RODRIGO DE HOLANDA MENDONCA
Projetos de Pesquisa
Unidades Organizacionais
Instituto Central, Hospital das Clínicas, Faculdade de Medicina - Médico
LIM/45 - Laboratório de Fisiopatologia Neurocirúrgica, Hospital das Clínicas, Faculdade de Medicina
LIM/45 - Laboratório de Fisiopatologia Neurocirúrgica, Hospital das Clínicas, Faculdade de Medicina
8 resultados
Resultados de Busca
Agora exibindo 1 - 8 de 8
bookPart Polirradiculoneurite inlamatória aguda (Síndrome de Guilain-Barré)(2021) MENDONçA, Rodrigo de Holanda- Immune-Mediated Necrotizing Myositis Presenting with Cutaneous Lesions(2021) SOUZA, Bruno de Castro e; FASCIANI, Isaura A.; SWICZAR, Bethania C. C.; MENDONCA, Rodrigo de H.; VALENTE, Neusa Y. S.
- Real-World Data from Nusinersen Treatment for Patients with Later-Onset Spinal Muscular Atrophy: A Single Center Experience(2021) MENDONCA, Rodrigo H.; POLIDO, Graziela J.; MATSUI, Ciro; SILVA, Andre M. S.; SOLLA, Davi J. F.; REED, Umbertina C.; ZANOTELI, EdmarBackground: Spinal muscular atrophy (SMA) is a motor neuron disease associated with progressive muscle weakness and motor disability. Objective: This study aims to report the evaluation of nusinersen, an antisense oligonucleotide, on motor function in patients with SMA types 2 and 3. Methods: This single-center retrospective observational study assessed nusinersen therapy outcomes, measured by HSMFSE or CHOP-INTEND scales, in patients with SMA types 2 and 3, compared to untreated patients, for at least 24 months. Results: A total of 41 patients with SMA types 2 and 3 under nusinersen treatment were included. In 30 treated patients (mean age: 10.6 years; 14 with SMA type 2), the mean change in HFMSE scores was +1.47 points (SD = 0.4) and +1.60 points (SD = 0.6) after 12 and 24 months of treatment, respectively. In contrast, the control group (N= 37) (mean age: 10.2 years; 20 with SMA type 2) presented a mean change of -1.71 points (SD = 0.02) and -3.93 points (SD = 0.55) after 12 and 24 months of follow-up, respectively. The most severe patients under nusinersen treatment (N= 11) showed a change of +2.37 (SD = 1.13) on the CHOP-INTEND scale after 12 months of follow-up. Disease duration at the beginning of treatment was the main predictor of functional improvement. Despite functional gain and motor stabilization, treatment with nusinersen did not prevent the progression of scoliosis. Conclusions: Our data provide evidence for the long-term safety and efficacy of nusinersen use in the treatment of later-onset SMA, and patients with shorter disease duration showed better response to treatment.
bookPart Miopatias metabólicas e miopatias inflamatórias(2021) ZANOTELI, Edmar; MENDONçA, Rodrigo de Holanda- Motor unit number index (MUNIX) in children and adults with 5q-spinal muscular atrophy: Variability and clinical correlations(2021) MENDONCA, Rodrigo Holanda; MACHADO, Ligia Maria Sotero; HEISE, Carlos Otto; POLIDO, Graziela Jorge; MATSUI, Ciro; SILVA, Andre Macedo Serafim; REED, Umbertina Conti; ZANOTELI, EdmarSpinal muscular atrophy (SMA) is a motor neuron disease associated with progressive muscle weakness and motor disability. The motor unit number index (MUNIX) is a biomarker used to assess loss of motor units in later-onset SMA patients. Twenty SMA patients (SMA types 3 and 4), aged between 7 and 41 years, were clinically evaluated through the Hammersmith Motor Functional Scale Expanded and the Spinal Muscular Atrophy-Functional Rating Scale. The patients underwent compound motor action potential (CMAP) and MUNIX studies of the right abductor pollicis brevis, abductor digiti minimi and tibialis anterior (TA) muscles. Age-matched healthy controls (n = 20) were enrolled to obtain normative CMAP and MUNIX values from the same muscles. Compared to healthy controls, SMA patients showed significant reductions in MUNIX values among all muscles studied, whereas CMAP showed reductions only in the weaker muscles (abductor digiti minimi and TA). MUNIX variability was significantly higher in the SMA group than in the control group. MUNIX variability in TA correlated with CMAP variability. Motor functional scores correlated with TA MUNIX. The MUNIX study is feasible in later-onset SMA patients, and TA MUNIX values correlate with disease severity in patients with mild motor impairment.
- Child Neurology: A Case of FHL1-Related Disease Presenting as Inflammatory Myopathy(2021) SILVA, Andre Macedo Serafim; CAMELO, Clara Gontijo; MATSUI-JUNIOR, Ciro; MENDONCA, Rodrigo de Holanda; CAMPOS, Lucia Maria; ELIAS, Adriana Maluf; SILVA, Clovis Artur; REED, Umbertina Conti; ZANOTELI, Edmar
- Managing intrathecal administration of nusinersen in adolescents and adults with 5q-spinal muscular atrophy and previous spinal surgery(2021) MENDONCA, Rodrigo de Holanda; FERNANDES, Hermann Dos Santos; PINTO, Rafael Barbero Schimmelpfeng; MATSUI JUNIOR, Ciro; POLIDO, Graziela Jorge; SILVA, Andre Macedo Serafim da; GROSSKLAUSS, Luis Fernando; REED, Umbertina Conti; ZANOTELI, EdmarBackground: Spinal muscular atrophy (SMA) is a neurodegenerative disease of lower motor neurons associated with frequent occurrence of spinal deformity. Nusinersen is an antisense oligonucleotide that increases SMN protein level and is administrated by frequent intrathecal lumbar injections. Thus,spinal deformities and previous spinal surgery are important challenges for drug delivery in SMA.Objective: To report imaging methods used for Nusinersen injection in SMA patients. Methods: Nusinersen injection procedures in SMA types 2 and 3 patients who had previous spinal surgery were analyzed retrospectively to describe the imaging and puncture procedures, as well as the occurrence of complications. Results: Nine SMA patients (14 to 50 years old) underwent 57 lumbar punctures for nusinersen injection. Six patients had no interlaminar space available; in five of them, a transforaminal approach was used, and another one underwent a surgery to open a posterior bone window for the injections. Transforaminal puncture was performed using CT scan in three cases and fluoroscopy in the other two, with a similar success rate. One patient in the transforaminal group had post-procedure radiculitis, and another one had vagal reaction (hypotension). In three cases, with preserved interlaminar space, injections were performed by posterior interlaminar puncture, and only one adverse event was reported (post-puncture headache). Conclusion: In SMA patients with previous spinal surgery, the use of imaging-guided intervention is necessary for administering intrathecal nusinersen. Transforaminal technique is indicated in patients for whom the interlaminar space is not available, and injections should always be guided by either CT or fluoroscopy.
- Clinical Outcomes in Patients with Spinal Muscular Atrophy Type 1 Treated with Nusinersen(2021) MENDONCA, Rodrigo de Holanda; POLIDO, Graziela Jorge; MATSUI JR., Ciro; SOLLA, Davi Jorge Fontoura; REED, Umbertina Conti; ZANOTELI, EdmarBackground: Spinal muscular atrophy type 1 (SMA1) is a motor neuron disease associated with progressive muscle weakness, ventilatory failure, and reduced survival. Objective: To report the evaluation of the nusinersen, an antisense oligonucleotide, on the motor function of SMA1. Methods: This was a longitudinal and observational study to assess the outcomes of nusinersen therapy in SMA1 patients using the HINE-2 and CHOP-INTEND scales. Results: Twenty-one SMA1 patients (52.4% males) were included; the mean age at first symptoms was 2.7 months (SD = +/- 1.5), and the mean disease duration at first dose was 34.1 (SD = +/- 36.0) months. During posttreatment, the mean gain on the CHOP-INTEND was 4.9, 5.9, 6.6, and 14 points after 6, 12, 18, and 24 months, respectively. Starting medication with a disease duration of less than 12 months and/or without invasive ventilation were predictors of response on CHOP-INTEND. Of the patients, 28.6% acquired a motor milestone or gained at least three points on the HINE-2. The daily time for ventilatory support was reduced after treatment in most of the patients with noninvasive ventilation at baseline. No change in the daytime use of ventilation was observed in most of the patients using invasive ventilation at baseline. Conclusions: Nusinersen produces improvements in motor and respiratory functions, even in long-term SMA1 patients. However, patients under invasive ventilation at the beginning of the treatment experience little benefit.