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Outcome of salvage intra-arterial chemotherapy for recurrent retinoblastoma.

PURPOSE: To examine the outcome of salvage intra-arterial chemotherapy (IAC) for patients with recurrent retinoblastoma after the initial course of IAC and determine the factors influencing clinical outcome. METHODS: A total of 73 eyes of 71 patients with recurrent retinoblastoma undergoing salvage IAC after initial successfully IAC between May 2014 and May 2019 were retrospectively reviewed for clinical outcomes. Ocular survival and progression-free survival were used to examine the efficacy of salvage IAC. The factors influencing clinical outcomes were determined using univariate and multiv

Copy number evolution and its relationship with patient outcome-an analysis of 178 matched presentation-relapse tumor pairs from the Myeloma XI trial.

Structural chromosomal changes including copy number aberrations (CNAs) are a major feature of multiple myeloma (MM), however their evolutio … ent of acquired del(17p) in CCND2 over CCND1 tumors. Acquired CNAs were not influenced by high-dose melphalan or lenalidomide maintenance randomization. A branching evolution pattern was significantly associated with inferior overall survival (OS; hazard ratio (HR) 2.61, P = 0.0048). As an individual lesion, acquisition of gain(1q) at relapse was associated …

Long-term renal survival of γ3-heavy chain deposition disease: a case report.

BACKGROUND: Monoclonal immunoglobulin deposition disease (MIDD) is characterized by the non-amyloid deposition of monoclonal immunoglobulin … not observed. Urinary protein was not decreased and renal function further deteriorated. Therefore, melphalan plus prednisolone (MP) therapy was initiated. After 4 courses of MP therapy, the clinical parameters, including proteinuria, serum creatinine, albumin, and complement level (C3 and C4) were ameliorated. To date, the patient has been followed for 28 …

Novel approaches to the treatment of primary amyloidosis.

Primary (AL, amyloid light-chain) amyloidosis is a plasma cell disorder in which deposits of amyloid light-chain protein cause progressive o … macroglossia are other common features. The median survival is one to two years. Conventional-dose melphalan as standard treatment can prolong the median duration of survival by about ten months, but the clinical response rates with improvement of impaired organ function are low. Up-front high-dose chemotherapy with autologous peripheral blood stem cell tr …

[Long-term osteoporosis with multiple fractures as the early monosymptomatic stage of multiple myeloma].

A 44 year old man with kappa-light plasmacytoma is presented who had been treated for osteoporosis and multiple bone fractures since 12 years. On admittance he had a rapidly progressive myeloma kidney and an extensive extramedullary tumor within the pelvis. Remission was induced using combination chemotherapy according to the VMCP-protocol. It is discussed that the long-lasting osteoporosis of the present case represents an unusually long, monosymptomatic early stage of a multiple myeloma rather than an independent primary disease.

Therapy-related leukemia. A report of five patients and a review of the literature.

5 patients developed acute nonlymphocytic leukemia (ANLL) 2-4 years following the use of various cytotoxic agents for other primary disease. Alkylating agents were responsible for development of ANLL in 3 patients while mitomycin-C and methotrexate appear to be linked with leukemia transformation in the remaining 2 patients. 3 patients had a prolonged preleukemic phase preceding ANLL. Pancytopenia observed in 4 of 5 patients favors drug-induced stem cell damage leading to relatively resistant leukemia. Although the incidence of secondary leukemia is not very high, careful use of cytotoxic agen

Pediatric Nonmalignant Hematopoietic Cell Transplantation in the Era of Precision Conditioning and Gene Therapy.

The landscape of pediatric hematopoietic cell transplantation for nonmalignant disorders has evolved through disease-adapted conditioning, p … aplastic anemia approaches 98% 3-year survival. Fludarabine-based platforms combined with busulfan, melphalan, or treosulfan support durable engraftment with reduced toxicity, while matched unrelated, haploidentical, and cord blood donors expand access. GT now offers a parallel curative platform for select sickle cell disease, transfusion-dependent beta-tha …

Alternative conditioning regimens for hemoglobinopathy gene therapy: balancing efficacy, toxicity, and the next frontier.

Gene therapy for sickle cell disease and transfusion-dependent thalassemia is now widely available, yet its broader application is constrain … lity and potential genotoxicity. Emerging strategies aim to mitigate these risks. Reduced-intensity melphalan shows early promise but raises concerns regarding the durability of engraftment, whereas treosulfan offers a potentially safer profile, although data in the autologous setting are lacking. Antibody-based, nongenotoxic approaches represent a transfor …

How to treat hepatic extra-medullary involvement of myeloma: a case report and review of literature.

Extramedullary hepatic involvement is particularly rare. Here, we summarize published reports of hepatic myeloma treatment achieving infrequent and short-lived responses. Approach to a newly diagnosed multiple myeloma patient who presented with diffuse intrahepatic infiltration and cholestasis under frontline quadruplet induction will be presented here to complement the review. Contrary to earlier reports, he achieved a rapid response in/outside or marrow with elranatamab monotherapy. Importantly, this response enabled subsequent high dose melphalan and autologous stem cell transplantation (AS…

Culture-Proven Haemophilus influenzae Endophthalmitis After Intravitreal Topotecan for Retinoblastoma.

PURPOSE: To describe the management and clinical outcome of severe infectious endophthalmitis after intravitreal (IVT) topotecan injection for retinoblastoma in an eye with potential vision. METHODS: A single case was reviewed. RESULTS: An 8-month-old child with bilateral retinoblastoma receiving systemic chemotherapy developed acute endophthalmitis in the only eye with potential vision 3 days after the sixth IVT topotecan injection. Urgent 23-gauge pars plana vitrectomy was performed to clear dense vitritis and maintain oncologic control using a closed-system approach, melphalan irrigation …

Structural basis of anticancer drug recognition and amino acid transport by LAT1.

LAT1 (SLC7A5) transports large neutral amino acids and plays pivotal roles in cancer proliferation, immune response and drug delivery. Despi … rane helix 3 (TM3) and bending TM10. Physiological substrates like ʟ-Phe lack such effects, whereas melphalan poses steric hindrance, explaining its inhibitory activity. The "classical" system L inhibitor BCH induces an occluded state critical for transport, confirming its substrate-like behavior. These findings provide a structural basis for substrate reco …

Trend of treatment strategy for amyloid light-chain amyloidosis: a-single center experience.

Before 2000, AL amyloidosis was considered an incurable disease. However, in the 2000s, autologous peripheral blood stem cell transplantation therapy using vincristine + doxorubicin + dexamethasone with high-dose melphalan (VAD + SCT) was initiated. Bone marrow cells are the site of AL amyloid production. By normalizing bone marrow cells, VAD + SCT not only reduces amyloid production but also improves renal tissue damaged by amyloid deposition. However, VAD + SCT was only applied to patients who met the criteria, such as being under 65 years of age and not affecting the heart. At our hospital,…

Utility of routine pulmonary function test after autologous hematopoietic cell transplantation in lymphoma.

This study aims to evaluate the predictive value of routine pulmonary function testing (PFT) at the 12-month mark post-autologous hematopoietic cell transplant (AHCT) in identifying clinically significant lung disease in lymphoma survivors. In 247 patients, 173 (70%) received BEAM (carmustine, etoposide, cytarabine, melphalan), and 49 (20%) received TBC (thiotepa, busulfan, cyclophosphamide) conditioning regimens. Abnormal baseline PFT was noted in 149 patients (60%). Thirty-four patients had a significant decline (reduction of >/= 20% in DLCO or FEV1 or FVC) in post-AHCT PFT, with the highest…

Рефлексивный ГИЛЬЯЙН-БАРРЕ СИНДРОМ В ХОДЕ С АЗИМПТОМАТИЧЕСКИМ МНОГОЛЕМЕНТОМ МИЛОМЫ, С КОТОРЫМ БУДЕТ ОБРАЩАТЬСЯ С НИЗКИМ РИТЕКСИМАБОМ.

Несмотря на чрезвычайную редкость, синдром Гильен-Барре (ГБС) был признан неврологическим осложнением множественной миэломы с переменной реакцией на плазмеферез (PEX), иммуноглобулины (IVIG) и антимиеломную терапию. В настоящем документе сообщается о случае, когда пациентка с асимптоматической множественной миеломой (АММ), первоначально представлявшая себя как PEX- и IVIG-refractory GBS. После отказа PEX, IVIG и антимиеломатерапии (bortezomib, elphalan и padnison) пациент в конечном итоге успешно лечился ритуксимой с низкой дозой ().100 мг/м2 в неделю four Дозы). Насколько нам известно, это пе

Совпадение в аннотации оригинала
Despite being extremely rare, Guillain-Barré syndrome (GBS) has been recognized as a neurological complication of multiple myeloma, with var … as PEX- and IVIG-refractory GBS. After failure of PEX, IVIG, and anti-myeloma therapy (bortezomib, melphalan, and prednisone), the patient was eventually successfully treated with low-dose rituximab (100 mg/m2 per week in four doses). To the best of our knowledge, this is the first case to report successful treatment of refractory GBS potentially associate …

Multiple myeloma involving the gastrointestinal tract in an English Springer Spaniel.

A 10-year-old, entire male, English Springer Spaniel was referred for evaluation of weight loss, polyuria, polydipsia and gastrointestinal t … l plasma cell involvement. The dog entered complete clinical remission seven weeks after starting a melphalan/prednisolone protocol. The dog was euthanised 475 days after starting treatment due to cervical pain and collapse. At the time of euthanasia, blood work was not supportive of a relapse of multiple myeloma. To the authors' knowledge, this is the firs …

Efficacy of first-line treatment options in transplant-ineligible multiple myeloma: A network meta-analysis.

BACKGROUND: Despite major therapeutic advances, the rational choice of the most appropriate first-line regimen in newly diagnosed transplant … ival (OS). RESULTS: Based on surface under cumulative ranking curve values, daratumumab-bortezomib-melphalan-prednisone (Dara-VMP) and daratumumab-lenalidomide-dexamethasone (Dara-Rd28) showed superiority compared to other combinations regarding 12-, 24-, 36-, and 48-month PFS. Dara-VMP also ranked first for 12-, 24-, 36-, and 48-month OS. CONCLUSION: Our …

[Systemic varicella-zoster infection during ixazomib-containing multiagent chemotherapy for multiple myeloma].

A 58-year-old man received high-dose melphalan with autologous peripheral blood stem cell transplantation for multiple myeloma in stringent complete response (sCR). Relapse occurred 4 years after the transplantation, and he was placed on ixazomib, lenalidomide, and dexamethasone (IRd) and achieved sCR. On the 10th day of the 10th course of IRd, he developed fever followed by generalized skin eruption with vesicles, headache, and dizziness. Varicella-zoster virus (VZV) antigen from the vesicle and VZV-DNA from the cerebrospinal fluid were detected, and he was diagnosed with systemic VZV infecti…

Synthesis, cytotoxicity and carbonic anhydrase inhibitory activities of new pyrazolines.

A series of polymethoxylated-pyrazoline benzene sulfonamides were synthesized, investigated for their cytotoxic activities on tumor and non-tumor cell lines and inhibitory effects on carbonic anhydrase isoenzymes (hCA I and hCA II). Although tumor selectivity (TS) of the compounds were less than the reference compounds 5-Fluorouracil and Melphalan, trimethoxy derivatives 4, 5, and 6 were more selective than dimethoxy derivatives 2 and 3 as judged by the cytotoxicity assay with the cells both types originated from the gingival tissue. The compound 6 (4-[3-(4-methoxyphenyl)-5-(3,4,5-trimethoxyph…

How we treat systemic light-chain amyloidosis.

Systemic light-chain (AL) amyloidosis is a multisystem disease characterized by organ toxicity and damage due to monoclonal free light chains, which are produced by a neoplastic clone of plasma cells in bone marrow. Current treatment strategies target the clone in order to decrease the production of the pathologic light chains and thereby stop or reverse organ toxicity and damage. AL amyloidosis remains a formidable and often incurable disease despite treatment options that include corticosteroids, cytotoxic chemotherapy, risk-adapted melphalan, autologous hematopoietic stem cell transplantati…