 4.5%, p = 0.016) and fungal (12.1% vs. 1.1%, p = 0.003) etiologies were more common in this period in this group. The 100-day and 2-year cumulative incidence of IRM was 15% and 21% for the haplo-HCT and 5.6% and 17% for the HLA-identical group; no significant differences were observed between the groups. Fungal pathogens mainly contributed to IRM (33.3%). Infections were the most common cause of mortality (40/81, 49.4%). There were significant differences in donor/recipient CMV serostatus between transplant groups (0.002). CONCLUSIONS: No differences in IRM were observed based on allo-HCT type, with more haplo-HCT patients suffering from severe infections in the pre-engraftment period. Studies to assess future prevention, diagnostic, and treatment strategies to reduce IRM are warranted.

Wessely corneal ring phenomenon: An unsolved pathophysiological dilemma.

The cornea is a densely innervated avascular tissue showing exceptional inflammatory and immune responses. The cornea is a site of lymphangiogenic and angiogenic privilege devoid of blood and lymphatic vessels that limits the entry of inflammatory cells from the adjacent and highly immunoreactive conjunctiva. Immunological and anatomical differences between the central and peripheral cornea are also necessary to sustain passive immune privilege. The lower density of antigen-presenting cells in the central cornea and the 5:1 peripheral-to-central corneal ratio of C1 are two main features conferring passive immune privilege. C1 activates the complement system by antigen-antibody complexes more effectively in the peripheral cornea and, thus, protects the central corneas' transparency from immune-driven and inflammatory reactions. Wessely rings, also known as corneal immune rings, are noninfectious ring-shaped stromal infiltrates usually formed in the peripheral cornea. They result from a hypersensitivity reaction to foreign antigens, including those of microorganism origin. Thus, they are thought to be composed of inflammatory cells and antigen-antibody complexes. Corneal immune rings have been associated with various infectious and noninfectious causes, including foreign bodies, contact lens wear, refractive procedures, and drugs. We describe the anatomical and immunologic basis underlying Wessely ring formation, its causes, clinical presentation, and management.

Rheumatoid arthritis-associated peripheral ulcerative keratitis outcomes after early immunosuppressive therapy.

BACKGROUND/AIMS: To evaluate the role of early immunosuppressive therapy (IMT) in the management of rheumatoid arthritis (RA)-associated peripheral ulcerative keratitis (PUK). METHODS: Single-centre, retrospective, comparative cohort study. Patients with RA-associated PUK were divided into two groups; those exposed to and those not exposed to early IMT, defined as administrating therapy within the first 4 weeks from the PUK onset. Outcomes included PUK recurrence, control of inflammation and development of ocular complications, including corneal scarring and perforation, cataract formation or progression and permanent visual loss. RESULTS: A total of 52 eyes from 36 patients were included for analysis; 37 (71.2%) eyes received early IMT and 15 (28.8%) eyes did not. Follow-up time was 41.2+53.3 months (range: 4-236 months). While early IMT was a protective factor (HR 0.345, 95% CI 0.126 to 0.946, p=0.039), late RA diagnosis after PUK onset (HR 4.93, 95% CI 1.75 to 13.85, p=0.002) and retarded (≥2 months) control of inflammation (HR 8.37, 95% CI 1.88 to 37.16, p=0.005) were risk factors for PUK recurrence. Late IMT (OR 7.75, 95% CI 2.00 to 29.99, p=0.003), an unknown diagnosis of RA at first visit (OR 4.14, 95% CI 1.15 to 14.91, p=0.030) and at least one PUK recurrence (OR 6.42, 95% CI 1.71 to 24.07, p=0.006) were risk factors for visual loss. Survival analysis rendered eyes exposed to early IMT a lower risk of PUK recurrence (p=0.039). CONCLUSION: Eyes with RA-associated PUK exposed to early IMT were more likely to achieve earlier inflammatory control, fewer recurrences and had better visual outcomes.

Outcomes and survival predictors of Latin American older adults with acute myeloid leukemia: Data from a single center.

INTRODUCTION: Acute myeloid leukemia (AML) is most commonly presented in older adults; however, it appears 10 years earlier in Latin American countries. Clinical evolution in older adults from this populations has not been characterized. We analyzed outcomes and survival predictors. METHODS: Patients ≥ 55 years old diagnosed with AML at a hematology referral center from 2005 to 2020 receiving intensive chemotherapy (IC), low-dose cytarabine (LDAC) and best supportive care (BSC) were included. Survival analysis included the Kaplan-Meier and Cox models and the cumulative incidence of relapse (CIR). RESULTS: Seventy-five adults were included and the overall survival (OS) was 4.87, 1.67 and 1.16 months, using IC, LDAC and BSC, respectively. The IC led to a higher OS (p < 0.001) and was a protective factor for early death, at a cost of more days spent hospitalized and more non-fatal treatment complications; non-significant differences were found between the LDAC and BSC. Eight (10.7%) patients underwent hematopoietic cell transplantation, with a higher OS (p = 0.013). Twenty (26.7%) patients achieved complete remission; 12 (60%) relapsed with a 6-month CIR of 57.9% in those < 70 years old vs. 86.5% in those ≥ 70 years old, p = 0.034. Multivariate analysis showed the white blood cell count (WBC) and IC had a significant impact on the patient survival, whereas chronological age and the Charlson comorbidity index (CCI) did not. CONCLUSION: AML in low-middle income countries demands a different approach; the IC improves survival, even with a high incidence of relapse, and should be offered as first-line treatment. Eligibility criteria should include WBC and a multidimensional evaluation. The age per se and the CCI should not be exclusion criteria to consider IC.

Congenital eyelid imbrication syndrome in a Hispanic newborn: case report and review of the literature.

A 6-hour newborn boy presented with bilateral overriding of the upper eyelids during sleep and spontaneous eyelid eversion. Physical examination revealed canthal tendon laxity and hyperemia of the tarsal conjunctiva of both eyes. A diagnosis of congenital eyelid imbrication syndrome (CEIS) was established. The cornea and remaining adnexal structures were healthy; therefore, conservative management with lubricant ointment was initiated. On day 4, complete resolution of upper eyelid overriding and absence of spontaneous eversion was observed. CEIS is a rare eyelid malposition disorder in which the upper eyelid overrides the lower. Most CEIS cases described in the literature present at birth, occur in Asian patients, are associated with floppy eyelid, and resolve spontaneously. The authors present the first report of CEIS in a Hispanic newborn and a literature review of previously reported cases.

Reproducibility, Repeatability, and Correlation of Central Corneal Thickness Measurement with the Pentacam Scheimpflug System and Ultrasound Pachymetry.

BACKGROUND/AIM: The importance of an accurate determination of central corneal thickness (CCT) relies on its diagnostic and therapeutic implications in glaucoma, corneal ectasias, corneal edema and endothelial function monitoring, and refractive surgery suitability, among others. We aimed to analyze the repeatability, reproducibility, correlation, and laterality variations of CCT measurements performed with the Pentacam HR and the standard of care ultrasound A-scan (USP). METHODS: A cross-sectional study including CCT measurements of healthy individuals was performed by three independent examiners with the Pentacam HR and USP. Intra-observer and inter-observer variations were calculated with intra-class correlation coefficients (ICCs). Bland-Altman plots and 95% limits of agreement (95% LoA) were used to assess the agreement between devices. Linear correlation was calculated with Pearson's coefficient. RESULTS: Thirty individuals (60 eyes), including 10 (33.3%) men and 20 (66.6%) women, with a mean age of 30.0 ± 9.1 years, were studied. No statistical differences were found in CCT measurements between Pentacam HR (range 500 - 609 µm) and USP (range 498 - 628 µm). There was a high degree of correlation in repeatability and reproducibility of each independent device (ICC > 0.90). Pearson's correlation between 1 vs. 2, 2 vs. 3, and 3 vs. 1 Pentacam HR attempts were 0.914, 0.958, and 0.925, respectively (p < 0.001). Corresponding results for USP were 0.957, 0.957, and 0.943 (p < 0.001). The Pentacam HR tended to overestimate CCT by a mean difference of 3.77 µm (95% LoA, - 24.9 - 18.4). Right eyes were also overestimated (- 3.6 ± 14.1 µm) with the Pentacam HR device, whereas left eyes were underestimated (1.3 ± 11.1 µm). CONCLUSIONS: The Pentacam HR device provides reliable operator-independent estimates of CCT. Right eyes exhibited a tendency to overestimate with the Pentacam HR. We suspect this difference is due to USP underestimation related to patients' position while performing the study. In clinically relevant scenarios, performing a third measurement and cautiously measuring right eyes can provide higher accuracy.

Novel RB1 germline mutation in a healthy man.

BACKGROUND: Retinoblastoma (Rb) most frequently presents as a unilateral sporadic disease up to 40% of cases, however, arise from a monoallelic germline pathogenic variant. Only 10% of the germline mutations are inherited, and high penetrance is seen in up to 90% of these cases. As an effort to optimize counseling and screening, mutations are classified according to inheritance patterns. However, RB1 spectrum is highly heterogeneous, and information for unaffected carriers remains scarce. MATERIALS AND METHODS: The Mexican family of a 5-month-old patient diagnosed with Rb was studied. The family consisted of five individuals (father, mother, and three siblings). Genetic testing using a next-generation sequencing assay targeting RB1 with oligonucleotide baits designed to capture its exons and 20 bases flanking intronic sequences was performed in every family member. Clinical history and a complete ophthalmological examination (best-corrected visual acuity, slit-lamp biomicroscopy, macular optical coherence tomography, fundus autofluorescence, optical coherence tomography angiography, and electrophysiological studies) were performed in members testing positive to RB1 mutation. RESULTS: The father and her five-month-old daughter tested positive for a non-synonymous RB1 mutation c.459del (p.Lys154Serfs*21). The girl presented with bilateral retinoblastoma, successfully treated with cryotherapy and intravenous chemotherapy. The father had no relevant findings on imaging studies or ophthalmologic evaluation. CONCLUSIONS: This report describes a rare case of a novel low-penetrance RB1 germline mutation. Long-term follow-up of the father will include periodic evaluation of the eyes and orbits, and surveillance for systemic sarcoma and secondary malignancies. Implications for unaffected individuals need to be further studied.

Clinical profile of melanocytic lesions of the ocular surface in a Hispanic population.

PURPOSE: To determine the prevalence, clinical characteristics, and demographic factors of melanocytic lesions of the ocular surface, such as racial melanosis, primarily acquired melanosis, conjunctival nevus, and conjunctival melanoma in a Hispanic population. MATERIALS AND METHODS: A cross-sectional and observational study was undertaken in a tertiary referral ophthalmological center in northern Mexico from December 2020 to April 2021. All patients attending an ophthalmology specialty clinic were screened during their first visit in order to detect melanocytic lesions of the ocular surface. Demographic factors, clinical characteristics, and diagnosis and treatment were recorded. RESULTS: 227 patients were screened for melanocytic lesions. Melanocytic lesions were identified in 114 patients (50.2%). The prevalence of the different melanocytic lesions in the screened population was racial melanosis, 45.3%; primary acquired melanosis, 3.5%, and conjunctival nevus 1.3%. No conjunctival melanoma was identified in the screened population. Primary acquired melanosis was more common in the fifth to sixth decade of life and in females. Racial melanosis showed no gender predilection and was also more common in the fifth to sixth decade of life. Only 1 melanocytic lesion (a primary acquired melanosis) required medical treatment with excisional biopsy and cryotherapy. CONCLUSION: The prevalence of racial melanosis is remarkably high in the Hispanic population. While less prevalent, primary acquired melanosis is also present in a considerable percentage of Hispanic patients. Both melanocytic lesions exhibit demographic characteristics that match those previously reported in the medical literature.

Hematopoietic stem cell transplantation for uncommon immune-mediated neurological disorders: A literature review.

BACKGROUND: Chronic inflammatory demyelinating polyneuropathy (CIDP), stiff-person syndrome (SPS), neuromyelitis optica spectrum disorders (NMOSD) and severe refractory myasthenia gravis (MG) are immune-mediated neurological diseases that severely affect patients' functionality and quality of life, with a considerable percentage undergoing relapse or not responding to conventional treatment options. Autologous hematopoietic stem cell transplantation (auto-HSCT) has emerged as a potential second-line treatment alternative. METHODS: We performed a literature review in PubMed/Medline, EMBASE, Web of Science and Cochrane Library from inception to September 2021 of reported cases and studies of CIDP, SPS, NMOSD and MG that underwent HSCT as a treatment option. RESULTS: A total of 173 patients who underwent HSCT were found, including 32 patients described in case reports and 60 in a phase 2 clinical trial with CIDP, 29 patients with SPS, 42 patients with NMOSD and 10 patients with refractory MG. Complete remission was documented in 68/92 patients with CIDP, 13/29 with SPS and 10/10 with MG. From the NMOSD cases, 24/42 were relapse-free at last follow-up, with 13/33 having negative anti-AQ4 antibodies after HSCT. From all the included studies, only 8/173 patients received an allogeneic HSCT, 4/8 after a failed auto-HSCT. All showed clinical improvement and disease remission. CONCLUSION: HSCT has the potential to induce long-term remission in patients with CIDP, NMOSD, SPS or MG with adequate safety and tolerability. Collaboration between centers is needed to implement larger, homogeneous prospective studies, focusing on immunological correlates of favorable long-term response.

Autologous ATG-free hematopoietic stem cell transplantation for refractory autoimmune rheumatic diseases: a Latin American cohort.

Autologous hematopoietic stem cell transplantation (HSCT) has been recognized as treatment alternative for patients with severe, refractory autoimmune rheumatic diseases (ARDs). Usually, anti-thymocyte globulin (ATG)-containing conditioning regimens are employed; however, ATG is unavailable in some developing nations. We report our 15-year clinical experience autografting patients with ARDs with an ATG-free conditioning regimen and a brief assessment of patient-reported outcomes post-HSCT. All patients had active disease and were resistant to multiple lines of treatment. Event-free survival (EFS) was assessed using the Kaplan-Meier method. Eight patients underwent autologous HSCT. Diagnoses included juvenile idiopathic arthritis (n = 3), systemic lupus erythematosus (n = 2), systemic sclerosis (n = 2), and rheumatoid arthritis (n = 1). Median time from diagnosis to HSCT was 3 years (0.75-19). Hematological recovery was documented in all recipients, and 4 patients (50%) completed the procedure in a completely ambulatory setting. Five (62.5%) patients achieved complete response and 3 (37.5%) partial response. The median EFS was 7 months (95% CI, 4.97-9.02), and the 1-year EFS rate was 21.9% (95% CI, 18.25-25.76). Transplant-related mortality was 0%, and 1 recipient died 8 years post-HSCT due to chronic kidney disease. Six (75%) patients presented steroid dosage reduction post-HSCT, and 2 (25%) perceived improvement in functionality despite having relapsed. HSCT is a viable treatment alternative for selected patients with severe therapy-resistant ARDs, as an improvement in disease management and quality of life was documented. The need remains to elucidate the characteristics of the optimal HSCT candidate, as well as the adequate conditioning regimen when ATG is not available. Key Points • Despite advances in the treatment of autoimmune rheumatic diseases, some patients remain refractory. In this context, autologous hematopoietic stem cell transplantation (HSCT) rises as a viable alternative. • Of 8 HSCT recipients with autoimmune rheumatic diseases, 5 (62.5%) patients achieved complete response and 3 (37.5%) partial response, with a 1-year event-free survival of 21.9%. • Transplant-related mortality was 0%, with 4 (50%) patients autografted in a completely outpatient setting. • Even when relapse presented, patients reported an improvement in functionality and quality of life; also, a better response to DMARDs and a reduction in steroid dependency post-HSCT were documented.